disease passed down through families (inherited) that mainly affects the bone marrow.

results in decreased production of all types of blood cells

Due to an abnormal gene that damages cells, which keeps them from repairing damaged DNA. - to inherit Fanconis Anemia, one must get one copy of the abnormal gene from each parent - Usually diagnosed in children between 2 and 15 years old.
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Have lower-than-normal numbers of WBC, RBC, and platelets - Not enough WBC can lead to infections. Lack of RBC may result in fatigue (anemia) - Lower-than-normal amount of platelets may lead o excess bleeding.
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Abnormal heart, lungs, and digestive tract Bone problems (esp the hips, spine or ribs, can cause a curved spine or scoliosis) Changes in the color of skin, such as: darkened areas of the skin called Caffe au lait spots; vitiligo Deafness due to abnormal ears Eye or eyelid problems Problems with the arms and hands, such as: missing, extra or misshapen thumbs Short height Small head Small testicles and genital changes

Other Possible Symptoms: - Failure to thrive - Learning disability - Low birth weight - Intellectual disability

Bone marrow biopsy CBC Developmental tests Drugs added to a blood sample to check for damage to chromosomes Hand x-ray and other imaging studies (CT scan, MRI) Hearing test HLA tissue typing (to find matching bone marrow donors) Ultrasound of kidneys Pregnant women may have amniocentesis or chronic villi sampling to diagnose the condition in their unborn child

Patients with mild to moderate blood cell changes who do not need a transfusion may only need regular check-ups and blood count checks. Monitor for other cancers, usually leukemia or cancers of the head, neck or urinary systems Medicines called growth factors (such as erythropoietin, G-CSF and GM-CSF) can improve blood counts for a short while Bone marrow transplant can cure blood count problems (best donor: brother or sister) Hormone therapy combined with low doses of steroids (hydrocortisone or prednisone) is prescribed to those who do not have bone marrow donor

Additional treatment: - antibiotics (possibly through IV) to treat infections - Blood transfusions to treat symptoms due to low blood counts

Poor outlook in those with low blood counts. New and improved transplants such as bone marrow transplant, may improved survival. More likely to develop several types of blood disorders and cancers, including leukemia, myelodysplastic syndrome, and cancer of the head, neck or urinary systems. Women with Fanconis anemia who become pregnant should be watched carefully by the doctor. They often need transfusions throughout pregnancy. Men with Fanconis anemia have decreassed fertility

Families with this condtion can have genetic counseling to have better understanding of their risks - Vaccination can reduce other complications such as pneumococcal pneumonia, hepatitis and varicella infections - They should avoid cancer-causing substances (carcinogens) and have regular check-ups to screen for cancer
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Fanconi

anemia is one of the few forms of aplastic anemia in which the response to androgens is more than 50%. Hematopoietic growth factors are occasionally helpful in Fanconi anemia.

Class Summary These enhance the production and urinary excretion of erythropoietin in anemias caused by bone marrow failure and often stimulate erythropoiesis in anemias caused by deficient red blood production. They appear to make hematopoietic stem cells more responsive to differentiation, but the exact mechanism is not clear. The usual agent is oral oxymetholone. Although oral androgens have a risk of liver toxicity, they are easier to use in children than parenteral androgens. The lowest effective dose should be used. Recent studies suggest that the less potent androgen, danazol, may be effective in delaying the onset of clinically significant cytopenias in patients with Fanconi anemia.

Dosing Forms & Strengths tablet: Schedule III 50mg Anemia Due to Deficient Red Cell Production 1-5 mg/kg PO qDay Includes acquired aplastic anemia, congenital anemia, myelofibrosis, & hypoplastic anemia due to admin of myelotoxic drugs Off-label: HIV-associated wasting Use with caution in children

This medication is a synthetic male hormone (androgen or anabolic steroid) used to treat a low red blood cell count (anemia). It works by increasing the amount of the hormone (erythropoietin) involved in the production of red blood cells. Due to the risk of serious, possibly life-threatening side effects, this medication must not be used to improve athletic performance or physical appearance. Oxymetholone does not enhance athletic ability. When used as directed under medical supervision, the risks are minima

Take this medication by mouth as directed by your doctor. It may be taken with food or milk if stomach upset occurs. Use this medication regularly in order to get the most benefit from it. Remember to use it at the same time(s) each day. Dosage is based on your medical condition and response to therapy. Do not increase your dose or take this more often than directed since the risk of side effects may be increased. It may take between 3-6 months before a benefit from this medication occurs.

Males Impotency Bladder irritability

Females Alopecia Menstrual irregularities

Pediatrics Diarrhea Nausea Vomiting Electrolyte & water retention Muscle cramps

WARNING: This medication may rarely cause serious, sometimes life-threatening liver problems including cysts, tumors, or liver failure. Tell your doctor immediately if you have dark urine, yellowing eyes or skin, persistent nausea/vomiting, stomach/abdominal pain, or unusual fatigue. In addition, this medication may affect your cholesterol level and increase your risk of heart disease (atherosclerosis) or blood vessel problems.

