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NCCN Guidelines for Patients Version 1.2016

Myelodysplastic
Syndromes
Presented with support from

Available online at NCCN.org/patients




NCCN Guidelines for Patients Version 1.2016

Myelodysplastic Syndromes
NCCN Foundation gratefully acknowledges support from the following individuals

NCCN and NCCN Foundation Boards of Directors

Drs. Al B. Benson III and Alanah Fitch Trisha Lollo


Joshua and Stephanie Bilenker Ray Lynch, CPA, MBA
Dr. and Mrs. Douglas W. Blayney James and Marilyn Mohler
Mara Bloom Lisle M. Nabell, MD
Rebecca Caires, MBA Michael and Gwyneth Neuss
Peter F. Coccia, MD and Phyllis I. Warkentin, MD Michael Parisi, MBA, MA
Gena Cook Barbara Parker, MD
Dr. and Mrs. Thomas A. DAmico Lori C Pickens, MHA
Dr. and Mrs. Timothy J. Eberlein Dorothy Puhy
Paul F. Engstrom Denise K. Reinke, MS, NP
David S. Ettinger, MD, FACP, FCCP Marc Samuels
Theresa J. Franco, RN, MSN Gerrie Shields
Brian Garofalo Dr. and Mrs. Samuel Silver
Jack A. Gentile, Jr. Susan C. & Robert P. Stein
Matt Kalaycio, MD The Honorable Ellen O. Tauscher
Mark F. Kochevar and Barbara Redmond Jeff and Colleen Walker
Heather Kopecky Robert C. Young, MD

NCCN and NCCN Foundation Staff Contributing $100 or More

Anonymous Lisa G. Kimbro, MBA, CPA


Robert W. Carlson, MD and Stacey Starcher Joan S. McClure
C. Lyn Fitzgerald, MJ Elizabeth Nardi
Kristina Gregory Marcie Reeder, MPH
Joseph Junod Gary J. and Marianne Weyhmuller

An additional fifty-five (55) donations were received from other NCCN staff members.

NCCN Guidelines for Patients


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Myelodysplastic Syndromes, Version 1.2016
NCCN Guidelines for Patients
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Myelodysplastic Syndromes, Version 1.2016

NCCN Guidelines for Patients Version 1.2016

Myelodysplastic
Syndromes (MDS)
Learning that you have cancer can be overwhelming. The goal of this book is
to help you get the best cancer treatment. It explains which cancer tests and
treatments are recommended by experts in myelodysplastic syndromes.

The National Comprehensive Cancer Network (NCCN) is a not-for-profit


alliance of 27 of the worlds leading cancer centers. Experts from NCCN have
written treatment guidelines for doctors who treat myelodysplastic syndromes.
These treatment guidelines suggest what the best practice is for cancer care.
The information in this patient book is based on the guidelines written for
doctors.

This book focuses on the treatment of myelodysplastic syndromes. Key points


of this book are summarized in the related NCCN Quick Guide. NCCN
also offers patient resources on chronic myelogenous leukemia, chronic
lymphocytic leukemia, acute lymphoblastic leukemia, and other cancer types.
Visit NCCN.org/patients for the full library of patient books, summaries, and
other patient and caregiver resources.

NCCN Guidelines for Patients


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Myelodysplastic Syndromes, Version 1.2016
Credits

NCCN aims to improve the care given to patients with cancer. NCCN staff work with experts to create helpful programs and resources for many
stakeholders. Stakeholders include health providers, patients, businesses, and others. One resource is the series of books for patients called
the NCCN Guidelines for Patients. Each book presents the best practice for a type of cancer. The patient books are based on clinical practice
guidelines written for cancer doctors. These guidelines are called the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines). Clinical
practice guidelines list the best health care options for groups of patients. Many doctors use them to help plan cancer treatment for their patients.
Panels of experts create the NCCN Guidelines. Most of the experts are from NCCN Member Institutions. Panelists may include surgeons,
radiation oncologists, medical oncologists, and patient advocates. Recommendations in the NCCN Guidelines are based on clinical trials and the
experience of the panelists. The NCCN Guidelines are updated at least once a year. When funded, the patient books are updated to reflect the
most recent version of the NCCN Guidelines for doctors. For more information about the NCCN Guidelines, visit NCCN.org/clinical.asp.

NCCN staff involved in making the guidelines for patients and doctors include:

NCCN Staff Lacey Marlow Rachael Clarke


Dorothy A. Shead, MS Associate Medical Writer Guidelines Data & Layout Coordinator
Director, Patient and Clinical Information NCCN Guidelines NCCN Marketing
Operations
Courtney Smith, PhD Susan Kidney
Laura J. Hanisch, PsyD Oncology Scientist/Medical Writer Graphic Design Specialist
Medical Writer/Patient Information Specialist

Endorsed and sponsored in part by


Aplastic Anemia and MDS International Foundation
As an organization that strongly supports educating patients and physicians about bone marrow failure
diseases like aplastic anemia, MDS, and PNH, the Aplastic Anemia and MDS International Foundation is
proud to sponsor this comprehensive resource for patients and their families. www.aamds.org

Endorsed by
The MDS Foundation, Inc. The Leukemia & lymphoma society (LLS)
The MDS Foundation, Inc. is pleased to endorse the NCCN Patient LLS is dedicated to developing better outcomes for
Guidelines: Myelodysplastic Syndromes (MDS) as an instrumental blood cancer patients through research, education and
resource to patients with MDS and caretakers. Providing a guide in patient services and is happy to have this comprehensive
a patient-friendly format offers patients access to understandable resource available to patients with MDS.
information and knowledge to assist them in making important www.LLS.org/informationspecialists
decisions about their care. www.mds-foundation.org

Supported by NCCN Foundation

The NCCN Foundation supports the mission of the National Comprehensive Cancer Network (NCCN)
to improve the care of patients with cancer. One of its aims is to raise funds to create a library of books for
patients. Learn more about the NCCN Foundation at NCCN.org/foundation.

2016 National Comprehensive Cancer Network, Inc. All rights reserved. The NCCN Guidelines for Patients and illustrations
herein may not be reproduced in any form for any purpose without the express written permission of NCCN.

National Comprehensive Cancer Network (NCCN)


275 Commerce Drive Suite 300
Fort Washington, PA 19034
215.690.0300

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Myelodysplastic Syndromes, Version 1.2016
Contents

Myelodysplastic
Syndromes (MDS)
4 How to use this book 63 Part 6
Making treatment decisions
5 Part 1 Offers tips for choosing the best treatment
MDS basics for you.
Explains how and where this type of
cancer starts. 73 Glossary:
74 Dictionary
15 Part 2 79 Acronyms
Testing for MDS
Describes the tests used to confirm this 82 NCCN Panel Members
type of cancer and plan treatment.
83 NCCN Member Institutions
25 Part 3
Prognostic scoring and risk 84 Index
groups
Describes how doctors rate and classify
the severity of MDS to help plan treatment.

31 Part 4
Overview of MDS treatments
Describes the types of treatments that may
be used to control MDS and its symptoms.

45 Part 5
Treatment guide
Presents treatment options based on the
risk group and symptoms.

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Myelodysplastic Syndromes, Version 1.2016
How to use this book

Who should read this book? The recommendations in this book are based on
science and the experience of NCCN experts.
The information in this book is about However, each patient is unique and these
myelodysplastic syndromesa group of cancers recommendations may not be right for you. Your
in which the bone marrow doesnt make enough doctors may suggest other tests or treatments
healthy, mature blood cells. Patients and those based on your health and other factors. If other
who support themcaregivers, family, and suggestions are given, feel free to ask your
friendsmay find this book helpful. It may help treatment team questions.
you discuss and decide with your doctors what
care is best.

Making sense of medical


terms
Where should I start
reading? In this book, many medical words are included.
These are words that you will likely hear from
Starting with Part 1 may be helpful for many your treatment team. Most of these words may
people. It explains what myelodysplastic be new to you, and it may be a lot to learn.
syndromes are. Knowing more about this cancer
may help you better understand its treatment. Dont be discouraged as you read. Keep reading
Parts 2 and 3 explain how doctors assess for and review the information. Be sure to ask your
this type of cancer and plan treatment. Part 4 treatment team to explain a word or phrase that
describes the types of treatments that may be you dont understand.
used. Part 5 is a guide to treatment options. Part
6 offers some helpful tips for anyone making Words that you may not know are defined in the
treatment decisions. text or in the Dictionary. Words in the Dictionary
are underlined when first used on a page.
Acronyms are defined in the text when first used
and are also defined in the Glossary. Acronyms
Does the whole book apply are words formed from the first letters of other
to me? words. One example is CBC for complete blood
count.
This book includes information for many
situations. Thus, you will likely not get every test
and treatment listed. Your treatment team can
point out what applies to you and give you more
information. As you read through this book, you
may find it helpful to make a list of questions to
ask your doctors.

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Myelodysplastic Syndromes, Version 1.2016
1
MDS basics

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Myelodysplastic Syndromes, Version 1.2016
1 MDS basics
7 What are blood cells?
8 What is MDS?
10 What are the types of MDS?
13 What are the symptoms of MDS?
14 Review

Youve learned that you have or may


have MDS. Part 1 explains some
basics about this cancer that may
help you learn about it and start to
cope. These basics may also help you
start planning for treatment.

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Myelodysplastic Syndromes, Version 1.2016
1 MDS basics What are blood cells?

What are blood cells?


Blood is made of many types of cells, called blood Blood stem cells go through a series of changes as
cells. The three main types of blood cells are they grow and develop into new blood cells. Blast
platelets, red blood cells, and white blood cells. cells are new, very young (immature) blood cells
Each type of blood cell has a different job. Platelets that grow into adult (mature) blood cells over time.
help control bleeding. Red blood cells carry oxygen Different types of blast cells become different types
throughout the body. White blood cells help fight of mature blood cells. Once they are mature, the
germs and infections. blood cells leave the bone marrow and enter the
bloodstream.
Blood cells are made from immature blood-forming
cells in the bone marrow. Bone marrow is the soft
tissue in the center of most bones. See Figure 1.1.
These blood-forming cells are called blood stem cells
or hematopoietic stem cells. Blood stem cells can
become any type of mature blood cell.

Figure 1.1
Blood cells in bone marrow

Bone marrow is the soft, sponge-like tissue in the center of most bones. Blood stem cells in the
bone marrow make all types of blood cells.

Illustration Copyright 2016 Nucleus Medical Media, All rights reserved. www.nucleusinc.com

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1 MDS basics What is MDS?

What is MDS?
Cancer is a disease of cells. MDS (myelodysplastic of DNA (deoxyribonucleic acid). DNA is grouped
syndromes) is a group of cancers that affect blood together into long strands called chromosomes. See
cells in the bloodstream and bone marrow. MDS Figure 1.2. Changes (mutations) in genes can cause
starts in the blood-forming cells (blood stem cells) of normal cells to become cancer cells. Exactly what
the bone marrow. causes the gene changes is not known.

Normal blood stem cells grow and then divide to MDS happens when changes in genes cause blood
make new red blood cells, white blood cells, and stem cells to become abnormal. This damages the
platelets as the body needs them. When the cells blood stem cells and bone marrow so they dont work
grow old or get damaged, they die. New blood cells as well as they should. The abnormal blood stem
are then made to replace the old ones. cells have trouble making enough new blood cells for
the body. They also make new blood cells that are
Inside of all cells are coded instructions for building abnormal (defective).
new cells and controlling how cells behave. These
instructions are called genes. Genes are a part

Figure 1.2
Chromosomes and genes in cells

Genes are coded instructions in cells for making new cells and controlling how cells behave.
Genes are a part of DNA, which is bundled into long strands called chromosomes. Every cell has
23 pairs of chromosomes.

Illustration Copyright 2016 Nucleus Medical Media, All rights reserved. www.nucleusinc.com

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1 MDS basics What is MDS?

The defective blood cells are different from normal MDS may continue to get worse over time. In a small
blood cells in a few key ways: number of cases, it may progress to a fast-growing
(aggressive) cancer called AML (acute myeloid
The cells have an abnormal size, shape, or leukemia). This can happen as more and more blast
look (appearance). This is called dysplasia. cells fill up the bone marrow. However, most patients
The cells do not grow into normal, mature with MDS do not develop AML.
blood cells and do not leave the bone marrow
as they should.
The cells may remain immature, as blast cells,
and not leave the bone marrow.
The cells may die too early in the bone
marrow or soon after they enter the
bloodstream.

Because of this damage, the bone marrow isnt


able to make enough healthy blood cells that the
body needs. The defective cells can build up and
overcrowd the bone marrow. As a result, even fewer
healthy blood cells may be made or survive. This
leads to a low number of red blood cells, white blood
cells, and/or platelets in the bloodstream.

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1 MDS basics What are the types of MDS?

What are the types of MDS? MDS subtypes in the WHO classification
system
MDS is divided (classified) into smaller groups based
on certain features of the bone marrow and blood MDS-UD
cells. These smaller groups are called subtypes. (MDS with unilineage dysplasia)
There are two systems that can be used to classify In this subtype, there is a low number of one type
the MDS subtypes. of blood cells in the bloodstream. The same type of
blood cells looks abnormal in the bone marrow. For
The first one was created in 1982 and is called the the affected cell type, at least 10 percent of the cells
FAB (French-American-British) classification system. look abnormal (show dysplasia). No blast cells are
The FAB system groups MDS into five subtypes. found in the bloodstream. Less than 5 percent of cells
This system is not used much today. The newer in the bone marrow are blast cells.
one is called the WHO (World Health Organization)
classification system. The 2016 Revision to the WHO MDS-RS
system groups MDS into seven main subtypes. The (MDS with ring sideroblasts)
WHO system also has a category called MDS/MPN In this subtype, there is a low number of red blood
(myelodysplastic/myeloproliferative neoplasms) with cells in the bloodstream. Red blood cells are also
four other subtypes. abnormal in the bone marrow. At least 15 percent of
young red blood cells in the bone marrow are ring
The WHO system is most commonly used today. sideroblasts. Less than 5 percents of cells in the bone
Most doctors use this system to diagnose MDS and marrow are blast cells. No blast cells are found in
classify MDS subtypes. The main factors that are the bloodstream. There is a normal number of white
used to define the MDS subtypes in the WHO system blood cells and platelets in the bloodstream and these
include: cells look normal in the bone marrow.

The type and number of low blood cell counts MDS-MD


(cytopenias), (MDS with multilineage dysplasia)
Which and how many types of blood cells In this subtype, there is a low number of one or more
in the bone marrow have an abnormal size, types of blood cells in the bloodstream. Two or more
shape, or look (dysplasia), types of blood cells look abnormal in the bone marrow.
The number of blast cells found in the blood Of the affected cell types, at least 10 percent of the
and bone marrow, cells look abnormal. Some young blood cells in the
The types of chromosome changes seen in bone marrow may be ring sideroblasts. Overall, less
bone marrow cells, and than 5 percent of cells in the bone marrow are blast
The presence of red blood cells that have too cells. No blast cells are found in the bloodstream.
much iron (ring sideroblasts).

