Alternative Donor Transplantation: Results of Parallel Phase II Trials Using HLA-Mismatched Related Bone Marrow or Unrelated Umbilical Cord Blood Grafts

Publication Type
Journal Article
Year of Publication
Brunstein, CG; Fuchs, EJ; Carter, SL; Karanes C; Costa, LJ; Wu, J; Devine, SM; Wingard, JR; Aljitawi, OS; Cutler, CS; Jagasia, MH; Ballen, KK; Eapen, M; O’Donnell, PV; Blood; Marrow Transplant Clinical Trials Network
Start Page
Date Published
Bone Marrow Transplantation/immunology; Child; Family; Female; Fetal Blood/immunology; Fetal Blood/transplantation; Graft Survival; Hematologic Neoplasms/immunology; Histocompatability Testing/Methods; HLA Antigens/analysis; HLA Antigens/immunology
The Blood and Marrow Transplant Clinical Trials Network conducted 2 parallel multicenter phase 2 trials for individuals with leukemia or lymphoma and no suitable related donor. Reduced intensity conditioning (RIC) was used with either unrelated double umbilical cord blood (dUCB) or HLA-haploidentical related donor bone marrow (Haplo-marrow) transplantation. For both trials, the transplantation conditioning regimen incorporated cyclophosphamide, fludarabine, and 200 cGy of total body irradiation. The 1-year probabilities of overall and progression-free survival were 54% and 46%, respectively, after dUCB transplantation (n = 50) and 62% and 48%, respectively, after Haplo-marrow transplantation (n = 50). The day +56 cumulative incidence of neutrophil recovery was 94% after dUCB and 96% after Haplo-marrow transplantation. The 100-day cumulative incidence of grade II-IV acute GVHD was 40% after dUCB and 32% after Haplo-marrow transplantation. The 1-year cumulative incidences of nonrelapse mortality and relapse after dUCB transplantation were 24% and 31%, respectively, with corresponding results of 7% and 45%, respectively, after Haplo-marrow transplantation. These multicenter studies confirm the utility of dUCB and Haplo-marrow as alternative donor sources and set the stage for a multicenter randomized clinical trial to assess the relative efficacy of these 2 strategies. The trials are registered at under NCT00864227 (BMT CTN 0604) and NCT00849147 (BMT CTN 0603).