Hematopoietic stem cell transplantation using single UM171-expanded cord blood: a single-arm, phase 1–2 safety and feasibility study

Publication date: Available online 6 November 2019Source: The Lancet HaematologyAuthor(s): Sandra Cohen, Jean Roy, Silvy Lachance, Jean-Sébastien Delisle, Anne Marinier, Lambert Busque, Denis-Claude Roy, Frédéric Barabé, Imran Ahmad, Nadia Bambace, Léa Bernard, Thomas Kiss, Philippe Bouchard, Pierre Caudrelier, Sévérine Landais, Fannie Larochelle, Jalila Chagraoui, Bernhard Lehnertz, Sophie Corneau, Elisa TomelliniSummaryBackgroundBenefits of cord blood transplantation include low rates of relapse and chronic graft-versus-host disease (GVHD). However, the use of cord blood is rapidly declining because of the high incidence of infections, severe acute GVHD, and transplant-related mortality. UM171, a haematopoietic stem cell self-renewal agonist, has been shown to expand cord blood stem cells and enhance multilineage blood cell reconstitution in mice. We aimed to investigate the safety and feasibility of single UM171-expanded cord blood transplantation in patients with haematological malignancies who do not have a suitable HLA-matched donor.MethodsThis single-arm, open-label, phase 1–2 safety and feasibility study was done at two hospitals in Canada. The study had two parts. In part 1, patients received two cord blood units (one expanded with UM171 and one unmanipulated cord blood) until UM171-expanded cord blood demonstrated engraftment. Once engraftment was documented we initiated part 2, reported here, in which patients received a single UM171-expanded cord blood un...
Source: The Lancet Haematology - Category: Hematology Source Type: research