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1 MDS basics What are the types of MDS?

MDS-EB1 MDS-EB-T
(MDS with excess blasts-1) (MDS with excess blasts in transformation)
In this subtype, one or more types of blood cells are In this subtype, the number of blast cells is much
low in the bloodstream and also look abnormal in the higher than normal. In the bloodstream, 5 to 19
bone marrow. The number of blast cells is slightly percent of cells are blast cells. In the bone marrow,
higher than normal. Less than 5 percent of cells in 20 to 30 percent of cells are blast cells. One or more
the bloodstream are blast cells. In the bone marrow, 5 types of blood cells are low in the bloodstream and
percent to 9 percent of cells are blast cells. also look abnormal in the bone marrow. (Please
note: This subtype is no longer included in the WHO
MDS-EB2 classification of MDS. But, NCCN experts suggest
(MDS with excess blasts-2) that this subtype be treated as MDS or AML based on
In this subtype, one or more types of blood cells are certain factors. Thus, MDS-EB-T is still included as
low in the bloodstream and also look abnormal in the a subtype of MDS in this book and in the treatment
bone marrow. The number of blast cells is higher than guidelines written for doctors.)
normal. Five to 19 percent of cells in the bloodstream
are blast cells and 10 to 19 percent of cells in the
bone marrow are blast cells.

MDS-U
(MDS, unclassified)
In this subtype, the features of the blood and bone
marrow dont fit any of the other subtypes. One or
more types of blood cells are low in the bloodstream,
but less than 10 percent of that cell type looks
abnormal in the bone marrow. Very few or no blasts
are found in the bloodstream on at least 2 occasions
and less than 5 percent of cells in the bone marrow
are blast cells. Cells in the bone marrow have at least
one abnormal chromosome change that is linked with
MDS.

MDS associated with isolated del(5q)


In this subtype, cells in the bone marrow have only
one abnormal chromosome change. This change is
called del(5q), which means that part of chromosome
5 is missing (deleted). There is a low number of red
blood cells in the bloodstream and the number of
platelets is normal or high. Less than 5 percent of
cells in the bone marrow are blast cells.

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1 MDS basics What are the types of MDS?

MDS/MPN subtypes in the WHO JMML


classification system (juvenile myelomonocytic leukemia)
In this subtype, there is a high level of monocytes
The MDS/MPN category includes subtypes that have in the bloodstream and bone marrow. Less than 20
both dysplastic and proliferative features. Dysplastic percent of cells in the bloodstream are blast cells.
refers to the bone marrow making blood cells that This subtype is a lot like CMML. The main difference
look and act abnormal. Proliferative refers to the bone is that JMML most often occurs in young children.
marrow making too many blood cells. The subtypes in
the MDS/MPN category are described next. MDS/MPN
(MDS/myeloproliferative neoplasm),
CMML-1 unclassifiable
(chronic myelomonocytic leukemia-1) This subtype is also referred to as Overlap
In this subtype, there is a high level of white blood syndrome. There is a high level of one or more types
cells called monocytes in the bloodstream. One or of blood cells in the bloodstream and bone marrow.
more types of blood cells in the bone marrow look One or more types of blood cells also look abnormal.
abnormal (show dysplasia). Less than 5 percent of But, the features of the blood and bone marrow dont
cells in the bloodstream are blast cells. And, less than fit any of the other MDS/MPN subtypes.
10 percent of cells in the bone marrow are blast cells.
MDS/MPN-RS-T
CMML-2 (MDS/myeloproliferative neoplasm with ring
(chronic myelomonocytic leukemia-2) sideroblast and thrombocytosis)
In this subtype, there is a high level of monocytes in In this subtype, there is a high level of one or more
the bloodstream. One or more types of blood cells in types of blood cells in the bloodstream and bone
the bone marrow look abnormal. In the bloodstream, marrow. At least 15 percent of young red blood cells
5 to 19 percent of cells are blast cells. In the bone in the bone marrow are ringed sideroblasts along with
marrow, 10 to 19 percent of cells are blast cells. a platelet count.

Atypical CML
(chronic myeloid leukemia), BCR-ABL1 negative
In this subtype, there is a high level of white blood
cells in the bloodstream. More than 10 percent of
the cells are very young neutrophils. But, less than
20 percent of cells in the bloodstream are blast
cells. There is a higher-than-normal number of blood
cells in the bone marrow. Less than 20 percent of
cells in the bone marrow are blasts. This subtype
has cells that look similar to CML when viewed with
a microscope. But, the cells lack the chromosome
and gene changes found in typical CML. The key
changes in typical CML are the abnormal Philadelphia
chromosome and the BCR-ABL1 gene. This subtype
is not treated the same as typical CML.

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1 MDS basics What are the symptoms of MDS?

What are the symptoms of A low number of healthy platelets is called


MDS? thrombocytopenia. Platelets help control bleeding
and help wounds heal. A low number of platelets can
People with MDS often have low levels of one or cause symptoms such as easy bruising and bleeding.
more types of blood cells in their bloodstream. A low
number of blood cells is called a cytopenia. Most But, MDS is a slow-growing cancer and may not
symptoms of MDS are caused by not having enough cause symptoms early. These symptoms can also be
healthy red blood cells, white blood cells, or platelets caused by many other common health conditions.
in the bloodstream.

A low number of healthy red blood cellscalled


anemiais very common. Red blood cells carry
oxygen throughout the body. A low number of red
blood cells can cause symptoms such as extreme
tiredness (fatigue), dizziness, weakness, shortness of
breath, and pale skin.

A low number of healthy white blood cells is called


neutropenia. White blood cells help the body fight
germs and infections. Thus, neutropenia can lead to
frequent or severe infections.

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Myelodysplastic Syndromes, Version 1.2016
1 MDS basics Review

Review
Blood cells are made in the soft tissue in the In MDS, the bone marrow makes abnormal
center of most bones called bone marrow. blood cells and doesnt make enough healthy,
mature blood cells for the body.
A blood stem cell is a cell from which all other
types of blood cells are made. MDS is divided into smaller groups based
on the features of the bone marrow and
MDS is a group of cancers that affect blood
blood cells. These smaller groups are called
cells in the bone marrow and bloodstream.
subtypes.

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2
Testing for MDS

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Myelodysplastic Syndromes, Version 1.2016
2 Testing for MDS
16 Medical history
17 Physical exam
18 Blood tests
20 Bone marrow biopsy and aspiration
22 Genetic tests
24 Review

Medical history
Treatment planning starts with testing.
Your medical history includes any health events in
This section describes the tests that your life and any medicines youve taken. A medical
are used to check for and confirm history is needed for treatment planning. See Chart
2.1 for a full list of the tests that are recommended
(diagnose) MDS. These tests also
before treatment for MDS. Your doctor will ask about
help doctors plan treatment. This illnesses, injuries, and health problems that you have
information can help you use the had. This includes the timing and severity of any low
blood cell counts (cytopenias). Your doctor will also
Treatment guide in Part 5. It may also ask about prior infections, abnormal bleeding, and the
help you know what to expect during number of blood transfusions youve had.
testing.
Your doctor will also ask about any symptoms youve
had that may be due to MDS. This information may
affect which cancer treatment is best for you. It may
help to make a list of old and new medicines while at
home to bring to your doctors office.

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Myelodysplastic Syndromes, Version 1.2016
2 Testing for MDS Physical exam

Physical exam
Doctors usually perform a physical exam along with
taking a medical history. A physical exam is a review
of your body for signs of disease such as infection
and areas of unusual bleeding or bruising.

Your doctor may listen to your lungs, heart, and


intestines. Your doctor may also feel different parts of
your body to see if organs are of normal size, are soft
or hard, or cause pain when touched.

Chart 2.1 Tests for MDS

Needed for all patients May be useful for some patients

Medical history and physical exam Copper level

CBC (complete blood count) with differential HIV (human immunodeficiency virus) testing

Reticulocyte count HLA (human leukocyte antigen) typing

Blood smear HLA-DR15 typing

Serum EPO (erythopoietin) Flow cytometry

Iron, ferritin, folate, and vitamin B12 FISH (fluorescence in situ hybridization)

Assess for thyroid problems Molecular testing

Bone marrow biopsy and aspiration

Cytogenetic testing

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Myelodysplastic Syndromes, Version 1.2016
2 Testing for MDS Blood tests

Blood tests Reticulocyte count


Reticulocytes are younger (precursor) cells that
Doctors test blood to look for signs of disease and become mature red blood cells. The reticulocyte
to check your general health. Blood tests are done count is a measure of the number of reticulocytes in
along with other initial tests to help diagnose MDS. the bloodstream. It reflects how quickly reticulocytes
For a blood test, your doctor will insert a needle are being made and released by the bone marrow.
into a vein to remove a sample of blood. The blood This test is used to show if your bone marrow is
sample will then be sent to a lab for testing. At the lab, making the right number of new red blood cells. It can
a pathologist will examine the blood sample with a also help doctors find out the cause of anemia.
microscope and perform other tests.
Anemia is a low number of healthy red blood cells
CBC with differential in the bloodstream. The bodys normal response
A CBC (complete blood count) is a test that measures to anemia is for the bone marrow to make more
the number of blood cells in a blood sample. It reticulocytes. A low reticulocyte count is a sign that
includes the number of white blood cells, red blood the bone marrow isnt working well.
cells, and platelets. Patients with MDS often have a
low number of one or more types of blood cells. A low Blood smear
number of blood cells is called a cytopenia. For a blood smear, a drop of blood is placed on
a slide so it can be viewed with a microscope. A
The CBC should include a differential. The differential pathologist will assess the size, shape, type, and
measures the different types of white blood cells in maturity of the blood cells. This test can help count
the sample. Some types of white blood cells include the different types of blood cells in a sample. It is
neutrophils, monocytes, and lymphocytes. also used to look for defects in blood cells such as an
abnormal shape or size (dysplasia). A key feature of
A CBC with differential is given with other tests when MDS is that is causes blood cells to look abnormal.
MDS is first suspected. This test is not used alone
to diagnose MDS, but it can tell your doctor about This test may also be used to check for blast cells in
your overall health. It will show if you have the right the bloodstream. Normally, blast cells are only found
number of healthy red blood cells, white blood cells, in the bone marrow. But, in some cases of MDS,
and platelets. It can also guide other tests that may a small number of blast cells may be found in the
be needed to find out why blood cell counts are low. bloodstream.

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2 Testing for MDS Blood tests

Other tests to assess the cause of Track your health information and
low blood cell counts
blood test results in one place. This
A number of health conditions can also cause a low
number of red blood cells and other problems similar can help you understand how your
to MDS. To confirm MDS, more blood tests will be MDS is progressing and how treatment
done to rule out other health conditions. The main is working. Being able to put your
tests that may be used to learn more about the cause
of low blood cell counts are described next.
hands on your health records quickly
is a critical part of being a strong self-
Serum EPO advocate.
EPO (erythropoietin) is a substance that tells
(stimulates) the bone marrow to make more red - Patient Advocate
blood cells. The body normally makes more EPO in AAMDS International Foundation
response to low levels of oxygen, which is carried
in red blood cells. Measuring the amount of EPO in Assess for thyroid problems
the blood can help find out the cause of anemia. The The thyroid makes hormones that help control how
amount of EPO in the blood is called serum EPO. The fast the body uses energy. The hormones also affect
serum EPO level is measured in mU/mL (milliunits other body functions. An underactive thyroidwhen it
per milliliter). isnt making enough hormonescan lead to anemia.
Thus, a test of the amount of TSH (thyroid-stimulating
A low level of EPO can cause anemia and may be a hormone) in the blood may be used to check how well
sign of a health problem other than MDS. A low level the thyroid is working. A high level of TSH in the blood
of EPO can also make anemia worse in a person with can be a sign that the thyroid isnt making enough
MDS. Most patients with MDS-related anemia have hormones.
relatively low serum EPO levels.
Copper level
Iron, ferritin, folate, and vitamin B12 Copper is a mineral that helps with many processes
Iron is a mineral that the body needs to make red in the body. A low level of copper can cause the
blood cells. Iron is an important part of hemoglobin number of red blood cells and white blood cells to be
the protein in red blood cells that carries oxygen. low. It can also cause blood cells to have an abnormal
Ferritin is a protein that binds to iron and stores it for size, shape, or look. Assessing the level of copper in
use in the body. The amount of ferritin in the blood the blood is not a standard test for MDS. But, it may
reflects the amount of iron stored in the body. be done in certain cases to rule out other causes of
the abnormal appearance or number of blood cells.
Folate and vitamin B12 are other nutrients in the body
that are needed to make red blood cells. A shortage HIV testing
of any one of these substances can cause anemia. A HIV (human immunodeficiency virus) can cause
shortage of folate or vitamin B12 can also cause red low blood cell counts. It can also cause blood cells
blood cells to have an abnormal shape, size, or look. to have an abnormal size, shape, or look. In certain
cases, tests may be done to rule out HIV as the
cause of these symptoms.

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HLA typing Bone marrow biopsy and


HLAs (human leukocyte antigens) are special aspiration
proteins found on the surface of most cells in the
body. The unique set of HLA proteins on a persons To confirm MDS, a sample of bone marrow must be
cells is called the HLA type or tissue type. All cells removed from your body for testing. A bone marrow
in a single person have the same HLA type. This biopsy removes a small piece of solid bone along with
helps the body to tell its own cells apart from foreign a small amount of soft bone marrow inside the bone.
cells. It also affects how the body responds to foreign A bone marrow aspiration removes a small amount of
substances. liquid bone marrow from inside the bone. Often, both
tests are done at the same time on the back of the hip
HLA typing is a blood test that finds a persons HLA bone. You will likely lie on your side during this test.
type. This test is not needed for all patients with MDS. See Figure 2.1.
It is only used in certain cases, such as to find the
right donor for treatment with a platelet transfusion or You may be given a light sedative before the test.
hematopoietic cell transplant. This is because your Your doctor will then clean the area of skin where
tissue type and the donors tissue type must be a the biopsy will be done. Next, you will receive local
near-perfect match for this treatment to work. anesthesia to numb the area of skin and bone
beneath. Once numb, a hollow needle will be inserted
HLA-DR15 typing into your skin and then pushed into the bone to
HLA-DR15 is a protein that is sometimes found on the remove the liquid bone marrow with a syringe. Then,
surface of MDS cells. This test is done on a sample a wider needle will be inserted into the bone and
of blood to check for the HLA-DR15 protein. It is not twisted to remove the solid bone and marrow sample.
required for all patients, but it may be used in some You may feel some pain while the samples are being
cases to help guide treatment options. Certain types removed. Your skin may be bruised for a few days.
of treatments that suppress the immune system may The samples will be sent to a lab for testing.
work better against MDS cells that have this protein.
Bone marrow tests
Flow cytometry At the lab, a pathologist will view the bone marrow
Flow cytometry looks at the proteins on the surface of samples with a microscope. A pathologist is a doctor
cells in a sample of blood or bone marrow. In some whos an expert in testing cells and tissue for signs
cases of MDS, this test may be used to identify the of disease. He or she will assess the size, shape,
specific type of cells present. type, structure, and maturity of the cells in the
sample. Doctors may refer to this as a morphologic
assessment.

