A Two Step Approach to Reduced Intensity Allogeneic Hematopoietic Stem Cell Transplantation for Patients With Hematologic Malignancies
I. To compare the overall survival (OS) rate at 2 years post treatment using the Jefferson 2
step reduced intensity conditioning (RIC) approach in patients with haploidentical family
donors with hematological malignancies in morphological or radiographic remission or with
chemosensitive, indolent diseases to historical OS rates in similar populations after RIC
matched donor HSCT as reported in the literature.
I. To compare the treatment-related mortality (TRM) rate at 2 years for patients treated on
this study to the historical TRM rates of patients undergoing RIC matched-sibling HSCT as
reported in the literature.
II. To compare the 2 year relapse rates and relapse related mortality of patients with
myeloid diseases to that of patients with lymphoid diseases who are treated on this Thomas
Jefferson University (TJU) RIC 2 step approach.
III. To determine the incidence and severity of graft-versus-host disease (GVHD) in patients
undergoing treated on the TJU RIC 2 step approach.
IV. To evaluate engraftment rates and lymphoid reconstitution in patients treated on the TJU
RIC 2 step approach.
V. To evaluate the incidence of TRM at 100 days in patients treated on the TJU RIC 2 step
REDUCED INTENSITY CONDITIONING: Patients receive fludarabine phosphate intravenously (IV)
over 60 minutes on days -11 to -8 and thiotepa IV over 2 hours on days -11 to -9. Patients
undergo total body irradiation (TBI) on day -6. Patients also receive cyclophosphamide IV
over 2 hours on days -3 and -2.
TRANSPLANTATION: Patients undergo donor lymphocyte infusion (DLI) on day -6 and cluster of
differentiation (CD)-34+ allogeneic peripheral blood stem cell transplantation (PBSCT) on
GVHD PROPHYLAXIS: Beginning on day -1, patients receive tacrolimus IV or orally (PO) with
taper beginning on day 42. Patients also receive mycophenolate mofetil IV twice daily (BID)
on days -1 to 28.
After completion of study treatment, patients are followed up periodically for 2 years.
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Overall Survival (OS) in patients with haploidentical family donors with hematological malignancies in morphological or radiographic remission or with chemosensitive, indolent diseases
The primary null hypothesis is that 2 year OS rate is at most 35%. This hypothesis will be rejected if the 95% confidence interval for year OS rate computed from the estimated Kaplan-Meier survival curves will be entirely above 0.35.
At 2 years
Dolores Grosso, DNP, CRNP
Thomas Jefferson University
United States: Food and Drug Administration
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