Among patients with hematologic malignancies, transplantation with omidubicel may yield faster platelet recovery, as well as reduce the risk of infection, compared with standard myeloablative umbilical cord blood transplantation (UCBT), according to research published in Blood. No survival difference was noted, however.
UCB has been essential for allogeneic hematopoietic stem cell transplantation over the past several decades. Yet compared with transplants derived from adult donors, UCBT is linked with greater risk of morbidity and mortality, as well as slower hematopoietic recovery.
Omidubicel, a cell product derived from a banked UCB unit that is individualized to patients, has previously shown promise as an alternative to UCBT among patients in preclinical and clinical study. For this randomized phase 3 trial (ClinicalTrials.gov Identifier: NCT02730299), researchers compared the safety and efficacy of omidubicel to that of UCBT in a cohort of patients with hematologic conditions.
Overall, 125 patients were randomly assigned to the omidubicel (62 patients) or the UCBT (63 patients) group. In the experimental and control arms, 48% vs 36% of patients were female, respectively, the median age was 40 vs 43 years, and 43% vs 52% of patients had a primary diagnosis of acute myeloid leukemia.
In the omidubicel arm, the median time to neutrophil engraftment was 12 days (95% CI, 10-14) vs 22 days in the UCBT arm (95% CI, 19-25; P <.001); the cumulative incidence of neutrophil engraftment was 96% vs 89%, respectively.
Patients in the experimental arm had both faster platelet recovery at 42 days (55% vs 35% in the UCBT arm; P =.028) and a lower risk of first grade 2-3 bacterial or invasive fungal infection (37% vs 57%, respectively; P =.027). Patients assigned to the omidubicel arm also spent fewer days out of hospital out of the initial 100 days post-transplantation (median, 61 days vs 48 in the control arm; P =.005).
There was, however, no difference noted in the rate of graft vs host disease or in survival between the 2 arms.
“The results of this trial demonstrate that omidubicel represents a major therapeutic advance and should be considered as a new standard of care for adult patients eligible for UCBT,” the authors wrote.
Disclosure: The study author(s) declared affiliations with biotech, pharmaceutical, and/or device companies. Please see the original reference for a full list of authors’ disclosures.
Horwitz ME, Stiff PJ, Cutler C, et al. Omidubicel vs standard myeloablative umbilical cord blood transplantation: results of a phase 3 randomized study. Blood. 2021;138(16):1429-1440. doi:10.1182/blood.2021011719