CT-314 Multicenter Long-Term Follow Up of Allogeneic Hematopoietic Stem Cell Transplantation With Omidubicel: A Pooled Analysis of Five Prospective Clinical Trials

Chenyu Lin, Aurelie Schwarzbach, Pau Montesinos, Patrick Stiff, Corey Cutler, Suhag Parikh, Claudio Brunstein, Caroline A. Lindemans, Rabi Hanna, Liang Piu Koh, Richard T. Maziarz, Amy K. Keating, William Y.K. Huang, Andrew R. Rezvani, David Valcarcel, Juliana F. Fernandes, Isabell S. Badell, Madan H. Jagasia, Olga Frankfurt, Ron RamJoseph P. McGuirk, Joanne Kurtzberg, Guillermo Sanz, Ronit Simantov, Mitchell E. Horwitz

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Context: Allogeneic hematopoietic stem cell transplantation (allo-HCT) with umbilical cord blood (UCB) is limited by the low number of hematopoietic stem cells. Omidubicel is an ex-vivo expanded stem cell product derived from UCB. Prospective clinical trials have demonstrated faster engraftment and fewer infections with omidubicel compared to UCB, but long-term outcomes are unknown. Objective: This is a pre-specified pooled secondary analysis of long-term outcomes with allo-HCT using omidubicel from 5 multicenter prospective trials. Design: Patients transplanted with omidubicel in one randomized phase III and four single-arm trials between 2006 and 2020 were followed for up to 10 years post-transplant. Setting: Twenty-six international academic transplant centers. Patients: Among 116 patients transplanted with either standalone omidubicel (n=92) or omidubicel with supplementary UCB (n=24), 97 (83.6%) engrafted with omidubicel, 11 (9.5%) with UCB, 2 mixed chimerism, 1 unevaluable, and 5 (4.3%) primary graft failure. This study included all patients aside from those engrafted with UCB (n=105). Median age was 42 (range, 2–62), 52.4% male, 30.5% non-white, and 30.5% high/very-high disease risk indices. Most common diseases were AML (40.1%), ALL (26.7%), MDS (13.3%), and sickle cell disease (7.6%). Results: Median follow-up was 22.0 months (range, 0.3–122.5). The 3-year OS and DFS were 62.5% (95%CI, 53.4–73.2) and 56.2% (95%CI, 47.0–67.1), respectively. Common causes of death included disease relapse (n=16) and infection (n=11). Three-year cumulative incidences of chronic GVHD and relapse were 37.8% (95%CI, 27.9–47.6) and 24.3% (95%CI, 16.1–33.3), respectively. Durable trilineage hematopoiesis was observed for up to 10 years. Similarly, median numbers of lymphoid subsets, including CD3+, CD4+, CD8+ T cells, CD19+ B cells, and CD16+/CD56+ NK cells were maintained within normal range for up to 8 years. Secondary graft failure was noted in 5 patients, 3 of whom underwent a second allo-HCT. Secondary hematologic malignancies included donor-derived myeloid neoplasm (dd-MN) (n=1; 40 months post-transplant) and PTLD (n=2; 17 and 20 months), with one death attributed to PTLD. Notably, there was also one case of dd-MN in the control UCB group of the phase III study. Conclusions: Omidubicel demonstrated durable long-term hematopoiesis and immune competence. One case of dd-MN was observed with both omidubicel and control UCB.

Original languageEnglish
Pages (from-to)S442
JournalClinical Lymphoma, Myeloma and Leukemia
Publication statusPublished - Oct 2022
Externally publishedYes


  • cellular therapy
  • CT
  • leukemia
  • long-term outcomes
  • lymphoma
  • stem cell transplantation


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