Multicenter Long-Term Follow-Up of Allogeneic Hematopoietic Cell Transplantation with Omidubicel: A Pooled Analysis of Five Prospective Clinical Trials
Chenyu Lin, Aurélie Schwarzbach, Jaime Sanz, Pau Montesinos, Patrick J. Stiff, Suhag Parikh, Claudio G. Brunstein, Corey Cutler, Caroline A. Lindemans, Rabi Hanna, Liang Piu Koh, Madan Jagasia, David Valcárcel, Richard T. Maziarz, Amy K. Keating, William Ying Khee Hwang, Andrew R. Rezvani, Nicole Karras, Juliana Folloni Fernandes, Vanderson Rocha, Isabel Badell, Ron Ram, Gary J. Schiller, Leonid Volodin, Mark C. Walters, Nelson Hamerschlak, Daniela Cilloni, Olga Frankfurt, Joseph P. McGuirk, Joanne Kurtzberg, Guillermo Sanz, Ronit Simantov, Mitchell E. Horwitz
Abstract
Omidubicel is an umbilical cord blood (UCB)-derived ex vivo-expanded cellular therapy product that has demonstrated faster engraftment and fewer infections compared with unmanipulated UCB in allogeneic hematopoietic cell transplantation. Although the early benefits of omidubicel have been established, long-term outcomes remain unknown. We report on a planned pooled analysis of 5 multicenter clinical trials including 105 patients with hematologic malignancies or sickle cell hemoglobinopathy who underwent omidubicel transplantation at 26 academic transplantation centers worldwide. With a median follow-up of 22 months (range, .3 to 122 months), the 3-year estimated overall survival and disease-free survival were 62.7% and 56.4%, respectively. With up to 10 years of follow-up, omidubicel showed durable trilineage hematopoiesis. Serial quantitative assessments of CD3+, CD4+, CD8+, CD19+, CD116+CD56+, and CD123+ immune subsets revealed median counts remaining within normal ranges through up to 8 years of follow-up. Secondary graft failure occurred in 5 patients (5%) in the first year, with no late cases reported. One case of donor-derived myeloid neoplasm was reported at 40 months post-transplantation. This was also observed in a control arm patient who received only unmanipulated UCB. Overall, omidubicel demonstrated stable trilineage hematopoiesis, immune competence, and graft durability in extended follow-up.