Clinical Trial Details

HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy (RAFA)

Complete Title: A Phase II Trial of Hematopoietic Stem Cell Transplantation for the Treatment of Patients with Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Risk-Adjusted Chemotherapy Only Cytoreduction with Busulfan, Cyclophosphamide and Fludarabine
Trial Phase: II
Investigator: K. Scott Baker

The purpose of this study is to determine whether the use of lower doses of busulfan and the elimination of cyclosporine will further reduce transplant-related side effects for patients with Fanconi Anemia (FA). Patients will undergo a transplant utilizing mis-matched related or matched unrelated donors following a preparative regimen of busulfan, fludarabine, anti-thymocyte globulin and cyclophosphamide.

Keywords:
  • Leukemia, Acute Myeloid (AML); Anemia, Aplastic; Fanconi Anemia; Hematologic Malignancies; Preleukemia; Bone Marrow Diseases; Leukemia; Pediatric Cancers, Miscellaneous; Myelodysplastic Syndromes (MDS); Leukemia, Myeloid; Neoplasms; Anemia
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