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Chapter 9: Matched Sibling Donor HSCT 183 transplant physician’s recommendation; and (4) the parental or adult patient’s decision. The decision-making process in the timing of transplantation is difficult and must include multiple factors: • The vast majority of patients will progress to aplastic anemia and/or MDS/AML without transplant. • Transplants for FA using matched sibling donors have a very good chance of success, at 85-90% in FA-specialized transplant centers; • However, transplants are associated with a risk of peritransplant mortality of 10-15% and a risk of chronic GvHD for a “minimum” of 12% (with unmodified transplants); • In general, results of transplants are better for patients with aplastic anemia than with MDS/ AML; • Results of transplants are generally better for patients who are younger, partly due to a lower risk of GvHD; and • The patient’s overall vital organ status, such as renal or hepatic function, influences the transplant outcome. In addition to these factors, the following are relative and absolute indications for transplantation of FA patients from matched sibling donors based on patients’ hematologic status and age: Absolute indications • Severe aplastic anemia and transfusion dependence. In this case, no trial of androgens prior to proceeding to BMT.
184 <strong>Fanconi</strong> <strong>Anemia</strong>: Guidelines for Diagnosis and Management • High-risk myelodysplastic syndrome; i.e., refractory anemia with high-risk chromosomal abnormalities (involving chromosomes 3 or 7) or marrow blast count >5%. • Acute myelogenous leukemia. Relative indications • Moderate isolated cytopenias or moderate aplastic anemia with evidence of progression towards transfusion dependence. • Low-risk myelodysplastic syndrome; i.e., refractory anemia with no chromosomal abnormalities or low-risk chromosomal abnormalities. Definitions of the Transplant Indications Significant cytopenia Platelet count
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Chapter 4 Gastrointestinal, Hepatic
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Chapter 12 Novel Treatment Options
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Chapter 13 Head and Neck Squamous C
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Chapter 14 The Adult Fanconi Anemia
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guidebook. - Fanconi Anemia Research Fund

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