Different Forms of Treatment
(Information used with the kind permission of FARF - www.fanconi.org)
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:
Haematopoietic (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 Doctors who participated in March 2003 in the development of the handbook Fanconi Anemia: Standards for Clinical Care (in corroboration with FARF) is that, if a transplant centre has had experience with fewer than 5 matched sibling donor transplants for FA, strong consideration should be given for referral to a transplant centre 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.
Information provided on this page about medications, treatments or products should not be construed as medical instruction or scientific endorsement. Always consult your Specialist / Medical Professional before taking any action based on this information.
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:
Haematopoietic (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 Doctors who participated in March 2003 in the development of the handbook Fanconi Anemia: Standards for Clinical Care (in corroboration with FARF) is that, if a transplant centre has had experience with fewer than 5 matched sibling donor transplants for FA, strong consideration should be given for referral to a transplant centre 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.
Information provided on this page about medications, treatments or products should not be construed as medical instruction or scientific endorsement. Always consult your Specialist / Medical Professional before taking any action based on this information.
