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:
Purpose: 

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.

Status: 
Recruiting
Study Date: 
Tue, 04/01/2014 to Wed, 05/01/2019
Bone Marrow Disease(s): 
myelodysplastic syndromes (MDS)
Intervention: 
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).