During this assessment, the pathologist will note any


signs of MDS, such as:

Cells that have an abnormal size or shape


(dysplasia),
An abnormal numbertoo many or too few
of any types of blood cells,

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An increased number of young cells or very The number of blast cells in the bone marrow is
young cells (blast cells), important. In a healthy person, less than 5 percent
More than the normal number of cells in the of cells in the bone marrow are blast cells. This
bone marrowcalled hypercellular bone means that less than 5 out of every 100 bone marrow
marrow, cells are blast cells. In a person with MDS, up to 19
Less than the normal number of cells in the percent of cells in the bone marrow may be blast
bone marrowcalled hypocellular bone cells. In some cases, MDS can progress to a more
marrow, and aggressive (fast-growing) cancer called AML. In AML,
Red blood cells that have too much or too little more than 19 percent of cells in the bone marrow are
iron. blast cells.

A key feature of MDS is that it causes dysplasia in one But, the cell assessment and iron stain are not the
or more types of blood cells. In some types of MDS, only tests that will be done on the bone marrow
red blood cells have too much iron. To check for this, samples. Tests of genes and chromosomes will also
a special dye called an iron stain is placed on the be done. These are called genetic tests and they are
sample of liquid bone marrow. The excess iron shows described in the next section.
up as small dots in a circle (ring) around the center of
the cells. Thus, these cells are called ring sideroblasts.

Figure 2.1
Bone marrow biopsy

Doctors use a bone marrow biopsy and aspiration to remove a sample of bone marrow for testing.
These tests are often done at the same time on the hip bone.

Illustration Copyright 2016 Nucleus Medical Media, All rights reserved. www.nucleusinc.com

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Genetic tests
Cytogenetic testing
Cytogenetic testing uses a microscope to examine
the chromosomes inside of cells. This type of test is
used to look for abnormal changes (defects) in the
chromosomes. It is often done on a sample of bone
marrow. It can also be done on a sample of blood.

For this test, a pathologist will look at a map of the


chromosomes under a microscope. This map is called
a karyotype. It will show if there are any abnormal
changes in the size, shape, structure, or number of
chromosomes.

There are many types of chromosome defects. Part


of a chromosome, or a whole chromosome, may
be missing. Or, there may be an extra copy of a
chromosome. Doctors use symbols and shortened
terms to describe the different types of chromosome
changes. A missing chromosome or missing part of a
chromosome is noted by a minus sign (-) or the word
del for deletion. An extra copy of a chromosome is
noted by a plus sign (+). For example: FISH
FISH (fluorescence in situ hybridization) uses special
-7 and del(7) both mean that a copy of dyes to find certain changes in a cells genes and
chromosome 7 is missing. chromosomes. It can be done on a sample of blood
del(7q) and 7q- both mean that the q part or bone marrow. This test can be used to check for
(long arm) of chromosome 7 is missing. The specific gene or chromosome changes that affect
p part is the short arm of the chromosome. the likely outcome of MDS. Knowing if the MDS cells
+7 means that there is an extra copy of have certain gene changes can help guide treatment
chromosome 7. choices. FISH is not used to confirm MDS and may
not be needed for all patients.
It is common for MDS cells to have abnormal
chromosomes. About half of patients with MDS have Molecular testing
one or more chromosome defects. In MDS cells, Molecular tests look for mutations in genes known
defects are commonly found in chromosomes 5, 7, to have an effect on cancer treatment or outcomes.
8, and 20. The type and number of chromosome Molecular testing can be done on a sample of blood
changes helps doctors assess the likely outcome or bone marrow removed from your body. Molecular
(prognosis). This information can also help guide testing may be done in some patients with MDS to
treatment options. look for the following gene changes.

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Recurrent gene mutations PDGFR gene mutation


There are certain gene mutations that are quite Doctors may use molecular tests to look for certain
common in MDS. These are called recurrent gene changes to the PDGFR (platelet-derived growth
mutations. DNA sequencing is a type of molecular factor receptor beta) gene. Changes to this gene
test that checks for specific gene mutations in cells. sometimes happen in a subtype of MDS called
Doctors use this test to find out which recurrent gene CMML. (See page 10 for details about subtypes.)
mutations are present in MDS cells. Certain mutations Certain treatments may work better against CMML
are linked with a better or worse prognosis. Thus, that has this gene mutation. Thus, knowing if cells
doctors may test for these common mutations to help have a PDGFR gene mutation can help guide
plan treatment. treatment options for some patients.

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3
2 Testing
DCIS for MDS Review
Genetic counseling | Treatment

Review
A medical history, physical exam, and blood Genetic tests check for abnormal changes in
tests can reveal signs of cancer. the genes and chromosomes of MDS cells.
They can be used to assess prognosis and
Different types of blood tests are done to
guide treatment choices.
assess the extent and cause of low blood cell
counts. Blood tests are also used to see if you
have an illness other than cancer.

A bone marrow biopsy removes a piece of


bone and marrow to test for cancer cells.
A bone marrow aspiration removes liquid
marrow. These tests are needed to confirm
MDS. Tests of bone marrow are also used to
assess the prognosis of MDS.

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3
Prognostic scoring and
risk groups

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and risk groups
27 Prognostic factors
27 Prognostic scoring systems
29 Risk groups: Lower risk versus
higher risk
30 Review

Prognostic scoring is how doctors


rate the severity of MDS. It is used to
assess the likely outcome (prognosis)
of MDS and plan treatment. The
rating, called a risk score, is used to
classify MDS into risk groups. Part 3
describes the key factors and scoring
systems that are used for MDS.

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Prognostic factors Prognostic scoring systems


A prognosis is a prediction of the pattern and outcome Doctors use a points system to rate the severity of
of a disease. As part of treatment planning, your MDS in each patient based on certain prognostic
doctors will assess the prognosis of MDS. A key aspect factors. This is called prognostic scoring. Prognostic
of the prognosis of MDS is the chance (risk) that it will scoring systems help predict outlook (such as
progress to AML. There are certain factors that affect survival) and the risk of progression to AML. They
the prognosis of MDS. These are called prognostic assign a risk score and risk group for MDS based on
factors. Doctors use these factors to help decide the prognostic factors.
if cancer treatment is needed right away and how
intensive treatment needs to be. Such factors include: First, each factor is given a score based on its
severity. A lower score means a better outlook. The
The MDS subtype, scores for all of the factors are then added together to
The number and severity of low blood cell make the overall risk score. The risk score describes
counts (cytopenias), how slow or fast MDS will likely grow and progress to
The percent of blast cells in the bone marrow, AML if not treated.
and
The type and number of chromosome The risk score is used to assign the risk group. A risk
changes. group includes cases of MDS that have the same or
similar risk scores. Cases of MDS in the same risk
Some factors are linked with better outcomes or a group will behave in a similar way. Doctors use the
lower chance that MDS will turn into AML. Other risk group to decide what type of treatment to use and
factors are linked with poorer outcomes or a higher when.
chance that MDS will turn into AML. Based on these
prognostic factors, doctors use a scoring system to There are three main prognostic scoring systems for
rate and classify the severity of MDS. MDS:

IPSS (International Prognostic Scoring


System)
IPSS-R (Revised International Prognostic
Scoring System)
WPSS (WHO classification-based Prognostic
Scoring System)

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IPSS IPSS-R
The IPSS was the first prognostic scoring system The IPSS-R was developed in 2012. It is an updated
for MDS to be broadly used. It was created almost (revised) version of the original IPSS. A key way the
20 years ago. Currently, it is still the most commonly IPSS-R differs is that it scores the types and severity
used scoring system. of low blood cell counts. It also scores a wider range of
chromosome changes. And, it classifies MDS into five
The IPSS scores three main factors to classify MDS risk groups instead of four. As a result, the IPSS-R is
into four risk groups. See Chart 3.1. The scores for all better at predicting prognosis than the IPSS.
of the factors are added together to make the overall
risk score. This is called the IPSS score and it is used This system gives points for five main prognostic
to assign the IPSS risk group. factors. See Chart 3.1. The points for each of the
factors are added together to make the IPSS-R score
and assign the IPSS-R risk group.

Chart 3.1 Prognostic scoring systems and risk groups

System Prognostic factors that are scored Risk groups based on total risk score

Percent of blast cells in bone marrow Low IPSS Risk score = 0


Score of 0, 0.5, 1.5, or 2 points
Chromosome changes Intermediate-1 IPSS Risk score = 0.5 to 1.0
IPSS
Score of 0, 0.5, or 1 point Intermediate-2 IPSS Risk score = 1.5 to 2
Number of low blood counts
Score of 0 or 1 point High IPSS Risk score = 2.5 or higher

Percent of blasts in the bone marrow Very Low IPSS-R Risk Score = 1.5 or lower
Score of 0, 1, 2, or 3 points
Chromosome changes Low IPSS-R Risk Score = 2 to 3
Score of 0, 1, 2, 3, or 4 points
Hemoglobin level
IPSS-R Intermediate IPSS-R Risk Score = 3.5 to 4.5
Score of 0, 1, or 1.5 points
Platelet count
High IPSS-R Risk Score = 5 to 6
Score of 0, 0.5, or 1 point
Neutrophil count
Score of 0 or 0.5 points Very High IPSS-R Risk Score = 6.5 or higher

Very Low WPSS Risk Score = 0


MDS subtype
Score of 0, 1, 2, or 3 points Low WPSS Risk Score = 1
Chromosome changes
WPSS Intermediate WPSS Risk Score = 2
Score of 0, 1, or 2 points
Presence of severe anemia High WPSS Risk Score = 3 to 4
Score of 0 or 1 point
Very High WPSS Risk Score = 5 to 6

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WPSS Higher-risk MDS is more likely to progress faster or


The WPSS is also a newer scoring system. But, it is turn into AML more quickly without treatment. It may
not used as often as the IPSS or IPSS-R. A key way cause more symptoms and health problems in a short
the WPSS differs from the other two systems is that amount of time. Thus, more intensive treatment is
it includes the MDS subtype as a prognostic factor. often needed.
As for low blood cell counts, the WPSS gives a score
based on the presence or absence of severe anemia. Doctors define risk groups with information from
large groups of patients. Thus, a risk group gives
Like the IPSS-R, this system also has five risk an average risk. Some people will do better than
groups. In this system, points are given for three main expected. Others will do worse. Other factors
prognostic factors. See Chart 3.1. The points for each not used for assigning risk groups are also very
of the factors are added together to make the overall important. Such factors include your age, general
risk score. This is called the WPSS score and it is health, ability to do daily activities, and other health
used to assign the WPSS risk group. conditions.

A key point to remember is that these scoring


systems and risk groups do not predict how MDS
Risk groups: Lower risk versus will respond to treatment. They only help predict how
higher risk MDS will likely behave over time without treatment.

When planning treatment, doctors often look at Risk groups also give doctors a general idea of how
the risk groups in terms of lower-risk and higher- long a group of patients might live with MDS without
risk MDS. Lower-risk MDS includes certain risk treatment. This does not apply to patients who
groups from each of the scoring systems. Likewise, receive treatment for MDS. It also does not predict
higher-risk MDS includes risk groups from each of how long a specific patient might live. Some patients
the scoring systems. Chart 3.2 shows how the risk will live many more years and others will live fewer
groups are split into these two main categories. years. If you want to learn more, talk to your doctor or
nurse. They will be able to give you information that is
In general, lower-risk MDS is more likely to grow and specific to you.
progress slowly over time. It may not cause many or
severe symptoms for a long time. Thus, less intensive
treatment is often used.

Chart 3.2 Risk groups: Lower-risk versus higher-risk MDS

Lower-risk MDS Higher-risk MDS

IPSS Low and Intermediate-1 IPSS Intermediate-2 and High


IPSS-R Very Low, Low, Intermediate IPSS-R Intermediate, High, Very High
WPSS Very Low, Low, Intermediate WPSS High, Very High

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Review
A prognosis is a prediction of the pattern and MDS is classified into risk groups based on
outcome of disease. the risk score. Doctors use risk groups to
decide what type of treatment is needed and
Something that affects and helps predict
when.
the likely outcome of a disease is called a
prognostic factor. When planning treatment, doctors look at the
risk groups in terms of lower-risk MDS and
Doctors use prognostic scoring systems
higher-risk MDS.
to rate the severity of MDS to help plan
treatment.

A risk score is a rating of the severity of MDS.


It describes how slow or fast MDS will likely
grow and progress to AML without treatment.

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Overview of MDS treatments

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4 Overview of MDS
treatments
32 Supportive care
36 Chemotherapy
38 Immunotherapy
39 Hematopoietic cell transplant
40 Donor lymphocyte infusion
42 Clinical trials
44 Review

Supportive care
Part 4 describes the main types of
Supportive care is treatment given to relieve the
treatment for MDS. Knowing what a symptoms caused by cancer and side effects of
treatment is will help you understand cancer treatment. It doesnt treat the cancer itself.
The goal of supportive care is to improve quality of life
your treatment options listed in the
and relieve any discomfort you may have.
Treatment guide in Part 5. There is
more than one treatment for MDS. Supportive care is an important part of the overall
treatment for MDS. It can address many needs. One
Not every person will receive every example is treatment for physical and emotional
treatment listed in this chapter. symptoms. It can also help with treatment decisions
and coordination between health care providers.

Low blood cell counts are very common in patients


Learn as much as you can about MDS with MDS. This includes a low number of red
treatment options, and keep learning. blood cells (anemia), a low number of platelets
This puts you in the drivers seat and (thrombocytopenia), and a low number of white
blood cells (neutropenia). Low blood cell counts
makes you a stronger advocate for your can cause many symptoms and affect quality of life.
best care and treatment. Thus, supportive care often aims to treat low blood
cell counts to lessen the related symptoms. The main
- Patient Advocate
types of supportive care that are used for MDS are
AAMDS International Foundation described next.

