Clinical Genetics Branch
6120 Executive Blvd, MSC 7231
Rockville, MD20892-7231
Tel 301-594-7642
FAX 301-496-1854
June 17, 2007
To Whom It May Concern:
I would like to endorse the need for Ilya Mracek to have a bone marrow transplant for his bone marrow failure due to Fanconi’s Anemia (FA). This transplant needs to be performed at an institution that has extensive experience with transplantation particularly for this disease. FA is a genetic disease with a defect in DNA repair, and a high rate of aplastic anemia, from which Ilya suffers at this time. Transplant preparative and post-procedure regimens are constantly changing and improving, and the most recent FA protocols are not in the published literature, because they are too new. The toxicities and complications of transplant in FA are extensive, and require that the entire transplant team be experienced in the management of FA patients. This includes not only the senior physicians, but also the junior doctors (who receive the first call when things go wrong), all the nurses, and the technicians and everyone in a supporting role. Within the United States, there are only 3 major centers which perform the majority of the transplants for FA, despite more than 30 pediatric transplant centers. In Europe, there are only twomajor centers that are recognized for their expertise in FA transplants, namely Berlin and Paris.
Transplant in FA patients is a difficult and challenging procedure, and, at least in the USA, the families always want to be treated at a center that has expertise in FA, not just in transplant. This becomes even more important when the donor is not a sibling, but is unrelated, whether it is an adult or a cord blood. I therefore would like to most strongly support the need for this transplant to be performed in Berlin. Dr Ebell at ChariteHospital is an expert in FA transplants, and the child should be given the best chance for survival that can be provided.
Please note that these opinions are personal and do not reflect the policy/opinion of the NCI/NIH.
Yours sincerely,
Blanche P Alter, MD, MPH