| 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.
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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. |
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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.
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