Multicenter Long-Term Follow-Up of Allogeneic Hematopoietic Cell Transplantation with Omidubicel: A Pooled Analysis of Five Prospective Clinical Trials

• Published in: Transplantation and cellular therapy 2023-05, Vol.29 (5), p.338.e1-338.e6
• Main Authors: Lin, Chenyu, Schwarzbach, Aurelie, Sanz, Jaime, Montesinos, Pau, Stiff, Patrick, Parikh, Suhag, Brunstein, Claudio, Cutler, Corey, Lindemans, Caroline A., Hanna, Rabi, Koh, Liang Piu, Jagasia, Madan H., Valcarcel, David, Maziarz, Richard T., Keating, Amy K., Hwang, William Y.K., Rezvani, Andrew R., Karras, Nicole A., Fernandes, Juliana F., Rocha, Vanderson, Badell, Isabel, Ram, Ron, Schiller, Gary J., Volodin, Leonid, Walters, Mark C., Hamerschlak, Nelson, Cilloni, Daniela, Frankfurt, Olga, McGuirk, Joseph P., Kurtzberg, Joanne, Sanz, Guillermo, Simantov, Ronit, Horwitz, Mitchell E.
• Format: Article
• Language: English
• Subjects: Allogeneic stem cell transplantation; Clinical trial; Cord blood; Disease-Free Survival; Ex vivo expansion; Follow-Up Studies; Hematopoietic Stem Cell Transplantation; Humans; Long-term follow-up; Multicenter Studies as Topic; Prospective Studies
• ISSN: 2666-6367; 2666-6375; 2666-6367
• DOI: 10.1016/j.jtct.2023.01.031

•Omidubicel is an ex vivo expanded stem cell product derived from umbilical cord blood (UCB).•We conducted a pooled analysis of long-term outcomes from 5 clinical trials evaluating omidubicel transplantation.•The 3-year overall survival and disease-free survival were 62.7% and 56.4%, respectively.•Durable long-term trilineage hematopoiesis and immune competence up to 8 years were noted.•One case of donor-derived myelodysplastic syndrome each was seen with omidubicel and standard UCB transplantation. 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. [Display omitted]