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Blood transfusions antibiotics may be needed. Antibiotics are drugs that


A blood transfusion is a slow injection of whole are used to treat infections caused by bacteria.
blood or parts of blood into a vein. A red blood cell
transfusion may be used to treat anemia that is Drugs for bleeding
causing symptoms. Symptoms of anemia include Thrombocytopenia is a condition in which the number
extreme tiredness (fatigue) and shortness of breath. of healthy platelets is too low. This can cause easy
A red blood cell transfusion can help to relieve these bruising or bleeding. In some cases, bleeding
symptoms for a short time. But, more transfusions problems may be severe or may not improve with
may be needed over time. Most patients with MDS platelet transfusions. If this happens, treatment with
will need one or more red blood cell transfusions at drugs may be needed.
some point.
Aminocaproic acid is a drug that may be used to
Thrombocytopenia can cause symptoms such as control bleeding problems in some patients. It is in a
easy bruising or bleeding. A platelet transfusion may class of drugs called antifibrinolytic agents. This type
be used to treat bleeding problems. of drug works by stopping blood clots from breaking
down too quickly.
Iron chelation therapy
Iron is a mineral found in red blood cells. Receiving a Blood cell growth factors
large number of red blood cell transfusions can cause Blood cell growth factors are substances that cause
too much iron to build up in the body. This is called new blood cells to grow in the bone marrow. Growth
iron overload. Excess iron can collect in and damage factors are made naturally in the body. But, they can
organs such as the heart, liver, and pancreas. also be made in a lab to use as treatment for low
blood cell counts. Growth factors may be used to treat
Iron chelation therapy is used to treat iron overload. anemia or neutropenia that is causing symptoms.
It is the use of drugscalled iron chelatorsthat The growth factors that may be used for patients with
bind to excess iron to remove it from the body. MDS are described next.
Deferoxamine and deferasirox are iron chelators that
may be used for patients with MDS. White blood cell growth factors
White blood cell growth factors may be used as
Deferoxamine is a liquid that is slowly injected treatment for patients with frequent infections due to
under the skin over several hours. This is called a neutropenia. There are two main types of white blood
subcutaneous infusion. Deferasirox may be given as cell growth factors. One is called G-CSF (granulocyte
a tablet that is dissolved in water. Or, it may be given colony-stimulating factor). The other is called GM-
as a pill that is swallowed. With either drug, treatment CSF (granulocyte-macrophage colony-stimulating
is given once a day until iron levels have decreased factor).
to a safe level.
G-CSF helps the body to make a type of white blood
Antibiotics cell called neutrophils. GM-CSF helps the body to
A low number of white blood cells can increase the make many types of white blood cells. These drugs
risk of infections. In some cases, infections may be are liquids that are injected under the skin.
frequent or severe. If this happens, treatment with

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Red blood cell growth factors


Red blood cell growth factors may be used as Caring for yourself:
treatment for patients with anemia that is causing Advice from a survivor
symptoms. These drugs are also called ESAs
(erythropoiesis-stimulating agents) because they MDS affects your bone marrow and blood, but it is
stimulate the bone marrow to make new red blood not all of who you are. It is only a part of the whole
cells.
person that is you. That is why it is so important
not to lose sight of caring for and advocating for
ESAs can increase red blood cell counts in some
patients. Such patients may then need fewer red yourself. If you have been waiting for a chance to
blood cell transfusions. This not only helps with do this for yourself, now is the time.
symptoms like fatigue and shortness of breath, but
also helps to improve overall quality of life. There are many things you can do for yourself to
improve your sense of wellness and of well-being:
Epoetin alfa and darbepoetin alfa are ESAs that may
be used for patients with MDS. Both drugs are liquids Learn all you can about MDS. Attend a nearby
that are injected under the skin. These drugs work conference.
best in patients with lower levels of serum EPO. In Find a community of MDS people.
some cases, G-CSF may be given along with an ESA Join a support group at your hospital
to improve how well it works. or cancer center.
Join an online community through
Platelet growth factors AAMDS International Foundation or
TPO (thrombopoietin) is a substance that helps the another trusted organization.
body to make platelets. TPO is made naturally in the
Cultivate a positive spirit.
body. But, drugs that act like TPO can also be made
Seek out and build upon support from
in a lab. Romiplostim and eltrombopag are drugs that
family and friends.
act like TPO. Some studies have shown that these
Take part in activities that are
drugs might help treat low platelet counts in certain
meaningful and satisfying to you.
patients with MDS. They may help to increase the
Eat healthy meals. Get enough rest.
number of platelets and decrease the risk of bleeding.
Sleep.
Try to get regular exercise as it can improve
energy and well-being.
Take a walk around a beautiful park, a
riverbank, or your local mall.
Take a community class.
Try tai chi, yoga, or chi-gung.

Ruth Horsfall, MDS survivor, patient advocate,


and member of the NCCN Panel for MDS

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Stress and symptom control

Cancer and its treatments can cause Feelings of anxiety and depression are
bothersome symptoms. The stress of having common among people with cancer. At your
cancer can also cause symptoms. There are cancer center, health care providers such as
ways to treat many symptoms, so tell your doctors, nurses, cancer navigators, social
treatment team about any that you have. workers, and other experts can help. Help
can include support groups, talk therapy,
or medication. Some people also feel better
You may lose sleep before, during, and after by exercising, talking with loved ones, or
treatment. Getting less sleep can affect your relaxing.
mood, conversations, and ability to do daily
tasks. If possible, allow yourself to rest, let
people do things for you, and talk with your You may be unemployed or miss work
doctor about sleep medication. Behavioral during treatment. Or, you may have too
sleep medicinea type of talk therapymay little or no health insurance. Talk to your
also help. treatment team about work, insurance,
or money problems. They will include
information in the treatment plan to help you
manage your finances and medical costs.

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Chemotherapy on genes that promote the growth of normal, healthy


Chemotherapy is the use of drugs to destroy cells in the bone marrow.
abnormal cells in the body. But, the drugs can
also affect normal cells. Many people refer to this Azacitidine is a liquid that is injected into a vein or
treatment as chemo. under the skin. A treatment cycle often consists of 7
days of treatment followed by 21 days of rest. At least
Different types of chemotherapy drugs work in 4 to 6 cycles should be given. If treatment works well,
different ways to kill abnormal cells or stop new ones cycles may repeat until it stops working.
from being made. Thus, more than one drug may be
used. When only one drug is used, its called a single Decitabine is a liquid that is slowly injected into a
agent. A combination regimen is the use of two or vein. This is called an IV (intravenous) infusion. The
more chemotherapy drugs. IV infusion takes 1 to 3 hours and must be given in
a hospital or clinic. The 1-hour infusion is given once
Chemotherapy is given in cycles of treatment days a day for 5 days in a row. This is repeated every 6
followed by days of rest. This allows the body to weeks. The 3-hour infusion is given every 8 hours
recover before the next cycle. Cycles vary in length for 3 days in a row. This is repeated every 4 weeks.
depending on which drugs are used. The number With either treatment schedule, at least 4 to 6 cycles
of treatment days per cycle and the total number of should be given. If treatment works well, cycles may
cycles given also varies. repeat until it stops working.

Some chemotherapy drugs are liquids that are High-intensity chemotherapy


injected into a vein or under the skin with a needle. High-intensity chemotherapy is treatment with high
Other chemotherapy drugs may be given as a pill doses of strong drugs that are more likely to cause
that is swallowed. The main chemotherapy drugs severe side effects. High-intensity chemo includes
used for MDS are described next. Chart 4.1 lists the drugs and regimens that are used to treat AML.
chemotherapy drugs and other drugs that are used to Because of the risk of very severe side effects, you
treat MDS. may need to stay overnight in the hospital during
treatment.
Low-intensity chemotherapy
Low-intensity chemotherapy is treatment with drugs
that are less likely to cause severe side effects. These
drugs are often given in an outpatient settingthis
means that you dont have to spend the night in the
hospital. There are two low-intensity chemo drugs
approved to treat MDS:

Azacitidine (Vidaza), and


Decitabine (Dacogen).

These drugs are a type of chemotherapy called


hypomethylating agents. They work by blocking DNA
that helps abnormal cells grow. This helps to turn

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Chart 4.1 Drug treatments for MDS

Generic name Brand name (sold as) Type of drug

Azacitidine Vidaza Chemotherapy (low-intensity)

Decitabine Dacogen Chemotherapy (low-intensity)

Cytarabine Cytosar-U Chemotherapy (high-intensity)

ATG (anti-thymocyte globulin) Thymoglobulin Immunosuppressive therapy

Cyclosporine Neoral Immunosuppressive therapy

Lenalidomide Revlimid Immunomodulator

Thalidomide Thalomid Immunomodulator

There are a number of high-intensity drugs for AML few. Some side effects can be very serious while
that could also be used for certain patients with MDS. others can be unpleasant but not serious. Most side
These drugs work in different ways to kill unhealthy effects appear soon after treatment starts and will go
cells in your blood and bone marrow. They tend to away after treatment ends. But, other side effects are
work well against fast-growing cells. But, they are long-term and may appear years later.
very toxic and kill normal cells along with cancer cells.
Thus, these drugs are often only used for MDS that is The side effects of chemotherapy depend on many
more likely to progress to AML. The goal is to kill all of factors. This includes the drug, the dose, and the
the abnormal cells in your body to stop the MDS from person. In general, side effects are caused by the
turning into AML. death of fast-growing cells, which are found in the
intestines, mouth, and blood. Thus, common side
Most of the high-intensity drugs are given as IV effects of chemotherapy are nausea, vomiting,
infusions. If you will have high-intensity chemo, ask diarrhea, not feeling hungry, hair loss, and low blood
your doctor how many cycles are needed and how cell counts.
many days of treatment are in each cycle.
Not all side effects of chemotherapy are listed here.
Side effects of chemotherapy Be sure to ask your treatment team for a complete
A side effect is an unhealthy or unpleasant physical list of common and rare side effects. If a side effect
or emotional condition caused by treatment. Each bothers you, tell your treatment team. There may be
treatment for MDS can cause side effects. The ways to help you feel better. There are also ways to
reactions to chemotherapy differ between people. prevent some side effects.
Some people have many side effects. Others have

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Myelodysplastic Syndromes, Version 1.2016
4 Overview of MDS treatments Immunotherapy

Immunotherapy sometimes happen. But, this can be managed with


anti-allergic drugs such as prednisone. Possible side
The immune system is the bodys natural defense effects of cyclosporine are kidney damage, not feeling
against infection and disease. Immunotherapy is hungry, high blood pressure, and gum swelling.
treatment with drugs that use the immune system
to help the body fight cancer. Different types of Immunomodulators
immunotherapy drugs affect the immune system in Immunomodulators are drugs that modify different
different ways. The two types of immunotherapy that parts of the immune system. Lenalidomide is an
may be used to treat MDS are described next. immunomodulator that is used to treat MDS. It is
approved to treat MDS with cells that are missing
Immunosuppressive therapy part of chromosome 5. This chromosome change is
IST (immunosuppressive therapy) is the use of drugs referred to as del(5q).
that lower (suppress) parts of the immune system. In
some types of MDS, the immune system may attack Lenalidomide treats cancer in more than one way. As
the bone marrow by mistake. This can cause the an immunomodulator, it boosts the immune system.
bone marrow to not work well and not make enough In this way, it may help the bone marrow to make
blood cells. IST drugs help to stop the immune normal blood cells and help kill abnormal cells in the
system from attacking the bone marrow. bone marrow. It also helps stop cancer cells from
increasing in number. Treatment with this drug may
ATG (antithymocyte globulin) and cyclosporine are lessen the need for red blood cell transfusions in
the main IST drugs that are used to treat MDS. This some patients. It works best against MDS with the
type of treatment doesnt work well for all cases of del(5q) chromosome change.
MDS. It tends to work better when MDS has certain
features that are linked with an immune system Lenalidomide is made as a pill that is swallowed. It
attack. Such features include: is given in cycles of treatment days followed by days
of rest. A cycle may consist of 21 days (3 weeks) of
Presence of HLA-DR15 protein on the MDS treatment and 7 days (1 week) of rest. It may also be
cells, given every day for 28 days (4 weeks). Cycles may
A low number of cells in the bone marrow, and repeat until the cancer progresses or side effects
Younger age (<60 years) in patients with become severe.
lower-risk MDS.
Common side effects include low blood cell counts,
ATG is given as an IV infusion over a few hours diarrhea, itching, rash, and fatigue. Ask your
in the hospital. It is often given for 4 or 5 days in a treatment team for a full list of side effects.
row. Cyclosporine comes as a pill or a liquid that
is swallowed. It is given once or twice a day and is
continued for at least 6 months.

Common side effects of ATG include fever, chills,


and rash at the time of the infusion. Other common
side effects are fever, swelling, tiredness, and hives.
Though much less common, severe allergic reactions

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Myelodysplastic Syndromes, Version 1.2016
4 Overview of MDS treatments Hematopoietic cell transplant

Hematopoietic cell transplant Conditioning treatment


Before the transplant, you will receive high-dose
An HCT (hematopoietic cell transplant) is a treatment chemotherapy. This is called conditioning treatment
that destroys cells in the bone marrow then replaces since it prepares (conditions) your body to receive
them with new, healthy blood-forming cells. These the donated blood stem cells. The chemotherapy
blood-forming cells are called blood stem cells or destroys normal cells and cancer cells in your bone
hematopoietic stem cells. Thus, this treatment is also marrow. It also greatly weakens your immune system
called a stem cell transplant. so that your body doesnt kill the transplanted blood
stem cells.
The goal of an HCT is to cure cancer by replacing
unhealthy blood stem cells with healthy ones that will There are two main types of conditioning treatment
attack cancer cells. This is done by suppressing the that can be used before the HCT. High-dose
bone marrow and cancer with chemotherapy then conditioning consists of high doses of strong (high-
transplanting healthy blood stem cells. The healthy intensity) chemotherapy drugs. Reduced-intensity
blood stem cells will grow, form new bone marrow conditioning consists of lower doses of strong
and blood cells, and attack remaining cancer cells. chemotherapy drugs or low-intensity drugs. Radiation
therapy may also be given as part of conditioning
For the treatment of MDS, blood stem cells from a treatment.
donor are used for the transplant. This is called an
allogeneic HCT. Before the transplant, special testing High-dose conditioning can cause very bad side
must be done to make sure the donor is a good effects and not all patients can tolerate it. Your doctor
match for you. HLA typing is used to find a persons will look at a number of factors to decide if you are
tissue type, called an HLA type. (See page 20 for healthy enough for this treatment. Such factors
more details on HLA typing.) include your age, your health status, the MDS risk
group, and the number of blast cells in your bone
An allogeneic HCT creates a new immune system marrow.
for your body. Another benefit of this transplant is the
GVL (graft-versus-leukemia) effect. The GVL effect Side effects are often worse in patients who are older
is an attack on the cancer cells by the transplanted or have other serious health problems. Thus, high-
blood stem cells. The steps of treatment with an dose conditioning is often only used for younger,
allogeneic HCT are described next. healthier patients. Reduced-intensity conditioning is
often used for patients who are older or less healthy
overall.

