Hematopoietic Stem Cell Transplantation in High Risk Patients With Fanconi Anemia MT2002-02
- Determine whether the incidence of neutrophil engraftment is acceptable in high-risk
patients with Fanconi's anemia treated with busulfan, cyclophosphamide, fludarabine,
and antithymocyte globulin followed by allogeneic hematopoietic stem cell
- Determine the tolerability of mycophenolate mofetil in these patients.
- Determine the incidence of acute and chronic graft-vs-host disease in patients treated
with this regimen.
- Determine the incidence of major infections in patients with a history of major
infections treated with this regimen.
- Determine the incidence of relapse in patients with refractory anemia with excess
blasts, refractory anemia with excess blasts in transformation, or acute myeloid
leukemia treated with this regimen
- Determine the probability of 1-year survival of patients treated with this regimen.
OUTLINE: Patients are stratified according to donor/recipient HLA type (identical vs other).
- Cytoreductive combination chemotherapy: Patients receive busulfan intravenously (IV)
over 2 hours twice daily on days -7 and -6 and cyclophosphamide IV over 2 hours and
fludarabine IV over 30 minutes once daily on days -5 to -2.
- Graft failure prophylaxis: Patients receive methylprednisolone IV twice daily on days
-5 to 30 and anti-thymocyte globulin IV over 4-6 hours twice daily on days -5 to -1.
- Graft-vs-host disease prophylaxis: Patients receive cyclosporine IV over 2 hours twice
daily on days -3 to 100 (if patient has a matched sibling donor) or days -3 to 180 (if
patient has another donor type). Patients also receive mycophenolate mofetil orally or
IV twice daily on days -3 to 45.
- Allogeneic hematopoietic stem cell transplantation (HSCT): Patients undergo allogeneic
HSCT (using bone marrow or umbilical cord blood) on day 0. Patients receive filgrastim
(G-CSF) subcutaneously beginning on day 1 and continuing until blood counts recover.
After completion of study treatment, patients are followed periodically for 3 years.
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Percent of Graft Failure
Graft failure = ANC <5 x 10^8/L by day 30.
Margaret L. MacMillan, MD
Masonic Cancer Center, University of Minnesota
United States: Food and Drug Administration
|Masonic Cancer Center, University of Minnesota||Minneapolis, Minnesota 55455|