Before taking oxymetholone, tell your doctor or pharmacist if you are allergic to it; or if you have any other allergies. This product may contain inactive ingredients, which can cause allergic reactions or other problems. Talk to your pharmacist for more details. If you have diabetes, oxymetholone may increase your blood sugar level. Check your blood (or urine) glucose level frequently, as directed by your doctor and promptly report any abnormal results. Your diabetes medicines may need to be adjusted.

Caution is advised when using this drug in children because they may be more sensitive to its effects. This medication may stunt growth in children under 18 years of age. Periodic bone x-rays may be used to monitor the drug's effects on bone growth. Oxymetholone may also affect sexual development in children. Consult your doctor for more details. This medication must not be used during pregnancy due to the risk of harm to an unborn baby. If you become pregnant or think you may be pregnant, inform your doctor immediately. It is recommended that young girls and women of child-bearing age use effective birth control while taking this drug.

This medication must not be used during pregnancy. It may harm an unborn baby. In women of childbearing age, this medication should be started during their menstrual period. If not, then they should have a negative pregnancy test before starting this medication. It is important to prevent pregnancy while using this medication. Consult your doctor for more details and to discuss the use reliable forms of non-hormonal birth control (such as condoms, diaphragm with spermicide) while using this medication. If you become pregnant or think you may be pregnant, tell your doctor immediately.

Danazol has rarely caused very serious (possibly fatal) blood clots (such as stroke), liver disease, and increased pressure on the brain (benign intracranial hypertension). Seek immediate medical attention if you have any symptoms of these side effects, including weakness on one side of the body, slurred speech, vision changes, severe stomach/abdominal pain, dark urine, persistent nausea/vomiting, yellowing eyes/skin, mental/mood changes (such as confusion), severe/persistent headache.

This medication is used in women to treat pelvic pain and infertility due to a certain uterus disorder (endometriosis) and also to treat breast pain/tenderness/nodules due to a certain breast condition (fibrocystic breast disease). It is also used in both men and women to prevent swelling of the abdomen/arms/legs/face/airway due to a certain congenital disease (hereditary angioedema). It works by decreasing the amount of hormones made by the ovaries. These hormones usually make the conditions worse. For the treatment of angioedema, danazol helps to increase the amount of a certain protein in your body's defense system (immune system).

Take this medication by mouth, usually twice daily or as directed by your doctor. You may take this medication with or without food, but it is important to choose one way and take this medication the same way with every dose. The dosage is based on your medical condition and response to treatment. Use this medication regularly to get the most benefit from it. To help you remember, take it at the same times each day. Tell your doctor if your condition does not improve or if it worsens.

Weight gain, acne, flushing, sweating, voice changes (hoarseness, change in pitch), abnormal growth of body hair (in women), vaginal dryness/irritation, or decreased breast size may occur. If any of these effects persist or worsen, tell your doctor or pharmacist promptly. Tell your doctor immediately if any of these unlikely but serious side effects occur: swelling hands/ankles/feet, menstrual changes (such as spotting, missed periods), mental/mood changes (such as nervousness, mood swings). A very serious allergic reaction to this drug is rare. However, seek immediate medical attention if you notice any symptoms of a serious allergic reaction, including: rash, itching/swelling (especially of the face/tongue/throat), severe dizziness, trouble breathing.

This medication is used to treat severe bleeding caused by problems with the blood clotting system, which helps you to stop bleeding. Aminocaproic acid works by affecting the blood clotting system, thereby slowing/stopping bleeding after surgery, in some bleeding conditions, or in severe liver disease.

Take this medication by mouth exactly as directed by your doctor. It is important to take each dose at the correct time for this medication to be most effective. Do not skip any doses or take this medication more often than prescribed. If you are using the liquid form of this medication, measure your dose with a special measuring spoon or device. Do not use a household spoon because it may not provide the correct dose. Dosage is based on your medical condition and response to therapy. Inform your doctor if your condition persists or worsens.

Headache, stomach pain, loss of appetite, nausea, vomiting, diarrhea, unusual tiredness, dizziness, stuffy nose, or watery eyes may occur. If any of these effects persist or worsen, tell your doctor or pharmacist promptly. Seek immediate medical attention if any of these rare but very serious side effects occur: chest pain, seizures. A very serious allergic reaction to this drug is rare. However, seek immediate medical attention if you notice any symptoms of a serious allergic reaction, including: rash, itching/swelling (especially of the face/tongue/throat), severe dizziness, trouble breathing.

This medication stimulates the blood system (bone marrow) to make white blood cells, helping you fight infections. This medication is given to those whose ability to make white blood cells has been reduced. Filgrastim (also known as GCSF, or granulocyte colony stimulating factor) is a man-made version of a certain natural substance found in the body. It is produced using a certain bacteria.

