A Two Step Approach to Reduced Intensity Allogeneic Hematopoietic Stem Cell Transplantation for High Risk Hematologic Malignancies
1. To compare the rate of disease-free survival (DFS) at 1 year post hematopoietic stem cell
transplant (HSCT) in patients undergoing HSCT treated on this successor Thomas Jefferson
University (TJU) 2 Step reduced intensity conditioning (RIC) haploidentical regimen and
compare it with that of the initial 2 Step RIC regimen.
1. To assess the 100 day regimen-related mortality (RRM) in patients undergoing HSCT on
this treatment protocol.
2. To determine the incidence and severity of graft-versus-host disease (GVHD) in patients
undergoing treatment on this regimen.
3. To evaluate engraftment rates and lymphoid reconstitution in patients treated on this
4. To assess overall survival at 1 and 3 years past HSCT in patients treated on this
REDUCED INTENSITY CONDITIONING: Patients receive fludarabine phosphate intravenously (IV)
over 60 minutes on days -15 to -12, thiotepa IV over 2 hours on days -15 to -13, donor
lymphocyte infusion (DLI) on day -6, and cyclophosphamide IV over 2 hours on days -3 and -2.
Patients also undergo total-body irradiation (TBI) on day -10.
TRANSPLANT: Patients undergo allogeneic peripheral blood stem cell transplant (PBSCT) on day
GVHD PROPHYLAXIS: Patients receive tacrolimus IV on days -1 to 42 followed by taper and
mycophenolate mofetil IV twice daily (BID) on days -1 to 28.
After completion of study treatment, patients are followed up periodically.
Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Disease-free survival (DFS)
The primary null hypothesis is that 1 year DFS rate is at most 35%. 35% is the rounded number (actual 36%) representing the DFS at 1 year of patients treated on the initial TJU 2 Step RIC HSCT trial and consistent with the outcome of patients treated on similar protocols outside of our institution.
Dolores Grosso, RN, CRNP, DNP
Thomas Jefferson University
United States: Food and Drug Administration
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