HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy
Start Date
4/1/2014
Completion Date
12/1/2028
Summary
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.
Detailed Description
The trial proposed is a three arm phase II treatment protocol designed to investigate the safety and efficacy of risk-adjusted chemotherapy-based cytoreductive regimen plus a CD34+ selected T-cell depleted peripheral blood stem cell (PBSC) stem cell transplant for the treatment of patients with Fanconi anemia and severe hematologic disease. Candidates for this trial will include patients with Fanconi anemia presenting with severe marrow failure (transfusion dependent) or myelodysplastic syndrome, or acute myelogenous leukemia for whom an allogeneic stem cell transplant is indicated.
Eligibility Criteria
Age Range: No minimum to No maximum
Interventions
Busulfan
Cyclophosphamide
Fludarabine
rabbit ATG
G-CSF
Peripheral blood stem cell
Conditions
Locations
Memorial Sloan Kettering Cancer Center
New York, New York 10174
United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio 45229
United States
Fred Hutchinson Cancer Research Center
Seattle, Washington 98109
United States