Omidubicel vs standard myeloablative umbilical cord blood transplantation: results of a phase 3 randomized study

Abstract

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.

Optimizing Blood Stem Cell Transplants Through Cellular Engineering

“…The results showed that the omidubicel transplant recipients had a shorter median time to neutrophil engraftment of 12 days compared to 22 days for patients in the control group. Omidubicel recipients also had a higher cumulative incidence (CI) of neutrophil engraftment, as well as a faster platelet recovery, a lower incidence of bacterial and fungal infections, and they spent more time out of hospital during the first 100 days post-transplant as compared to the control group 11 .…”

“…NiCord recipients also had a higher incidence of platelet engraftment (81%) 100 days after HSCT, with faster platelet engraftment and recovery 10 . In another study conducted with omidubicel, formerly known as NiCord, 125 patients with hematologic malignancies received either omidubicel or standard umbilical cord blood transplantation (UCBT) 11 . The results showed that the omidubicel transplant recipients had a shorter median time to neutrophil engraftment of 12 days compared to 22 days for patients in the control group.…”

Strategies for Success With Umbilical Cord Haematopoietic Stem Cell Transplantation in Children With Malignant and Non-Malignant Disease Indications

“…Such expansion technologies have been mainly trialed however in malignant diseases. Any expansion must allow preservation of graft T-cells, to permit the graft versus leukaemia effect ( Horwitz et al, 2014 ; Lindemans et al, 2014 ; Cohen et al, 2020 ; Horwitz et al, 2021 ).…”

Hematopoiesis during Ontogenesis, Adult Life, and Aging

“…Recently published results of phase III trial using transplantation of standalone NAD-expanded units confirm earlier conclusions on significant reduction of time for neutrophil and platelet recovery compared to control, and reduced incidence of infections. No statistically significant differences with control were observed for recipient survival and graft-versus-host disease (GvHD) [221]. Current studies on ex vivo expansion of HSCs and the available results of clinical trials are described in more detail in a recent review by Zimran et al [222].…”