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
  • Leukemia
  • Pediatric Cancers, Miscellaneous
  • Myelodysplastic Syndromes (MDS)
  • Leukemia, Myeloid
  • Neoplasms
  • Anemia
  • Preleukemia
  • Bone Marrow Diseases
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Adult
Pediatric
II
K. Scott Baker
9213
NCT02143830
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
Leukemia, Acute Myeloid (AML)
Anemia, Aplastic
Fanconi Anemia
Hematologic Malignancies
Leukemia
Pediatric Cancers, Miscellaneous
Myelodysplastic Syndromes (MDS)
Leukemia, Myeloid
Neoplasms
Anemia
Preleukemia
Bone Marrow Diseases