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Fanconi Anemia and Bone Marrow Failure Multidisciplinary Clinic

 Fanconi Anemia and Bone Marrow Failure Multidisciplinary Clinic
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 Blood Disorders
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Treatment for bone marrow failure depends on the cause of the condition and the severity of the illness. Other factors include:
  • The child's age, overall health, and medical history
  • His or her tolerance for therapeutic medications, procedures, and therapies
  • Expectations for the course of the disorder
Treatment options may include:
  • Drug therapy- depending on the type and cause of marrow failure, medical therapy may include corticosteroids, androgens, and hematopoietic growth factors in inherited bone marrow failures, as well as agents like antithymocyte globulin and cyclosporin in acquired aplastic anemia. In some cases, the use of prophylactic antibiotics may be prescribed.

  • Hematopoietic stem cell transplant (HSCT) - this therapy can provide the only cure for some forms of marrow failure. HSCT, also called a bone marrow transplant, involves replacing diseased bone marrow with healthy marrow from another person (usually a close relative). HSCT is a very complex treatment, and not all children with bone marrow failure are candidates for this therapy.

    HSCT requires a genetically-matched or closely matched donor and carries significant risks. If HSCT is a treatment option, we will coordinate evaluation and treatment with the experts from Dana-Farber/Children’s Hospital Cancer Care’s Pediatric Stem Cell Transplant Program.

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