This medication is given by injection into a vein or under the skin, usually once a day or as directed by your doctor, until the proper blood counts are reached. Dosage is based on your medical condition, weight, and response to therapy. Use the exact amount of drug prescribed by your doctor. Too little drug may not protect you against infections. Too much drug may cause your body to make too many white blood cells. Avoid shaking this medication; doing so may make the drug ineffective.

Aching in the bones and muscles may occur. Taking a nonaspirin pain reliever such as acetaminophen may help with this pain. Ask your doctor or pharmacist for more details. Nosebleeds or injection site reactions such as redness, swelling, itching, lumps or bruising may also occur. If any of these effects persist or w Tell your doctor immediately if any of these rare but very serious side effects occur: easy bleeding/bruising, bloody urine, bloody vomit, fast/irregular heartbeat, fever, muscle pain, joint pain, fast breathing, trouble breathing. Rarely, possibly fatal damage to the spleen may occur. Seek immediate medical attention if you experience the following side effects: stomach/abdominal pain, and/or shoulder pain. orsen, notify your doctor or pharmacist promptly.

Common tests for Fanconi's anemia include: Bone marrow biopsy Complete blood count (CBC) Developmental tests Drugs added to a blood sample to check for damage to chromosomes Hand x-ray and other imaging studies (CT scan, MRI) Hearing test HLA tissue typing (to find matching bone-marrow donors) Ultrasound of the kidneys Pregnant women may have amniocentesis or chorionic villous sampling to diagnose the condition in their unborn child.

Patients with mild to moderate blood cell changes who do not need a transfusion may only need regular check-ups and blood count checks. The health care provider will closely monitor the person for other cancers, usually leukemia or cancers of the head, neck, or urinary system. A bone marrow transplant can cure the blood count problems of Fanconi's anemia. (The best donor is a brother or sister whose tissue type matches the patient.) Persons who have had a successful bone marrow transplant still need regular check-ups because of the risk for additional cancers.

Hormone therapy combined with low doses of steroids (such as hydrocortisone or prednisone) is prescribed to those who do not have a bone marrow donor. Most patients respond to hormone therapy. But everyone with the disorder will quickly get worse when the drugs are stopped. In most cases, these drugs eventually stop working. Additional treatments may include: Antibiotics (possibly given through a vein) to treat infections Blood transfusions to treat symptoms due to low blood counts

Bone marrow transplant A bone marrow transplant is a procedure to replace damaged or destroyed bone marrow with healthy bone marrow stem cells. Bone marrow is the soft, fatty tissue inside your bones. Stem cells are immature cells in the bone marrow that give rise to all of your blood cells.

Autologous bone marrow transplant: "Auto" means "self." Stem cells are removed from you before you receive high-dose chemotherapy or radiation treatment and stored in a freezer (cryopreservation). After high-dose chemotherapy or radiation treatments are done, your stems cells are put back in your body to add to your normal blood cells. This is called a "rescue" transplant. Allogeneic bone marrow transplant: "Allo" means "other." Stem cells are removed from another person, called a donor. Most times, the donor must at least partly match you genetically. Special blood tests are done to determine if a donor is a good match for you. A brother or sister is most likely to be a good match. However, sometimes parents, children, and other relatives may be good matches. Donors who are not related to you may be found through national bone marrow registries.

Umbilical cord blood transplant: Stem cells are removed from a newborn baby's umbilical cord immediately after birth. The stem cells are stored until they are needed for a transplant. Umbilical cord blood cells are so immature, there is less of a need for matching.

Ablative (myeloablative) treatment: High-dose chemotherapy, radiation, or both are given to kill any cancer cells. This also kills all healthy bone marrow that remains, and allows new stem cells to grow in the bone marrow. Reduced intensity (nonmyeloablative) treatment, also called a mini transplant: Patients receive lower doses of chemotherapy and radiation before a transplant. This has allowed older patients, and patients with other health problems to have a transplant.

A stem cell transplant is done after chemotherapy and radiation is complete. The stem cells are delivered into your bloodstream through a tube called a central venous catheter. The process is similar to getting a blood transfusion. The stem cells travel through the blood into the bone marrow. Usually, no surgery is needed.

Bone marrow harvest. This minor surgery is performed under general anesthesia, meaning the donor will be asleep and pain-free during the procedure. The bone marrow is removed from the back of both hip bones. The amount of marrow removed depends on the weight of the person who is receiving it. Leukapheresis. First, the donor is given 5 days of shots to help stem cells move from the bone marrow into the blood. During leukapheresis, blood is removed from the donor through an IV line in a vein. The part of white blood cells that contains stem cells is then separated in a machine before being returned to the donor.

A bone marrow transplant replaces bone marrow that either is not working properly or has been destroyed (ablated) by chemotherapy or radiation. Your doctor may recommend a bone marrow transplant if you have: Certain cancers, such as leukemia, lymphoma, and multiple myeloma A disease that affects the production of bone marrow cells, such as:
Aplastic anemia Congenital neutropenia Severe immunodeficiency syndromes Sickle cell anemia Thalassemia

Had chemotherapy that destroyed your bone marrow