Hematopoietic Stem Cell Transplant At The Rescue Of Acute

Myelofibrosis
Stem cell transplant: Hematopoietic cell transplant (HCT) has all the powerful elements in
its treatment procedures to offer better and curative treatment opportunities for patients suffering
from acute Myelofibrosis. This is a rare kind of cancer, which prevents the growth of blood-
producing cells, and destroys the existing and new cells more rapidly. And as a result, the bone
marrow develops scars with the development of fibrous tissues within the bone marrow. So, with
the occurrence of this disease, a series of health complications occur in the patients.

Health Complications of Acute Myelofibrosis

This type of leukemia belongs to the group of Myeloproliferative Neoplasms. About

30,000 of the USA citizens are suffering from this disease when their blood begins
overproducing the red blood cells. So, the cells cannot produce enough blood in the body, and
the body tries to move the blood production to the spleen and liver. And as the bone marrow
cannot perform well, it pressurizes the spleen and liver and enlarges them. The disease causes
blood clots and extreme bleeding to the people with acute myelofibrosis.

Other complications include weakness, fatigue, anemia, abdominal pain, low platelet counts and
prevalence of infections.

People between 50 and 70 are at high risk of developing this disease.

Treatment Options for Acute Myelofibrosis

The treatment options depend on the symptoms and the intensity of the complications. There are
different drugs used to treat this disease.

A drug called Ruxolitinib targets the inhibitors known as JAK2 and other associated mutants.
The chemical compounds of the drug effectively work and restrict the functions of the mutant
protein tyrosine kinase, which can be transformed and active in people with myelofibrosis.

There are other drugs too, such as danazol, thalidomide, and erythropoietin. The most effective
and the curative treatment process seem to be the stem cell transplant or the bone
marrow transplant. However, a fewer number of patients can benefit from the transplant,
which also involves treatment-related morbidity and mortality.
 Image- Flickr.com

Varied Stem Cell Options for the Treatment of Myelofibrosis

Allogeneic hematopoietic cell transplantation (HCT) is the major treatment

choice for myelofibrosis. As older patients cannot stand the toxicity of the Full-Intensity
Conditioning (FIC) because of its high rate of non-relapse mortality (NRM), healthy and
young patients are not advised to opt for this option. This is the reason why Reduced-Intensity-
Conditioning (RIC) provides much better treatment options for MF.

Read-

 Acute Biphenotypic Leukemia Treatment With Stem Cell Therapy

Candidacy for RIC

Since Myelofibrosis is a disease of elderly people, and it triggers more risks associated with poor
transplantation results. So, the determination to perform RIC prior to HSCT relies upon several
variables such as - patients and disease-related parameters, performance capability, age, and
complication biology as per the standard Dynamic International Prognostic
Scoring System or (DIPSS). The determination of the candidature for the RIP is based on
the risk factors. They are as follows as below:

1. >65 years age

2. Hemoglobin count <10g/dL
 3. White blood cell counts >25x109/L
4. Peripheral blood blasts ≥ 1% and
5. Constitutional signs

Patients with low or intermediate-1 risks factors may opt for the stem cell transplant as per
another scoring system, Dynamic International Prognostic Scoring System, but they are not
without any side effects. Certain mutations such as JAK2, MPL, and CALR cause the
occurrences of primary myelofibrosis in the patients with low or intermediate-1 risk, cannot opt
for the RIC. Contrary to it, patients with inhibitors or mutant proteins such as SRSF2 and
ASXL1 require an instant transplant.

Eligibility of the Donor

The type of donor determines the survival rates for the patients. According to various
studies, human leukocyte antigen (HLA), matched donor, preferably with siblings is the
ideal donor type. It offers 75% of successful results for 2-year overall survival. It is followed by
the matched unrelated donor. With this type of donor, the survival rate becomes weaker and 32%
success rate is established for 2-year overall survival. And with the RIC, patients with matched
donor transplant can survive for five years at a percentage of 56, matched unrelated donor
survival rates is at 48%, while 34% of success rate is established for the mismatched unrelated
donor type.

It is uncertain if the Splenomegaly offers any survival opportunities, prior to SCT. It leads to
several health complications, including impairment of the liver functions.