HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy

"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."

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.

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.

Arms A, B and C - 4 doses will be given prior to transplant to promote engraftment.

All patients will also receive G-CSF post-transplant to foster engraftment.

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).