HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy (RAFA) | Aplastic Anemia and MDS International Foundation

Clinical Trial: NCT02143830

HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy (RAFA)
For more details on this clinical trial, including contact information, please see this trial’s listing on clinicaltrials.gov:

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.

Study Date: 
Mon, 04/28/2014 to Wed, 05/01/2019
Bone Marrow Disease(s): 
aplastic anemia
myelodysplastic syndromes (MDS)
Drug: Busulfan A standard dose of busulfan, associated with excellent outcomes in our previous trial will be used for young patients with marrow aplasia (arm A). A higher dose of busulfan will be used in younger patients with MDS and AML (arm B) to maximize disease control. A lower dose of busulfan will be used in older patients (arm C) to minimize toxicity. Other Names: Myleran Busulfex IV Drug: Cyclophosphamide Arms A, B and C - Cytoxan will be given as a 1-2 hour infusion for 4 days. The dose will be adjusted according to patients ideal body weight for obese patients. Other Name: Cytoxan Drug: Fludarabine Arms A, B and C - Fludarabine will be given IV over 30 minutes daily for 4 days. The dose will be adjusted according to renal function according to Institutional guidelines. Other Name: Fludara Drug: rabbit ATG Arms A, B and C - 4 doses will be given prior to transplant to promote engraftment. Other Name: thymoglobulin Drug: G-CSF All patients will also receive G-CSF post-transplant to foster engraftment. Other Names: Granulocyte colony-stimulating factor filgrastim neupogen Biological: Peripheral blood stem cell The source of stem cells for all patients will be peripheral blood stem cells (PBSC) induced and mobilized by treatment of the donor with G-CSF for 4-6 days. T-cell depletion will be uniformly performed by positive CD34 selection with the use of the Miltenyi system (CliniMACS device).