Omidubicel Versus Standard Myeloablative Umbilical Cord Blood Transplantation: Results of a Phase III Randomized Study.
Omidubicel is an ex vivo expanded hematopoietic progenitor cell, and non expanded myeloid and lymphoid cell product derived from a single umbilical cord blood unit. We report results of a phase III trial to evaluate the efficacy of omidubicel compared to standard umbilical cord blood transplantation (UCBT) Between January 2017 and January 2020, 125 patients aged 13 65 with hematologic malignancies were randomized to omidubicel versus standard UCBT. Patients received myeloablative conditioning and graft versus host disease (GvHD) prophylaxis with a calcineurin inhibitor and mycophenolate mofetil. The primary endpoint was time to neutrophil engraftment. The treatment arms were well balanced and racially diverse. Median time to neutrophil engraftment was 12 days (95% CI 10 14 days) and 22 days (95% CI 19 25 days) (p<0.001) for the omidubicel and control arms, respectively. The cumulative incidence of neutrophil engraftment was 96% and 89% for patients receiving omidubicel and control transplants, respectively. The omidubicel arm had faster platelet recovery (55% vs. 35% recovery by 42 days, p=0.028) a lower incidence of first grade 2/3 bacterial or invasive fungal infections (37% vs. 57% p=0.027) and spent more time out of hospital during the first 100 days following transplant (median 61 vs. 48 days, p=0.005) than controls. Differences in GvHD and survival between the two arms were not statistically significant. Transplantation with omidubicel results in faster hematopoietic recovery and reduced early transplant related complications as compared to standard UCBT. The results suggest that omidubicel may be considered as a new standard of care for adult patients eligible for UCBT.
Blood