Non-Oncologic Blood Disorders Patient & Family Handbook

A Family Guide to Non-Oncologic Blood Disorders

How successful is the treatment? SCT currently is the only curative option for FA. Some doc- tors believe patients benefit from receiving SCT with the use of Fludarabine, a type of chemotherapy, as part of the SCT regimen. The severity of FA varies and requires long-term monitoring for cancer as well as other organ abnormalities. Stem Cell Transplant Indications SCT from a human leukocyte antigen-identical sibling who does not have FA or a matched unrelated donor would be indicated in your child if they have moderate to severe bone marrow failure.

Treatment options include: • Stem cell transplantation (SCT)

• Androgen therapy—androgens are artificial male hor- mones that can help your child make more blood cells for longer periods. Androgens are effective at increasing red blood cell and platelet counts, but not the white blood cells. This option is for those without a matched sibling or transplant as an option. The standard recom- mended androgen is Oxymetholone. • Hematopoietic growth factors—medications that help stimulate and raise the neutrophil count • Supportive therapy—treatment with transfusions as indicated and/or antibiotics for infections

Notes from Your Child’s Medical Team

Fanconi Anemia

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