Umbilical cord blood (UCB) is a rich source of stem cells for transplantation in patients with hematologic malignancies. However, its widespread utilization has been limited by the generally slow rate of hematopoietic recovery. In a first-in-human pilot study of an ex-vivo expanded UCB product (NiCord) used in combination with nicotinamide to expand repopulating hematopoietic stem cells, stable NiCord-derived hematopoiesis was observed for more than 7 years. In this multicenter phase I/II single-arm study, 36 patients with high-risk hematologic malignancies and no readily available matched adult donor were transplanted with a single, expanded UCB graft (NiCord) after myeloablative conditioning. The primary outcomes were the incidence of neutrophil engraftment at 42 days and the incidence of secondary graft failure. Results were compared to data from a matched cohort of 146 patients who underwent UCB transplantation between 2010 and 2013 from the Center for International Blood and Marrow Transplant Research (CIBMTR). Researchers found that the age-adjusted cumulative incidence of neutrophil engraftment at 42 days after transplantation was higher in the NiCord recipients (94%) than in the CIBTMR comparator cohort (85%) (p<0.001). One NiCord recipient experienced primary graft failure, and two patients experienced secondary graft failure. The cumulative incidence of grade 2 to 4 graft-versus-host disease (GVHD) at day 100 was 44% in the NiCord recipients (95% CI 28% to 60%), and the cumulative incidence of chronic GVHD at 3 years was 40% (95% CI 24% to 57%). Overall, this study suggests that use of a stand-alone ex-vivo expanded UCB graft may be efficacious and safe. An ongoing phase III trial comparing NiCord to standard myeloablative UCB transplantation will provide conclusive results.
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