Treatment for Fanconi anemia includes the following
Androgens:
Approximately half of FA patients respond well to androgens (male hormones), which stimulate the production of red blood cells, and often, platelets. Sometimes white cell production is stimulated as well. This treatment may be effective for many years, but most patients eventually fail to respond. It is essential that the use of androgens is considered in the context of an eventual bone marrow transplant, as their use may affect adversely the ultimate success of a transplant.
Growth factors:
Hematopoietic (blood-stimulating) growth factors are also used. G-CSF stimulates the production of white blood cells and seems to be effective in FA patients. Other growth factors may be effective in combination.
Bone marrow transplantation:
At the present time, this is the only long-term cure for the blood defects in FA. This treatment has many risks associated with it, and the risks are compounded in FA patients because of their extreme sensitivity to radiation and chemotherapy. The consensus of the physicians who participated in March 2003 in the development of the handbook
Fanconi Anemia: Standards for Clinical Care is that, if a transplant center has had experience with fewer than 5 matched sibling donor transplants for FA, strong consideration should be given for referral to a transplant center with significant experience in transplants for FA. FA patients often experience complications which are not routine for other transplants, such as a marked increased risk in organ toxicity and in graft-versus-host disease (GVHD) and development of glucose intolerance, with most FA patients requiring insulin therapy.
Anemia treatment depends on the cause:
- Iron deficiency anemia. This form of anemia is treated with iron supplements, which you may need to take for several months or longer. If the underlying cause of iron deficiency is loss of blood - other than from menstruation - the source of the bleeding must be located and stopped. This may involve surgery.
- Vitamin deficiency anemias. Pernicious anemia is treated with injections - often lifetime injections - of vitamin B-12. Folic acid deficiency anemia is treated with folic acid supplements.
- Anemia of chronic disease. There's no specific treatment for this type of anemia. Doctors focus on treating the underlying disease. Iron supplements and vitamins generally don't help this type of anemia. However, if symptoms become severe, a blood transfusion or injections of synthetic erythropoietin, a hormone normally produced by the kidneys, may help stimulate red blood cell production and ease fatigue.
- Aplastic anemia. Treatment for this serious anemia may include blood transfusions to boost levels of red blood cells. You may need a bone marrow transplant if your bone marrow is diseased and can't make healthy blood cells. You may need immune-suppressing medications to lessen your immune system's response and give the transplanted bone marrow a chance to start functioning again.
- Anemias associated with bone marrow disease. Treatment of these various diseases can range from simple medication to chemotherapy to bone marrow transplantation. Treatment of these types of anemia usually involves a consultation from a blood specialist (hematologist).
- Hemolytic anemias. Managing hemolytic anemias includes avoiding suspect medications, treating related infections and taking drugs that suppress your immune system, which may be attacking your red blood cells. Short courses of treatment with steroids or gamma globulin can help suppress your immune system's attack on your red blood cells. If the condition has caused an enlarged spleen, you may need to have your spleen removed. The spleen - a small organ below your rib cage on the left side - filters out and stores defective red blood cells. Certain hemolytic anemias can cause the spleen to become enlarged with damaged red blood cells.
- Sickle cell anemia. Treatment for this incurable anemia may include the administration of oxygen, pain-relieving drugs, and oral and intravenous fluids to reduce pain and prevent complications. Doctors also commonly use blood transfusions, folic acid supplements and antibiotics. A bone marrow transplant may be an effective treatment in some circumstances. A cancer drug called hydroxyurea (Droxia, Hydrea) also is used to treat sickle cell anemia in adults.
