A Study of Cyclophosphamide, Fludarabine, and Antithymocyte Globulin Followed by Matched Sibling Donor Hematopoietic Cell Transplantation in Patients With Fanconi Anemia
- To determine the probability of engraftment in patients with Fanconi anemia treated
with cyclophosphamide, fludarabine phosphate, and antithymocyte globulin followed by
HLA-genotypically identical sibling donor hematopoietic stem cell transplantation that
is T-cell depleted.
- To evaluate the incidence of acute graft-versus-host disease (GVHD) and chronic GVHD in
patients treated with this regimen.
- To evaluate the incidence of regimen-related toxicity in these patients.
- To evaluate the 1-year survival of patients treated with this regimen.
- To evaluate the incidence of late secondary malignancies (e.g., squamous cell carcinoma
of the head and neck or cervix) in patients treated with this regimen.
- Preparative cytoreductive therapy: Patients receive cyclophosphamide IV over 2 hours on
days -6 to -3 and fludarabine phosphate IV over 30 minutes and anti-thymocyte globulin
IV over 4-6 hours on days -6 to -2.
- T-cell depleted donor hematopoietic stem cell transplantation: Patients undergo T-cell
depleted donor bone marrow or umbilical cord blood stem cell transplantation on day 0.
Patients also receive filgrastim (G-CSF) IV beginning on day 1 and continuing until
blood counts recover.
- Graft-versus-host disease prophylaxis: Patients receive cyclosporine IV over 2 hours or
orally every 8-12 hours beginning on day -3 and continuing until day 100, followed by a
taper. Patients will receive Mycophenolate Mofetil (MMF) therapy beginning on day -3
through day +30 or for 7 days after engraftment, whichever day is later, if no acute
GVHD. Engraftment is defined as 1st day of 3 consecutive days of absolute neutrophil
count [ANC] > 0.5 x 10^9/L.
After completion of study therapy, patients are followed periodically.
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
graft failure = absolute neutrophil count (ANC) <5 x 10^8/L and an acellular bone marrow aspirate/biopsy
From Day 1 to event
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|