Abstract

Omidubicel is an advanced cell therapy derived from umbilical cord blood (UCB) for use in allogeneic hematopoietic cell transplantation (HCT). A recent randomized phase 3 clinical trial demonstrated faster engraftment, shorter hospitalizations, and lower rates of infections with omidubicel when compared to standard UCB transplantation in patients with high-risk hematologic malignancies (Horwitz et al., Blood 2021). Despite the proven clinical benefits of omidubicel, its impact on health-related quality of life (HRQL) from the patient's perspective has not been described.This study reports on the analysis of patient-reported HRQL measures prospectively collected in the randomized phase 3 trial comparing omidubicel to standard UCB transplantation.A total of 108 patients at 33 international stem cell transplant centers received myeloablative allogeneic HCT with either omidubicel or standard UCB. Patients completed serial HRQL questionnaires at screening and on days 42, 100, 180, and 365 post-transplant. Administered HRQL surveys include the Functional Assessment of Cancer Therapy - Bone Marrow Transplant (FACT-BMT), a 37-item cancer-specific questionnaire assessing physical, functional, emotional, social/family, and HCT-specific well-being, and the EuroQol 5-Dimension 3-Level, a 5-item generic HRQL survey. A mixed model with repeated measures was used to compare HRQL changes from baseline between treatment arms. The average change in HRQL scores over time was compared by estimating the difference in the area under the curve (AUC) in each treatment group.Seventy-five patients (omidubicel n?=?37, standard UCB n?=?38) who completed the FACT-BMT at baseline and on =1 follow-up visit were included in this study. Baseline characteristics were similar between treatment arms. Over the first year post-transplant, the AUCs of mean change in physical, functional, and total FACT-BMT scores indicated significantly better HRQL with omidubicel (P < .05), with mean differences across time points ranging from 1.4-3.1, 1.6-3.2, and 7.2-11.0 points, respectively. The minimal clinically important difference was exceeded on at least 1 time point for each of these measures. The HRQL improvements from omidubicel were observed as early as 42 days post-transplant and persisted at 1 year, indicating the potential long-term benefits of omidubicel on HRQL. Across all patients, adverse clinical outcomes such as grade 3 viral infections and lower rates of neutrophil engraftment were associated with worse HRQL scores. The observed improvements in HRQL measures may reflect the known clinical benefits of omidubicel.Compared to standard UCB, allogeneic HCT with omidubicel resulted in significant and clinically meaningful improvements in patient-reported HRQL measures.

View details for DOI 10.1016/j.jtct.2022.09.018

View details for PubMedID 36179986