| Thorough History and Assessment:
Since Fanconi anemia is an extremely rare disease, the
reader is urged to contact the FA specialists listed in Treatment
and Testing Resources for the most current protocols in the diagnosis and
treatment of Fanconi anemia. Refer also to Fanconi
Anemia: A Handbook for Families and Their Physicians and
Fanconi
Anemia: Standards for Clinical Care (2003 edition) for more information. In
addition, check our FA Family Newsletter
or contact our Family Support Coordinator
for the latest news and treatments for FA. |
 Transplant
physicians Wolfram Ebell, Charite Hospital, Berlin; Farid Boulad, Memorial Sloan-Kettering
Cancer Center; Ingrid Kuehnle, Baylor College of Medicine; Ricardo Pasquini, Federal
University of Parana, Curitiba, Brazil; John Wagner and Margaret MacMillan, University
of Minnesota School of Medicine; and Eliane Gluckman, Hospital St. Louis, Paris
at the 2003 FA Scientific Symposium. |
|
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. Information
provided on this page about medications, treatments or products should not be
construed as medical instruction or scientific endorsement. Always consult your
physician before taking any action based on this information. |
