A Two Step Approach to Haploidentical Hematopoietic Stem Cell Transplantation for Patients in Remission From HLA Partially-Matched Related Donors-Effect of Maternal Donors on Outcomes
I. Examine the 1 year disease free survival (DFS) rate of patients with maternal donors or
sibling donors who share the maternal haplotype (maternal group) and compare them to
patients receiving cells from donors who have points from other characteristics such as
killer immunoglobulin-like receptor (KIR) ligand mismatching, minor histocompatibility
antigen (MHag) differences, or number of human leukocyte antigen (HLA) mismatches
I. Assess the incidences of relapse and graft-versus-host disease (GVHD) in maternal
recipients whose only eligible donors are offspring.
II. Assess the incidence of grades III-IV GVHD in female recipients with male donors.
III. Compare the rates of DFS in recipient-donor combinations in which there is at least 1
KIR ligand mismatch versus those without a KIR ligand mismatch.
Patients undergo total body irradiation (TBI) twice daily (BID) on days -9 to -6, undergo
donor lymphocyte infusion (DLI) on day -6, and receive cyclophosphamide intravenously (IV)
over 2 hours on days -3 and -2.
TRANSPLANT: Patients undergo haploidentical allogeneic hematopoietic stem cell transplant on
GVHD PROPHYLAXIS: Patients receive tacrolimus IV beginning on day -1 with taper beginning on
day 42, and mycophenolate mofetil IV BID from day -1 to day 28.
After completion of study treatment, patients are followed up at 90, 180, and 270 days, and
Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Disease-free survival (DFS)
Defined as the time to death, relapse or disease progression. The difference in DFS between maternal and non-maternal groups will be tested using log-rank test. The survival curves for each group will be estimated using the Kaplan-Meier estimator.
Dolores Grosso, DNP, CRNP
Thomas Jefferson University
United States: Institutional Review Board
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