Transplanting the stem cells


After the conditioning treatment, the blood stem
cells will be put into your body with a transfusion. A
transfusion is a slow injection of blood products into
a large vein. This process can take several hours to
complete.

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Myelodysplastic Syndromes, Version 1.2016
4 Overview of MDS treatments Donor lymphocyte infusion

The transplanted blood stem cells then travel to your Donor lymphocyte infusion
bone marrow and grow. They will make new, healthy
blood cells. This is called engraftment. It usually takes DLI (donor lymphocyte infusion) is a treatment in
about 2 to 4 weeks. which the patient receives lymphocytes from the
same person who donated blood stem cells for the
Until then you will have little or no immune defense. HCT. A lymphocyte is a type of white blood cell that
This puts you at high risk for infection and bleeding. helps the body fight infections. The purpose of a DLI
You will likely need to stay in a hospital in a very is to stimulate an immune response called the GVL
clean room for some time. It may take a few weeks effect. This treatment may be used for MDS that
or months for blood cells to fully recover so that your came back or progressed after an HCT.
immune system is back to normal.
Possible side effects of a DLI are similar to those
Side effects of allogeneic HCT caused by an allogeneic HCT. Because this treatment
A side effect is an unhealthy or unpleasant physical starts an immune response in your body, you are
or emotional condition caused by treatment. Common at risk for GVHD. Some other side effects of a DLI
side effects of chemotherapy, which is given before are weakened (suppressed) bone marrow and an
the transplant, are described on page 37. You will increased risk of infection.
likely feel tired and weak shortly after the transplant
while waiting for the new blood stem cells to grow in
the bone marrow.

Allogeneic HCTs have a high risk of GVHD (graft-


versus-host disease). GVHD is when the donated
cells see the cells in your body as foreign and attack
them. The parts of the body most commonly damaged
by GVHD are the skin, intestines, and liver.

GVHD is a serious side effect that can cause the


transplant to fail by stopping the donated blood stem
cells from growing in your bone marrow. GVHD can
happen within a few weeks after the transplant or
much later. Your doctor may give you medicine that
suppresses your immune system to try to prevent this
side effect.

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Myelodysplastic Syndromes, Version 1.2016
4 Overview of MDS treatments Complementary and alternative medicine

Complementary and alternative medicine

CAM (complementary and alternative medicine) is a group of


treatments that arent often given by doctors. There is much interest
today in CAM for cancer. Many CAMs are being studied to see if they
are truly helpful.

Complementary medicines are treatments given along with usual


medical treatments. While CAMs arent known to kill cancer cells,
they may improve your comfort and well-being. Two examples are
acupuncture for pain management and yoga for relaxation.

Alternative medicine is used in place of usual medicine. Some


alternative medicines are sold as cures even though they havent
been proven to work. If there was good proof that CAMs or other
treatments cured cancer, they would be included in this book.

It is important to tell your treatment team if you are using any CAMs.
They can tell you which CAMs may be helpful and which CAMs may
limit how well treatments work.

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Myelodysplastic Syndromes, Version 1.2016
4 Overview of MDS treatments Clinical trials

Clinical trials
New tests and treatments arent offered to the public
as soon as theyre made. They need to be studied
first. New uses of tests and treatments also need to
be studied. A clinical trial is a type of research that
studies a test or treatment.

Clinical trials study how safe and helpful tests and


treatments are. When found to be safe and helpful, they
may become tomorrows standard of care. Because of
clinical trials, the tests and treatments in this book are
now widely used to help people with MDS.

Tests and treatments go through a series of clinical Joining a clinical trial has benefits. First, youll have
trials to make sure theyre safe and work. Without access to the most current cancer care. Second, you
clinical trials, theres no way to know if a test or will receive the best management of care. Third, the
treatment is safe or helpful. Clinical trials are done in results of your treatmentboth good and badwill be
a series of steps, called phases. The four phases of carefully tracked. Fourth, you may help other patients
clinical trials are described next using the example of with cancer.
a new drug treatment:
Clinical trials have risks, too. Like any test or
Phase I trials aim to find the best dose and way to treatment, there may be side effects. Also, new tests
give a new drug with the fewest side effects. If a drug or treatments may not help. Another downside may
is found to be safe, it will be studied in a phase II trial. be that paperwork or more trips to the hospital may
be needed.
Phase II trials assess if a drug works for a specific
type of cancer. They are done in larger groups of To join a clinical trial, you must meet the conditions
patients with the same type of cancer. of the study. Patients in a clinical trial often have
a similar cancer type and general health. This is
Phase III trials compare a new drug to the standard to know that any improvement is because of the
treatment or to a placeboa substance with no treatment and not because of differences between
effects. These are randomized, meaning patients are patients. You also must review and sign a paper
put in a treatment group by chance. Phase III trials called an informed consent form to join a clinical trial.
are done to learn about short- and long-term side This form describes the study in detail, including the
effects and about safety. risks and benefits.

Phase IV trials test new drugs approved by the FDA Ask your treatment team if there is an open clinical
(U.S. Food and Drug Administration) to learn more trial that you can join. There may be clinical trials
about short-term side effects, long-term side effects, where youre getting treatment or at other treatment
and safety. They involve many patients with different centers nearby.
types of cancer.

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Myelodysplastic Syndromes, Version 1.2016
4 Overview of MDS treatments Notes

My notes

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Myelodysplastic Syndromes, Version 1.2016
4 Overview of MDS treatments Review

Review
Supportive care is treatment for the symptoms An HCT replaces damaged or diseased cells
or health conditions caused by cancer or in the bone marrow with healthy blood stem
cancer treatment. Supportive care is an cells.
important part of treatment for MDS.
Clinical trials give people access to new tests
Immunotherapy is treatment with drugs that and treatments that arent usually received.
modify the immune system to help the body
fight cancer.

Chemotherapy is the use of drugs to kill


abnormal cells in the body.

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Myelodysplastic Syndromes, Version 1.2016
5
Treatment guide

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45
Myelodysplastic Syndromes, Version 1.2016
5 Treatment guide
48 5.1 Lower-risk MDS with
anemia
Treatments for patients who have
lower-risk MDS and a low number
of red blood cells (anemia) that is
causing symptoms.

52 5.2 Lower-risk MDS without


anemia
Treatments for patients who have
lower-risk MDS and a low number
of white blood cells (neutropenia),
a low number of platelets
(thrombocytopenia), or an increased
number of blast cells in the bone
marrow.

54 5.3 Higher-risk MDS


Treatments for patients who have
higher-risk MDS.

58 5.4 Treatment for anemia


Treatments for anemia that is
causing symptoms.

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Myelodysplastic Syndromes, Version 1.2016
5 Treatment guide Myelodysplastic syndromes (MDS)

Treatment options
Part 5 is a guide through the There are multiple treatment options for MDS.
Treatment options that are best for you depend on a
treatment options for people with number of factors. This includes the features of the
MDS. This information is taken from cancer, such as the MDS subtype and risk score, as
well as your age and health status.
the treatment guidelines written
by NCCN experts of MDS. These The treatment options in Part 5 are organized by
treatment guidelines list options for MDS risk groups and symptoms. The timing, intensity,
and goal of treatment differs depending on the risk
people with MDS in general. Thus, group. To plan treatment, doctors put risk groups into
your doctors may suggest other two categories: lower-risk MDS and higher-risk MDS.
treatment for you based on your
Lower-risk MDS includes the following risk
health and personal wishes. Fully groups: IPSS Low and Intermediate-1; IPSS-R Very
discuss your treatment options with Low, Low, and Intermediate; and WPSS Very Low,
Low, and Intermediate.
your doctor.
Higher-risk MDS includes the following risk
groups: IPSS Intermediate-2 and High; IPSS-R
Much effort has been made to make this guide easy Intermediate, High, and Very High; and WPSS High
to read. Charts list the treatment options and map the and Very High.
steps through the treatment process. The text along
with each chart explains the information shown in the Lower-risk MDS is more likely to grow slowly and
chart. may not progress to AML for a long time. Thus,
low-intensity treatments are used first. The goal of
treatment for lower-risk MDS is to improve blood cell
counts, lessen the need for blood transfusions, and
improve quality of life.

Higher-risk MDS is more likely to grow faster and


progress to AML in a shorter amount of time. Thus,
high-intensity treatments are often tried first. The
goal of treatment for higher-risk MDS is to slow or
stop MDS progression to AML and help patients live
longer.

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5 Treatment
DCIS guide Lower-risk
Genetic counseling
MDS| with
Treatment
anemia

5.1 Lower-risk MDS with anemia

Chart 5.1.1 Initial treatment for lower-risk MDS with anemia

Test results Treatment options

del(5q) other chromosome changes Lenalidomide

Epoetin alfa G-CSF, or


No del(5q) other chromosome changes, and
Serum EPO 500 mU/mL
Darbepoetin alfa G-CSF

If likely to respond to IST:


ATG + cyclosporine
If not likely to respond to IST:
No del(5q) other chromosome changes, and
Azacitidine or decitabine,
Serum EPO >500 mU/mL
Consider lenalidomide, or

Clinical trial

Chart 5.1.1 shows the treatment options for patients effects. These tests are also used to check how well
with lower-risk MDS and anemia that is causing treatment is working. An outcome or improvement
symptoms. The options differ based on the types of caused by treatment is called a treatment response.
chromosome changes in the MDS cells and the level
of EPO in your blood. One key chromosome change The goal is to increase red blood cell counts and
is when MDS cells are missing part of chromosome 5. lessen the need for red blood cell transfusions.
This change is called del(5q). The amount of natural Doctors often assess the response about 2 to 4
EPO in your blood is called the serum EPO level. months after the start of treatment with lenalidomide.
If tests do not show a treatment response, then your
next options are the same as those listed for MDS
Treatment options for MDS with del(5q) without del(5q).

If MDS cells have the del(5q) chromosome If tests show a response, you should continue this
change, treatment with lenalidomide is treatment until it stops working or side effects get too
recommended. But, this drug should not be given severe. Over time, your doctor may lower the dose of
if you have a very low number of neutrophils or lenalidomide to lessen the side effects. (See page 38
platelets. Your doctors will give tests during treatment for more details about lenalidomide.)
to check your blood cell counts and watch for side

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Myelodysplastic Syndromes, Version 1.2016
5 Treatment guide Lower-risk MDS with anemia

Treatment options for MDS without The second option is to receive lenalidomide. This
del(5q) drug works best against MDS with the del(5q)
chromosome change (described above). But, it may
If tests show serum EPO 500 mU/mL, treatment also be helpful for some other patients. The third
with blood cell growth factors is recommended. The option is to receive treatment within a clinical trial.
two main options are epoetin alfa and darbepoetin (See Part 4 for more details about each treatment.)
alfa. These drugs are red blood cell growth factors
also called ESAs. ESAs act like EPO and stimulate Your doctor will give tests during treatment to check
the body to make more red blood cells. Thus, they if it is working well to improve blood cell counts
work best when there is a lower level of natural EPO and other signs of MDS. How long it takes to see a
in the blood. treatment response depends on which treatment you
have. If tests show a response, treatment may be
ESAs may be given alone or along with G-CSF. continued until it stops working. But, treatment may
G-CSF is a white blood cell growth factor. Studies need to be stopped early if side effects become too
show that adding G-CSF can improve how well ESAs severe. This is called treatment intolerance.
work for some patients. See Next steps at the end of
this section.

If tests show serum EPO >500 mU/mL, there are


four main treatment options to choose from. The
options are divided into two groups. The first group
Next steps:
includes IST drugs and the second one does not.
If tests do not show a treatment
To decide between options, your doctor will check
response, see Chart 5.1.2 on page 50
for factors linked with a higher chance that MDS will
respond to IST. Such factors include: hypocellular for the next treatment options. Chart
bone marrow, 5 percent blast cells in bone marrow, 5.1.2 also includes the next options
and MDS cells with the HLA-DR15 protein. IST also
tends to work better in patients who are 60 years of
for if initial treatment stops working or
age or younger. side effects become too severe. For
patients with serum EPO <500 mU/
If MDS is likely to respond to IST, then you will
receive ATG with cyclosporine. If MDS is not likely to mL, see Chart 5.4.1 on page 58 and
respond to IST, then you have a few other options to 5.4.2 on page 60 for more details
choose from. The first option is to receive azacitidine
or decitabine. Both are low-intensity chemotherapy
about anemia treatment with blood
drugs. They are also a lot alike in how they work cell growth factors.
against MDS. They can help to increase blood cell
counts and lessen the need for transfusions. They
can also help to slow MDS growth and progression
to AML. With either drug, at least 4 to 6 cycles of
treatment should be given to decide if it is working.

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5 Treatment guide Lower-risk MDS with anemia

Chart 5.1.2 Next treatment options for lower-risk MDS with anemia

Prior treatment Next treatment options

Epoetin alfa G-CSF, or


Consider ATG + cyclosporine
Darbepoetin alfa G-CSF

Azacitidine or decitabine,

ATG + cyclosporine Lenalidomide, or

Clinical trial

Azacitidine,
Clinical trial, or
Decitabine,
Lenalidomide, or
Consider allogeneic HCT for certain patients
Clinical trial

Chart 5.1.2 shows the next options for if prior After treatment with epoetin alfa or darbepoetin
treatment didnt work, stopped working, or had very alfa, your doctor may consider IST next. Your doctor
bad side effects. There are several options to choose will look at the features of the MDS cells and other
from. Which option is best for you depends on the factors to decide if this treatment might work well
treatment you had before. for you. (See page 38 for details.) If there is a good
chance that MDS will respond to IST, then you will
Your doctor will give tests during treatment to check receive ATG with or without cyclosporine. If MDS is not
how well its working. If tests show a treatment likely to respond to IST, then the next options are the
response, you will stay on that treatment as long same as those listed for after treatment with ATG or
as it is working well. It will be stopped once it is no cyclosporine.
longer working well. Or, it may be stopped early if side
effects get very bad. After treatment with ATG or cyclosporine, there
are three main options to choose from next. The
If one treatment doesnt work or has to be stopped, a first option is to receive low-intensity chemotherapy
different option will be tried. Each time this happens, with azacitidine or decitabine. The second option is
refer to the chart for the next options. These options to receive lenalidomide, unless you have a very low
are also described next. number of platelets or neutrophils. The third option is
to join a clinical trial.

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Myelodysplastic Syndromes, Version 1.2016
5 Treatment guide Lower-risk MDS with anemia

After treatment with azacitidine, decitabine,


lenalidomide, or a clinical trial, there are two main
options to choose from next. The first option is to
Next steps:
receive treatment within a clinical trial. An allogeneic
HCT may also be an option for some patients. But, it If tests show progression to higher-
is only recommended for patients with intermediate-
risk MDS, see Chart 5.3.1 on page
risk MDS and very low blood cell counts. See Next
steps at the end of this section. 54 for treatment options. Chart 5.3.1
also includes options for treatment
with an allogeneic HCT. For detailed
treatment of anemia, see Chart 5.4.1
on page 58.

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3
5 Treatment
DCIS guide Lower-risk
Genetic counseling
MDS| without
Treatment
anemia

5.2 Lower-risk MDS without anemia

Chart 5.2 Treatment for lower-risk MDS without anemia

Treatment options Treatment results Next options

Azacitidine or decitabine, Response Stay on current treatment

IST for certain patients, or


Clinical trial, or
No response, or
Clinical trial Progression
Consider allogeneic HCT for certain patients

Chart 5.2 shows the treatment options for patients If there is a good chance that MDS will respond
with lower-risk MDS but without anemia. Instead, the to IST, then you will receive ATG with or without
number of platelets or white blood cells is low. Or, the cyclosporine. The third option is to receive treatment
number of blast cells in the bone marrow is high. within a clinical trial.

Treatment options Treatment results and next options

There are three main treatment options to choose Your doctor will give tests during treatment to check
from. The first option is to receive azacitidine or if it is working well to improve blood cell counts
decitabine. Both are low-intensity chemotherapy and other signs of MDS. This is called a treatment
drugs. They are also a lot alike in how they work response. How long it takes to see a treatment
against MDS. They can help to increase blood cell response depends on which treatment you have.
counts and lessen the need for transfusions. They Sometimes MDS grows or gets worse during
can also help to slow MDS growth and progression treatment. This is called progression. The next
to AML. With either drug, at least 4 to 6 cycles of treatment options depend on how well initial treatment
treatment should be given to decide if it is working. worked.

Treatment with IST may also be an option for some If tests show a response, then you will stay on the
patients. Your doctor will look at the features of the same treatment as long as it keeps working. Once the
MDS cells and other factors to decide if this treatment MDS grows or gets worse, the next treatment options
might work well for you. (See page 38 for details.) will be tried.

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Myelodysplastic Syndromes, Version 1.2016
5 Treatment guide Lower-risk MDS without anemia

If tests dont show a response, or show


progression, there are two options to choose from
next. The first option is to receive treatment within
Next steps:
a clinical trial. An allogeneic HCT may also be an
option for some patients. But, it is only recommended If tests show progression to higher-
for patients with intermediate-risk MDS and very low
risk MDS, see Chart 5.3.1 on page 54
blood cell counts. See Next steps at the end of this
section. for treatment options. Chart 5.3.1 also
includes options for treatment with an
allogeneic HCT.

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3
5 Treatment
DCIS guide Higher-risk
Genetic counseling
MDS| Treatment

5.3 Higher-risk MDS

Chart 5.3.1 Initial treatment for higher-risk MDS

Assessment before treatment Treatment options

Allogeneic HCT,
Allogeneic HCT is a good option for you, and
Azacitidine or decitabine followed by allogeneic HCT, or
A well-matched donor is available
High-intensity chemo followed by allogeneic HCT

Azacitidine (preferred),
Allogeneic HCT may be a good option for you,
Decitabine, or
but A well-matched donor is not available
Clinical trial

Azacitidine (preferred),
Allogeneic HCT is not a good option for you, or
Decitabine, or
A well-matched donor is not available
Clinical trial

Chart 5.3.1 shows the treatment options for Your doctor will look at many factors to decide if high-
higher-risk MDS. To help decide which option is intensity treatment is a good option for you. Such
best for you, your doctor will first assess if you are factors include your personal preference, your age,
able to have high-intensity treatment. High-intensity how well you can do daily activities, and other health
treatment includes allogeneic HCT and high-intensity conditions. Another key factor is if there is a well-
chemotherapy. matched donor for the allogeneic HCT.

High-intensity treatments aim to slow or stop MDS If an allogeneic HCT is a good option for you
progression to AML and improve survival. But, they and a donor has been found, there are three main
can also cause very bad side effects. Side effects are options to choose from. The first option is to receive
often more severe for patients who are older or have an allogeneic HCT right away. High-dose conditioning
other health problems. Not all patients can tolerate or reduced-intensity conditioning may be used
the severe side effects. for the HCT. (See page 39 for more details about
conditioning treatment.)

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Myelodysplastic Syndromes, Version 1.2016
5 Treatment guide Higher-risk MDS

The second option is to receive azacitidine or The second option is to receive decitabine, which is
decitabine first and then an allogeneic HCT. This may also a low-intensity chemotherapy drug. It is a lot like
be used to lower the number of blast cells in your azacitidine in how it works against MDS. Decitabine
bone marrow before the HCT. It may also be a good can lower the chance that MDS will progress to
option if you are still waiting for a well-matched donor. AML in some patients. The third option is to receive
treatment within a clinical trial.
The third option is to receive high-intensity
chemotherapy first and then an allogeneic HCT. High-
intensity chemo as part of a clinical trial is preferred.
The high-intensity chemo can help to lower the
number of blast cells in your bone marrow before the
HCT. But, it can cause very severe side effects and
may not be a good choice for all patients. Next steps:
If an allogeneic HCT is not a good option for
you or a donor hasnt been found, there are three See Chart 5.3.2 on page 56 for the
options to choose from. The first option is to receive
next options after initial treatment for
azacitidine, a low-intensity chemotherapy drug.
This is the preferred option for patients with higher- higher-risk MDS.
risk MDS who are not able to have high-intensity
treatment. Azacitidine can lower the chance that MDS
will progress to AML in some patients. It can also help
some patients have improved blood cell counts and
live longer than they would without treatment. Your
doctor may refer to this as improved survival.

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Myelodysplastic Syndromes, Version 1.2016
5 Treatment guide Higher-risk MDS

Chart 5.3.2 Next treatment options for higher-risk MDS

Prior treatment Results Treatment options Next options

Consider second HCT or DLI,


Clinical trial, or
Relapse or
Allogeneic HCT Azacitidine or decitabine, or
no response
Supportive care
Clinical trial

Clinical trial, or
Continue treatment until
Response
it stops working
Supportive care
Azacitidine or
decitabine
Clinical trial, or
Relapse or
no response
Supportive care

Clinical trial, or
Relapse or
Clinical trial
no response
Supportive care

Chart 5.3.2 shows the next options used after initial If you had an allogeneic HCT, and tests show a
treatment for higher-risk MDS. There are several relapse or no response, there are three main options
options to choose from. Which option is best for you to choose from. The first option is to have a second
depends on which treatment you had before and how allogeneic HCT or a DLI. This may be a good option if
well it worked. the response to the first HCT lasted for a while before
tests showed a relapse.
Your doctor will give tests during treatment to check
how well its working. An outcome or improvement The second option is to receive azacitidine or
caused by treatment is called a treatment response. decitabine. Both are low-intensity chemotherapy
How long it takes to see a treatment response drugs. They are a lot alike in how they work against
depends on the type of treatment used. A relapse is MDS. With either drug, treatment should be continued
the return or worsening of MDS after a response or as long as it is working well. The third option is to join
period of improvement. a clinical trial.

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If any of these treatments dont work or stop working,


there are two more options to choose from. One
option is to receive a different treatment within a
clinical trial. Another option is to receive supportive
care only.

If you had azacitidine or decitabine, the next


options depend on how well the drug worked. With
either drug, at least 4 to 6 cycles should be given
before checking for a response. If tests show a
response, then treatment should be continued as long
as it keeps working.

If tests dont show a response, or show a relapse,


there are two more options to choose from. The first
option is to join a clinical trial. The second option is to
receive supportive care only.

If you had treatment within a clinical trial, and


the drug didnt work or stopped working, there are
two more options to choose from. The first option
is to receive a different drug or type of treatment
within a clinical trial. The second option is to receive
supportive care only.

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5 DCIS
Treatment guide Treatment
Genetic counseling
for anemia
| Treatment

5.4 Treatment for anemia

Chart 5.4.1 Initial care for anemia

Assessing anemia

Rule out and treat other possible causes of anemia

Replace iron, folate, or vitamin B12 if needed

Red blood cell transfusions

Supportive care

Chart 5.4.1 shows the initial steps to assess and time. You should also receive other supportive care
care for anemia. MDS is not the only health condition as needed. (For more details about supportive care,
that can cause anemia. A number of other factors can see page 32.)
cause or contribute to anemia. Thus, it is important
to check for any other health problems that might be
causing a low number of red blood cells. The tests
doctors use to assess anemia are described in Part 2
on page 16. Next steps:
Iron, folate, and vitamin B12 are nutrients in the body
that are needed to make red blood cells. A shortage See Chart 5.4.2 on page 60 for
of any one of these substances can cause anemia.
treatment options for MDS-related
Thus, your doctor will want to know if you have
the right amount of these nutrients. If any are low, anemia.
treatment with vitamin supplements may help.

Red blood cell transfusions are the standard of care


for patients with anemia that is causing symptoms.
A red blood cell transfusion is a slow injection of red
blood cells into a vein. This can help relieve anemia
symptoms quickly. But, this relief may only last for a
few months. More transfusions may be needed over

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My notes

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5 Treatment guide Treatment for anemia

Chart 5.4.2 Treatment for anemia that is causing symptoms

Tests results Treatment options Results Next options

del(5q) other Response Stay on lenalidomide


chromosome Lenalidomide
changes No response See Chart 5.1.1

Serum EPO 500 Epoetin alfa, or Response Continue treatment


mU/mL, and <15%
Ring sideroblasts Darbepoetin alfa No response Consider adding G-CSF

Serum EPO 500 Epoetin alfa + G-CSF, or Response Continue treatment


mU/mL, and 15%
Ring sideroblasts Darbepoetin alfa + G-CSF No response See Chart 5.1.1

Serum EPO
See options on Chart 5.1.1
>500 mU/mL

Chart 5.4.2 shows the treatment options for anemia to check your blood cell counts. These tests are
that is causing symptoms. The options differ based on also used to check how well treatment is working.
the features of the MDS cells and the level of EPO in An outcome or improvement caused by treatment is
your blood. The amount of natural EPO in your blood called a treatment response.
is called the serum EPO level.
The goal is to increase red blood cell counts and
One key feature is whether or not the MDS cells are lessen the need for red blood cell transfusions.
missing part of chromosome 5. This chromosome Doctors often assess the response about 2 to 4
change is called del(5q). Another key feature is the months after the start of treatment with lenalidomide.
amount of ring sideroblasts found in the bone marrow. If tests show a response, you should continue this
treatment until it stops working or side effects get too
If MDS cells have the del(5q) chromosome severe. Over time, your doctor may lower the dose of
change, treatment with lenalidomide is lenalidomide to lessen the side effects. (See page 38
recommended. But, this drug should not be given for more details about lenalidomide.)
if you have a very low number of neutrophils or
platelets. Your doctors will give tests during treatment

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5 Treatment guide Treatment for anemia

For patients with serum EPO 500 mU/ For patients with serum EPO >500 mU/mL,
mL, treatment with blood cell growth factors is treatment with red blood cell growth factors is not
recommended. The two main options are epoetin alfa recommended. EPO is made naturally in your body to
and darbepoetin alfa. Both drugs are red blood cell stimulate your bone marrow to make more red blood
growth factorsalso called ESAs. ESAs are drugs cells. Red blood cell growth factors are drugs that
that act like EPO, a substance that is made naturally act like EPO. These drugs dont work as well when
in your body to stimulate the growth of new red blood there is a higher level of natural EPO in the blood.
cells. Thus, these drugs work best when there is a Therefore, other types of treatments should be used.
lower level of natural EPO in the blood. See Next steps at the end of this section.

If less than 15 percent of cells in the bone marrow are


ring sideroblasts, then ESAs alone are suggested. If
15 percent or more of the bone marrow cells are ring
sideroblasts, then G-CSF should be given along with
an ESA. G-CSF is a white blood cell growth factor.
Next steps:
Studies show that adding G-CSF can improve how
well ESAs work for some patients. If tests dont show a treatment
response, or treatment stops working,
Epoetin alfa is given in high doses weekly.
Darbepoetin alfa is a long-acting drug and is given see Chart 5.1.1 on page 48 for more
once every 2 weeks. Low doses of G-CSF are given treatment options. Chart 5.1.1 also
weekly. (See page 33 for more details about blood
cell growth factors.)
includes treatment options for patients
with serum EPO >500 mU/mL.
The goal of treatment with blood cell growth factors
is to increase red blood cell counts and hemoglobin
level and decrease the number of red blood cell
transfusions needed. This often happens 6 to 8 weeks
after the start of treatment. If the treatment works, it
should be continued. If treatment with an ESA alone
isnt working well, then G-CSF may be added. If these
drugs do not improve your hemoglobin level within
several months, they should be stopped and other
treatment should be tried.

Your doctor will give tests during treatment to check


how well its working. If tests show a treatment
response, treatment should be continued until it stops
working. Over time, lower doses may be given based
on the treatment response.

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5 DCIS
Treatment guide Genetic
Notes
counseling | Treatment

My notes

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Making treatment decisions

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6 Making treatment
decisions
65 Its your choice
66 Questions to ask your doctors
70 Weighing your options
72 Websites and resources
72 Review

Having cancer can feel very stressful.


While absorbing the fact that you
have cancer, you must also learn
about tests and treatments. And,
the time you have to decide on a
treatment plan may feel short. Parts
1 through 5 described the test and
treatment options recommended by
NCCN experts. These options are
based on science and agreement
among NCCN experts. Part 6 aims
to help you make decisions that are
in line with your beliefs, wishes, and
values.

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6 Making treatment decisions It's your choice

Its your choice Every doctor visit is an opportunity to


The role patients want in choosing their treatment educate yourself. Take time to prepare
differs. You may feel uneasy about making treatment for every visit. Make a list of questions,
decisions. This may be due to a high level of stress. It and more importantly, make sure you
may be hard to hear or know what others are saying.
Stress, pain, and drugs can limit your ability to make
understand the answers. Nurses and
good decisions. You may feel uneasy because you physician assistants can also be great
dont know much about cancer. You may have never resources to answer your questions.
heard the words used to describe cancer, tests,
or treatments. Likewise, you may think that your - Patient Advocate
judgment isnt any better than your doctors. AAMDS International Foundation

Letting others decide which option is best may make


you feel more at ease. But, whom do you want to
make the decisions? You may rely on your doctors
alone to make the right decisions. However, your
doctors may not tell you which to choose if you have
multiple good options. You can also have loved
ones help. They can gather information, speak on
your behalf, and share in decision-making with your
doctors. Even if others decide which treatment you
will receive, you still have to agree by signing a
consent form.

On the other hand, you may want to take the lead


or share in decision-making. Most patients do. In
shared decision-making, you and your doctors share
information, weigh the options, and agree on a
treatment plan. Your doctors know the science behind
your plan but you know your concerns and goals. By
working together, you are likely to get a higher quality
of care and be more satisfied. Youll likely get the
treatment you want, at the place you want, and by the
doctors you want.

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6 Making treatment decisions Questions to ask your doctors

Questions to ask your doctors


You will likely meet with experts from different fields of medicine. Strive to have helpful talks with
each person. Prepare questions before your visit and ask questions if the person isnt clear. You can
also record your talks and get copies of your medical records. It may be helpful to have your spouse,
partner, or a friend with you at these visits. A patient advocate or navigator might also be able to come.
They can help to ask questions and remember what was said. Some suggested questions to ask your
doctors are listed on the next pages:

Whats my diagnosis and prognosis?


Its important to know that there are different types of cancer. Cancer can greatly differ even when
people have a tumor in the same organ. Based on your test results, your doctors can tell you which
type of cancer you have. He or she can also give a prognosis. A prognosis is a prediction of the pattern
and outcome of a disease. Knowing the prognosis may affect what you decide about treatment.

1. Where did the cancer start? In what type of cell?

2. Is this cancer common?

3. What is the cancer risk score and risk group? Does this risk score or group mean the cancer has
progressed a lot?

4. Is this a fast- or slow-growing type of MDS?

5. What are the chances that this type of MDS will become AML?

6. What other tests results are important to know?

7. How often are these tests wrong?

8. Would you give me a copy of the pathology report and other test results?

9. Can the cancer be cured? If not, how well can treatment stop the cancer from growing?

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What are my options?


There is no single treatment practice that is best for all patients. There is often more than one
treatment option along with clinical trial options. Your doctor will review your test results and
recommend treatment options.

1. What will happen if I do nothing?

2. Can I just carefully monitor the cancer?

3. Do you consult NCCN recommendations when considering options?

4. Are you suggesting options other than what NCCN recommends? If yes, why?

5. Do your suggested options include clinical trials? Please explain why.

6. How do my age, health, and other factors affect my options?

7. Which option is proven to work best?

8. Which options lack scientific proof?

9. What are the benefits of each option? Does any option offer a cure? Are my chances any better
for one option than another? Less time-consuming? Less expensive?

10. What are the risks of each option? What are possible complications? What are the rare and
common side effects? Short-lived and long-lasting side effects? Serious or mild side effects?
Other risks?

11. What can be done to prevent or relieve the side effects of treatment?

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What does each option require of me?


Many patients consider how each option will practically affect their lives. This information may be
important because you have family, jobs, and other duties to take care of. You also may be concerned
about getting the help you need. If you have more than one option, choosing the option that is the
least taxing may be important to you.

1. Will I have to go to the hospital or elsewhere? How often? How long is each visit?

2. Do I have a choice of when to begin treatment? Can I choose the days and times of treatment?

3. How do I prepare for treatment? Do I have to stop taking any of my medicines? Are there foods I
will have to avoid?

4. Should I bring someone with me when I get treated?

5. Will the treatment hurt?

6. How much will the treatment cost me? What does my insurance cover?

7. Will I miss work or school? Will I be able to drive?

8. Is home care after treatment needed? If yes, what type?

9. How soon will I be able to manage my own health?

10. When will I be able to return to my normal activities?

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What is your experience?


More and more research is finding that patients treated by more experienced doctors have better
results. It is important to learn if a doctor is an expert in the cancer treatment he or she is offering.

1. Are you board certified? If yes, in what area?

2. How many patients like me have you treated?

3. How many procedures like the one youre suggesting have you done?

4. Is this treatment a major part of your practice?

5. How many of your patients have had complications?

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Weighing your options your options. Choosing your cancer treatment is a


very important decision. It can affect your length and
Deciding which option is best can be hard. Doctors quality of life.
from different fields of medicine may have different
opinions on which option is best for you. This can be Support groups
very confusing. Your spouse or partner may disagree Besides talking to health experts, it may help to talk
with which option you want. This can be stressful. In to patients who have walked in your shoes. Support
some cases, one option hasnt been shown to work groups often consist of people at different stages of
better than another, so science isnt helpful. Some treatment. Some may be in the process of deciding
ways to decide on treatment are discussed next. while others may be finished with treatment. At
support groups, you can ask questions and hear
2nd Opinion about the experiences of other people with MDS.
The time around a cancer diagnosis can be very
stressful. People with cancer often want to start Compare benefits and downsides
treatment as soon as possible. They want to make Every option has benefits and downsides. Consider
the cancer go away before it spreads farther. While these when deciding which option is best for you.
cancer cant be ignored, there is time to think about Talking to others can help identify benefits and
and choose which option is best for you. downsides you havent thought of. Scoring each
factor from 0 to 10 can also help since some factors
You may wish to have another doctor review your test may be more important to you than others.
results and suggest a treatment plan. This is called
getting a 2nd opinion. You may completely trust your
doctor, but a 2nd opinion on which option is best can
help.

Copies of all of the test results need to be sent to


the doctor giving the 2nd opinion. Some people feel You have the right to a second opinion
uneasy asking for copies from their doctors. However, about your MDS diagnosis and treatment
a 2nd opinion is a normal part of cancer care. recommendations. So dont be afraid of
When doctors have cancer, most will talk with more
offending your doctor. Some health plans
than one doctor before choosing their treatment. even require a second opinion.
What's more, some health plans require a 2nd opinion.
- Patient Advocate
If your health plan doesnt cover the cost of a 2nd
opinion, you have the choice of paying for it yourself. AAMDS International Foundation

If the two opinions are the same, you may feel more
at peace about the treatment you accept to have.
If the two opinions differ, think about getting a 3rd
opinion. A 3rd opinion may help you decide between

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My notes

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6 Making treatment decisions Websites and resources | Review

Websites and resources Support groups and events

Aplastic Anemia and MDS International Aplastic Anemia and MDS International
Foundation Foundation
www.aamds.org/about/MDS www.aamds.org/support

American Cancer Society Leukemia and Lymphoma Society


www.cancer.org/cancer/ www.lls.org/support/support-groups
myelodysplasticsyndrome/index
The MDS Foundation
www.cancer.org/Treatment/ www.mds-foundation.org/patient-caregiver-
FindingandPayingforTreatment/index resources/

National Cancer Institute


www.cancer.gov/types/myeloproliferative

The Myelodysplastic Syndromes Foundation


www.mds-foundation.org/ Review
National Coalition for Cancer Survivorship Shared decision-making is a process in which
www.canceradvocacy.org/toolbox you and your doctors plan treatment together.

Asking your doctors questions is vital to


NCCN
getting the information you need to make
www.nccn.org/patients/resources/life_with_
informed decisions.
cancer/default.aspx
Getting a 2nd opinion, attending support
100 Questions & Answers about groups, and comparing benefits and
Myelodysplastic Syndromes downsides may help you decide which
by Jason Gotlib, MD, MS & Lenn Fetcher, treatment is best for you.
RN
www.mds-foundation.org/100-questions-
answers-about-myelodysplastic-syndromes/

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Glossary

Dictionary
Acronyms

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Glossary Dictionary

Dictionary
acute myeloid leukemia (AML) bone marrow aspiration
A fast-growing cancer that starts in the bone marrow and The removal of a small amount of liquid bone marrow to test
causes too many immature white blood cells to be made. for disease.

allogeneic hematopoietic cell transplant (HCT) bone marrow biopsy


A treatment in which the patient receives healthy, immature The removal of a small amount of solid bone and bone
blood-forming cells from another person to replace damaged marrow to test for disease.
or diseased cells in the bone marrow.
chemotherapy
anemia Treatment with drugs that kill abnormal cells or stop new
A condition in which the number of red blood cells is low. ones from being made.

BCR-ABL1 gene chromosomes


An abnormal gene that is formed when parts of Long strands that contain bundles of coded instructions in
chromosomes 9 and 22 break off and switch with each other. cells for making and controlling cells.
This gene is found on the Philadelphia chromosome and is
the key feature of chronic myeloid leukemia.
chronic myeloid leukemia (CML)
A slow-growing cancer that starts in the bone marrow and
biopsy causes too many white blood cells called granulocytes to
Removal of small amounts of tissue from the body to be form.
tested for disease.
clinical trial
blast cell Research on a test or treatment to assess its safety or how
An immature blood cell. well it works.

blood cell count complete blood count (CBC)


The number of blood cells in a sample of blood. A test of the number of blood cells in a sample.

blood cell growth factors conditioning treatment


Substances that cause new blood cells to grow in the bone Treatment that is used to destroy cells in the bone marrow
marrow. to prepare (condition) the body for a hematopoietic cell
transplant.
blood smear
A test in which a drop of blood is placed on a slide and cycle
viewed with a microscope to assess the size, shape, type, Days of treatment followed by days of rest.
and maturity of the blood cells.
cytogenetic testing
blood stem cell A test that uses a microscope to examine a cells
An immature blood-forming cell from which all other types of chromosomeslong strands of coded instructions in cells for
blood cells are made. Also called hematopoietic stem cell. making and controlling cells.

bloodstream cytopenia
Blood that flows throughout the body in small tubes called A condition in which the number of blood cells is low.
blood vessels.
del(5q)
bone marrow An abnormal chromosome change in which the q part of
The soft, sponge-like tissue in the center of most bones chromosome 5 is missing (deleted).
where blood cells are made.

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Glossary Dictionary

deoxyribonucleic acid (DNA) gene mutation


A chain of chemicals in cells that contains coded instructions An abnormal change in the coded instructions in cells for
for making and controlling cells. making and controlling cells.

diagnose genetic tests


To confirm or identify a disease or health condition. Tests of the coded instructions in cells that are needed to
make and control cells. These instructions are called genes
differential and they are grouped into long strands called chromosomes.
Measurement of the different types of white blood cells
present in a blood sample. graft-versus-host disease (GVHD)
A disease that occurs when transplanted blood stem cells
donor (immature blood-forming cells) from a donor attack a
A person who gives their organs, tissues, or cells to another
patients normal cells.
person.
graft-versus-leukemia (GVL) effect
donor lymphocyte infusion (DLI) An attack on cancer cells by transplanted blood stem cells
Procedure in which the patient receives white blood cells
(immature blood-forming cells) from a donor.
from the same person who donated blood-forming cells
(blood stem cells) for the stem cell transplant. granulocyte colony-stimulating factor (G-CSF)
A substance that helps (stimulates) the bone marrow to
dysplasia make more white blood cells called neutrophils. It is made
Cells have an abnormal size, shape, or look (appearance)
naturally in the body but can also be made in a lab.
when viewed with a microscope.
granulocyte-macrophage colony-stimulating factor
erythropoiesis-stimulating agent (ESA) (GM-CSF)
A drug that tells (stimulates) the bone marrow to make more
A substance that helps (stimulates) the bone marrow to
red blood cells.
make more of certain types of white blood cells. It is made
erythropoietin (EPO) naturally in the body but can also be made in a lab.
A substance that is made naturally in the body and that tells
hematopoietic cell transplant (HCT)
(stimulates) the bone marrow to make more red blood cells.
A treatment that replaces damaged or diseased cells in the
fatigue bone marrowsponge-like tissue in the center of bones
Severe tiredness despite getting enough sleep that limits where blood cells are madewith healthy blood-forming
ones ability to function. cells. Also called stem cell transplant.

flow cytometry hematopoietic stem cell or hematopoietic cell


A test that looks at certain substances on the surface of cells An immature blood-forming cell from which all other types of
to identify the type of cells present. blood cells are made. Also called blood stem cell.

fluorescence in situ hybridization (FISH) hemoglobin


A lab test that uses special dyes to look for abnormal A protein in red blood cells that carries oxygen.
changes in a cells genes and chromosomes.
high-intensity chemotherapy
folate Treatment with high doses of strong cancer drugs that are
A nutrient in the body that is needed to make red blood cells. more likely to cause severe side effects.

gene high-intensity treatment


A set of coded instructions in cells for making and controlling Treatment that is more likely to cause severe side effects
cells. and often requires a hospital stay.

higher-risk MDS
MDS that is more likely to progress faster or turn into acute
myeloid leukemia quickly if not treated.

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HLA-DR15 intravenous (IV)


An immune system protein that is found on the surface of Given by a needle or tube inserted into a vein.
some cells. The presence of the protein on MDS cells can
affect how well immunosuppressive therapy works.
intravenous (IV) infusion
A method of giving drugs slowly through a needle into a vein.
hormone
A chemical in the body that activates cells or organs.
iron
A mineral that is found in red blood cells and that the body
human leukocyte antigen (HLA) needs to make new red blood cells.
Special proteins on the surface of cells that help the body to
tell its own cells apart from foreign cells.
iron chelation therapy
Treatment that is used to remove excess iron from the body.
human leukocyte antigen (HLA) type
The unique set of proteins on the surface of cells that help
iron overload
The buildup of excess iron in the body.
the body to tell its own cells apart from foreign cells.

human leukocyte antigen (HLA) typing local anesthesia


A controlled loss of feeling in a small area of the body
A blood test that finds a persons HLA typethe unique set
caused by drugs.
of proteins on the surface of cells that help the body to tell its
own cells apart from foreign cells. low-intensity chemo
Treatment with cancer drugs that are less likely to cause
hypocellular bone marrow
severe side effects.
The number of cells in the bone marrow is lower than
normal. low-intensity treatment
Treatment that is less likely to cause severe side effects and
immune response
usually does not require a hospital stay.
The action of the bodys natural defense against infections
and disease in response to foreign substances. lower-risk MDS
MDS that is more likely to grow and progress slowly and may
immune system
not cause many or severe symptoms for a long time.
The bodys natural defense against infection and disease.

immunomodulators lymphocyte
A type of white blood cell that helps protect the body from
Drugs that change (modify) different parts of the immune
infection and disease.
system.

immunosuppressive therapy (IST) medical history


All health events and medications taken to date.
Treatment with drugs that weaken (suppress) the bodys
immune system. microscope
A tool that uses lenses to see things the eyes cant.
immunotherapy
Treatment with drugs that modify the immune system to help molecular test
the body fight cancer. Tests that look for abnormal changes in genes known to
have an effect on cancer treatment or outcomes.
International Prognostic Scoring System (IPSS)
A system that doctors use to rate the severity of MDS monocyte
and classify it into groups based on the likely outcome A type of white blood cell.
(prognosis).
mutation
intestine An abnormal change.
The organ that food passes through after leaving the
stomach.

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myeloproliferative neoplasm (MPN) red blood cell


A cancer in which the bone marrow makes too many red A type of blood cell that carries oxygen from the lungs to the
blood cells, white blood cells, or platelets. rest of the body.

neutropenia red blood cell growth factor


A condition in which the number of white blood cells called A substance that causes new red blood cells to grow in the
neutrophils is low. bone marrow. It is made naturally in the body but can also be
made in a lab to use as treatment.
neutrophil
A type of white blood cell that helps fight infections and has red blood cell transfusion
small particles (granules). A slow injection of red blood cells into a vein.

pathologist reduced-intensity conditioning


A doctor whos an expert in testing cells and tissue to find Treatment with lower doses of strong cancer drugs or
disease. low-intensity drugs that is used to destroy cells in the bone
marrow to prepare (condition) the body for a hematopoietic
Philadelphia chromosome cell transplant.
An abnormal chromosome that is formed when parts of
chromosomes 9 and 22 break off and switch with each regimen
other. It contains the abnormal BCR-ABL1 gene and is a key A treatment plan that specifies the dose, schedule, and
feature of chronic myeloid leukemia. duration of treatment.

physical exam relapse


A review of the body by a health expert for signs of disease. The return or worsening of cancer after a period of
improvement.
platelet
A type of blood cell that helps control bleeding. reticulocyte
Younger (precursor) cells that become mature red blood
platelet transfusion cells.
A slow injection of plateletsblood cells that help control
bleedinginto a vein. Revised International Prognostic Scoring System
(IPSS-R)
prognosis A newer system that doctors use to rate the severity of
The likely or expected course, pattern, and outcome of a
MDS and classify it into groups based on the likely outcome
disease based on tests.
(prognosis).
prognostic factor ring sideroblasts
Something that affects and helps predict the likely pattern
Young red blood cells that have too much iron and it shows
and outcome of a disease.
up as a circle (ring) around the center of the cells.
prognostic scoring system risk group
A system that doctors use to rate the severity of MDS
Classification of MDS based on its severity and the chance
and classify it into groups based on the likely outcome
(risk) that it will progress to AML (acute myeloid leukemia).
(prognosis).
risk score
progression A rating of the severity of MDS that describes how fast or
The course of a disease as it grows, gets worse, or spreads
slow it will likely grow and progress.
in the body.
sedative
protein A drug that helps a person to relax or go to sleep.
A chain of small chemical compounds important to every cell
in the body.

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Glossary Dictionary

serum EPO treatment response


The amount of natural erythropoietina substance made in An outcome or improvement in disease that is caused by
the body that causes red blood cells to growthat is found in treatment.
the blood.
U.S. Food and Drug Administration (FDA)
side effect A federal government agency that regulates drugs and food.
An unhealthy or unpleasant physical or emotional condition
caused by treatment.
vitamin B12
A nutrient in the body that is needed to make red blood cells.
subtype
Smaller groups that a type of cancer is divided into based on
white blood cell
A type of blood cell that helps fight infections in the body.
certain features of the cancer cells.

supportive care white blood cell growth factor


A substance that causes new white blood cells to grow in the
Treatment for the symptoms or health conditions caused by
bone marrow. It is made naturally in the body but can also be
cancer or cancer treatment.
made in a lab to use as treatment.
symptom
A new or changed health problem a person experiences that
WHO classification-based Prognostic Scoring
may indicate a disease.
System (WPSS)
A system that doctors use to rate the severity of MDS
thrombocytopenia and classify it into groups based on the likely outcome
A condition in which there is a low number of platelets (prognosis).
blood cells that help control bleeding.

transfusion
A slow injection of whole blood or parts of blood into a vein.

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Glossary Acronyms

Acronyms
AML HCT WPSS
acute myeloid leukemia hematopoietic cell transplant WHO classification-based Prognostic
Scoring System
ATG HIV
antithymocyte globulin human immunodeficiency virus

CBC HLA
NCCN Abbreviations and
complete blood count human leukocyte antigen Acronyms
CML IPSS NCCN
chronic myeloid leukemia International Prognostic Scoring National Comprehensive Cancer
System Network
CMML
chronic myelomonocytic leukemia IPSS-R NCCN Patient Guidelines
Revised International Prognostic NCCN Guidelines for Patients
DLI Scoring System
donor lymphocyte infusion
NCCN Guidelines
IST
DNA NCCN Clinical Practice Guidelines in
immunosuppressive therapy
deoxyribonucleic acid Oncology
IV
EPO intravenous
erythropoietin
MDS
ESA myelodysplastic syndromes
erythropoietin-stimulating agent
MDS/MPN
FAB myelodysplastic/myeloproliferative
French-American-British
neoplasms
FDA mU/mL
U.S. Food and Drug Administration
milliunits per milliliter
FISH RAEB
fluorescence in situ hybridization
refractory anemia with excess blasts
G-CSF RARS
granulocyte colony-stimulating factor
refractory anemia with ring sideroblasts
GM-CSF RCMD
granulocyte-macrophage granulocyte
refractory cytopenia with multilineage
colony-stimulating factor
dysplasia
GVHD RCUD
graft-versus-host disease
refractory cytopenia with unilineage
GVL dysplasia
graft-versus-leukemia
WHO
World Health Organization

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N C C N G U I D E L I N E S F O R PAT I E N T S
Patient-friendly versions of the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines)

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Acute Lymphoblastic Leukemia Malignant Pleural Mesothelioma Non-Small Cell Lung Cancer
Caring for Adolescents and Melanoma Ovarian Cancer
Young Adults (AYA)* Multiple Myeloma Pancreatic Cancer
Chronic Lymphocytic Leukemia Myelodysplastic Syndromes Prostate Cancer
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Colon Cancer Diffuse Large B-cell Lymphoma Stage 0 Breast Cancer
Esophageal Cancer Follicular Lymphoma
Stages I and II Breast Cancer
Mantle Cell Lymphoma
Hodgkin Lymphoma Stage III Breast Cancer
Mycosis Fungoides
Kidney Cancer Peripheral T-cell Lymphoma Stage IV Breast Cancer
Lung Cancer Screening

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NCCN Guidelines for Patients
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State Fundraising Notices

State Fundraising Notices

FLORIDA: A COPY OF THE OFFICIAL REGISTRATION gov/ocp.htm#charity. REGISTRATION WITH THE ATTORNEY
AND FINANCIAL INFORMATION OF NCCN FOUNDATION GENERAL DOES NOT IMPLY ENDORSEMENT. NEW YORK:
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and fair description of the programs and activities of NCCN STATE SOLICITATION LICENSING BRANCH AT 888-830-
Foundation; and (B) A financial statement or summary which 4989 (within North Carolina) or (919) 807-2214 (outside of
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IS AVAILABLE ON THE INTERNET AT www.njconsumeraffairs.

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NCCN Panel Members

NCCN Panel Members for


Myelodysplastic Syndromes
Peter L. Greenberg, MD/Chair Karin Gaensler, MD Benton Wheeler, MD
Stanford Cancer Institute UCSF Helen Diller Family St. Jude Childrens Research Hospital/
Comprehensive Cancer Center The University of Tennessee
Richard M. Stone, MD/Vice Chair Health Science Center
Dana-Farber/Brigham and Womens Guillermo Garcia-Manero, MD
Cancer Center The University of Texas Amer Zeidan, MBBS, MHS
MD Anderson Cancer Center Yale Cancer Center/
Aref Al-Kali, MD Smilow Cancer Hospital
Mayo Clinic Cancer Center Elizabeth A. Griffiths, MD
Roswell Park Cancer Institute
Rafael Bejar, MD, PhD
UC San Diego Moores Cancer Center David Head, MD
Vanderbilt-Ingram Cancer Center
John M. Bennett, MD
Consultant Virginia M. Klimek, MD
Memorial Sloan Kettering Cancer Center
Clara D. Bloomfield, MD
The Ohio State University Comprehensive Rami Komrokji, MD
Cancer Center - James Cancer Hospital Moffitt Cancer Center
and Solove Research Institute
Lisa A. Kujawski, MD
Uma Borate, MD University of Michigan
University of Alabama at Birmingham Comprehensive Cancer Center
Comprehensive Cancer Center
Lori J. Maness, MD
Carlos M. De Castro, MD Fred & Pamela Buffett Cancer Center
Duke Cancer Institute
Margaret R. ODonnell, MD
H. Joachim Deeg, MD City of Hope Comprehensive Cancer Center
Fred Hutchinson Cancer Research Center/
Seattle Cancer Care Alliance Daniel A. Pollyea, MD, MS
University of Colorado Cancer Center
Amy E. DeZern, MD, MHS
The Sidney Kimmel Comprehensive Paul J. Shami, MD
Cancer Center at Johns Hopkins Huntsman Cancer Institute
at the University of Utah
Amir T. Fathi, MD
Massachusetts General Hospital Brady L. Stein, MD, MHS
Cancer Center Robert H. Lurie Comprehensive Cancer
Center of Northwestern University
Olga Frankfurt, MD
Robert H. Lurie Comprehensive Cancer Peter Westervelt, MD, PhD
Center of Northwestern University Siteman Cancer Center at Barnes-
Jewish Hospital and Washington
University School of Medicine

For disclosures, visit www.nccn.org/about/disclosure.aspx.

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NCCN Member Institutions

NCCN Member Institutions


Fred & Pamela Buffett Cancer Center The Sidney Kimmel Comprehensive Stanford Cancer Institute
Omaha, Nebraska Cancer Center at Johns Hopkins Stanford, California
800.999.5465 Baltimore, Maryland 877.668.7535
nebraskamed.com/cancer 410.955.8964 cancer.stanford.edu
hopkinskimmelcancercenter.org
Case Comprehensive Cancer Center/ University of Alabama at Birmingham
University Hospitals Seidman Robert H. Lurie Comprehensive Cancer Comprehensive Cancer Center
Cancer Center and Cleveland Clinic Center of Northwestern University Birmingham, Alabama
Taussig Cancer Institute Chicago, Illinois 800.822.0933
Cleveland, Ohio 866.587.4322 www3.ccc.uab.edu
800.641.2422 UH Seidman Cancer Center cancer.northwestern.edu
uhhospitals.org/seidman UC San Diego Moores Cancer Center
866.223.8100 CC Taussig Cancer Institute Mayo Clinic Cancer Center La Jolla, California
my.clevelandclinic.org/services/cancer Phoenix/Scottsdale, Arizona 858.657.7000
216.844.8797 Case CCC Jacksonville, Florida cancer.ucsd.edu
case.edu/cancer Rochester, Minnesota
800.446.2279 Arizona UCSF Helen Diller Family
City of Hope Comprehensive 904.953.0853 Florida Comprehensive Cancer Center
Cancer Center 507.538.3270 Minnesota San Francisco, California
Los Angeles, California mayoclinic.org/departments-centers/mayo- 800.689.8273
800.826.4673 clinic-cancer-center cancer.ucsf.edu
cityofhope.org
Memorial Sloan Kettering University of Colorado Cancer Center
Dana-Farber/Brigham and Cancer Center Aurora, Colorado
Womens Cancer Center New York, New York 720.848.0300
Massachusetts General Hospital 800.525.2225 coloradocancercenter.org
mskcc.org
Cancer Center
Boston, Massachusetts University of Michigan
877.332.4294 Moffitt Cancer Center Comprehensive Cancer Center
dfbwcc.org Tampa, Florida Ann Arbor, Michigan
massgeneral.org/cancer 800.456.3434 800.865.1125
moffitt.org mcancer.org
Duke Cancer Institute
Durham, North Carolina The Ohio State University The University of Texas
888.275.3853 Comprehensive Cancer Center - MD Anderson Cancer Center
dukecancerinstitute.org James Cancer Hospital and Houston, Texas
Solove Research Institute 800.392.1611
Fox Chase Cancer Center Columbus, Ohio mdanderson.org
Philadelphia, Pennsylvania 800.293.5066
888.369.2427 cancer.osu.edu Vanderbilt-Ingram Cancer Center
foxchase.org Nashville, Tennessee
Roswell Park Cancer Institute 800.811.8480
Huntsman Cancer Institute Buffalo, New York vicc.org
at the University of Utah 877.275.7724
Salt Lake City, Utah roswellpark.org University of Wisconsin
877.585.0303 Carbone Cancer Center
huntsmancancer.org Siteman Cancer Center at Barnes- Madison, Wisconsin
Jewish Hospital and Washington 608.265.1700
Fred Hutchinson Cancer University School of Medicine uwhealth.org/cancer
Research Center/ St. Louis, Missouri
Seattle Cancer Care Alliance 800.600.3606 Yale Cancer Center/
Seattle, Washington siteman.wustl.edu Smilow Cancer Hospital
206.288.7222 seattlecca.org New Haven, Connecticut
206.667.5000 fredhutch.org St. Jude Childrens Research Hospital/ 855.4.SMILOW
The University of Tennessee yalecancercenter.org
Health Science Center
Memphis, Tennessee
888.226.4343 stjude.org
901.683.0055 westclinic.com

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Index

Index
acute myeloid leukemia (AML) 9, 11, 21, 27, 2930,
3637, 47, 49, 52, 5455, 66
anemia 13, 1819, 2829, 3234, 46, 4853, 58, 6061
blood cell growth factor 3334, 49, 61
blood stem cell 78, 14, 3940, 44
bone marrow 4, 712, 14, 1722, 24, 2728, 3334,
3640, 44, 46, 49, 52, 55, 6061
chemotherapy 3637, 3940, 44
chromosome 8, 1012, 2122, 24, 2728, 38, 48, 6067
clinical trial 42, 44, 4857, 67
cytopenia 10, 13, 16, 18, 27
dysplasia 8, 10, 12, 18, 2021
gene 8, 12, 2124, 36
hematopoietic cell transplant (HCT) 20, 3940, 44, 5056
higher-risk MDS 2930, 4647, 5457
high-intensity chemo 3637, 5455
immunosuppressive therapy (IST) 3738, 4852
lower-risk MDS 2930, 38, 4653
low-intensity chemo 36, 4950, 52, 5556
prognosis 2224, 2628, 30, 66
prognostic scoring 2630
risk group 2730, 39, 47, 66
risk score 2630, 47, 66
serum EPO 17, 19, 34, 4849, 6061
side effect 32, 3640, 42, 4850, 5455, 60, 67
subtype 1012, 14, 23, 2729, 47
supportive care 3235, 44, 5658
transfusion 16, 20, 3334, 3839, 4749, 52, 58, 6061

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NCCN Guidelines for Patients

Myelodysplastic
Syndromes
Version 1.2016

NCCN Foundation gratefully acknowledges our advocacy supporter, Aplastic Anemia and MDS International Foundation, and our
industry supporter Amgen, Inc. for their support in making available these NCCN Guidelines for Patients. NCCN independently
develops and distributes the NCCN Guidelines for Patients. Our industry supporters do not participate in the development of the NCCN
Guidelines for Patients and are not responsible for the content and recommendations contained therein.

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