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. 2023 Oct 10;7(19):5717-5726.
doi: 10.1182/bloodadvances.2023010599.

Improved outcomes of UM171-expanded cord blood transplantation compared with other graft sources: real-world evidence

Sandra Cohen  1   2 , Nadia Bambace  1   2 , Imran Ahmad  1   2 , Jean Roy  1   2 , Xiaoying Tang  3 , Mei-Jie Zhang  3 , Linda Burns  3 , Frédéric Barabé  4 , Léa Bernard  1   2 , Jean-Sébastien Delisle  1   2 , Thomas Kiss  1   2 , Silvy Lachance  1   2 , Denis-Claude Roy  1   2 , Olivier Veilleux  1   2 , Guy Sauvageau  1   2   5
Affiliations

Improved outcomes of UM171-expanded cord blood transplantation compared with other graft sources: real-world evidence

Sandra Cohen et al. Blood Adv. .

Abstract

Cord blood (CB) transplantation is hampered by low cell dose and high nonrelapse mortality (NRM). A phase 1-2 trial of UM171-expanded CB transplants demonstrated safety and favorable preliminary efficacy. The aim of the current analysis was to retrospectively compare results of the phase 1-2 trial with those after unmanipulated CB and matched-unrelated donor (MUD) transplants. Data from recipients of CB and MUD transplants were obtained from the Center for International Blood and Marrow Transplant Research (CIBMTR) database. Patients were directly matched for the number of previous allogeneic hematopoietic stem cell transplants (alloHCT), disease and refined Disease Risk Index. Patients were further matched by propensity score for age, comorbidity index, and performance status. Primary end points included NRM, progression-free survival (PFS), overall survival (OS), and graft-versus-host disease (GVHD)-free relapse-free survival (GRFS) at 1 and 2 years after alloHCT. Overall, 137 patients from CIBMTR (67 CB, 70 MUD) and 22 with UM171-expanded CB were included. NRM at 1 and 2 years was lower, PFS and GRFS at 2 years and OS at 1 year were improved for UM171-expanded CBs compared with CB controls. Compared with MUD controls, UM171 recipients had lower 1- and 2-year NRM, higher 2-year PFS, and higher 1- and 2-year GRFS. Furthermore, UM171-expanded CB recipients experienced less grades 3-4 acute GVHD and chronic GVHD compared with MUD graft recipients. Compared with real-world evidence with CB and MUD alloHCT, this study suggests that UM171-expanded CB recipients may benefit from lower NRM and higher GRFS. This trial was registered at www.clinicaltrials.gov as #NCT02668315.

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Conflict of interest statement

Conflict-of-interest disclosures: S.C. has received consulting fees from ExCellThera. S.C., J.R., S.L., and G.S. are eligible to receive royalties if UM171 is commercialized. G.S. is a major stockholder and chief executive/scientific officer of ExCellThera. S.C. and J.R. have received funding for clinical trials from ExCellThera. ExCellThera holds the license for UM171. L.J.B., M.-J.Z., and X.T. have collaborated on research with Gamida Cell, Inc. L. Burns has also collaborated on research with Astellas, bluebird bio, Gamida Cell, Kyowa Kirin, Mesoblast, Sanofi, and Vertex. The remaining authors declare no competing financial interests.

Figures

None
Graphical abstract
Figure 1.
Figure 1.
Engraftment. (A-B) Cumulative incidences of neutrophil (A) and platelet (B) engraftment of the UM171 CB cohort (UM171) and the CIBMTR CB controls (UCB). Neutrophil and platelet engraftment are improved in the UM171 CB cohort by Gray test. (C-D) Cumulative incidences of neutrophil (A) and platelet (B) engraftment of the UM171 CB cohort and the CIBMTR MUD PBSC controls (PBSC). Platelet engraftment is delayed for UM171 CB compared with MUD PBSC by Gray test.
Figure 2.
Figure 2.
Outcomes of UM171 CB Cohort and CIBMTR CB Controls. (A-B) Kaplan-Meier estimates of OS (A) and progression-free survival (B). (C) GRFS by Kaplan-Meier estimate of the UM171 CB cohort (UM171) appears superior to that of the CIBMTR CB controls (UCB) when compared with log-rank test. (D) Cumulative incidence of NRM of the UM171 CB cohort is lower than that of the CIBMTR CB controls when compared by Gray test.
Figure 3.
Figure 3.
GVHD. (A-B) Cumulative incidences of grade 3-4 acute GVHD (A) and chronic GVHD (B) of the UM171 CB cohort (UM171) and the CIBMTR CB controls (UCB). (C-D) Cumulative incidences of grade 3-4 acute GVHD (C) and chronic GVHD (D) of the UM171 CB cohort appear lower than those reported for the CIBMTR MUD PBSC controls (PBSC) by Gray test.
Figure 4.
Figure 4.
Outcomes of UM171 CB Cohort and CIBMTR MUD PBSC controls. (A) Kaplan-Meier estimates of OS in the UM171 CB cohort (UM171) and the CIBMTR MUD PBSC controls (PBSC) compared by log-rank test. (B) PFS appears superior by Kaplan-Meier estimate for the UM171 CB cohort compared with that of the CIBMTR MUD PBSC controls by log-rank test. (C) GRFS by Kaplan-Meier estimate appears superior for the UM171 CB cohort compared with that of the CIBMTR MUD PBSC controls by log-rank test. (D) Cumulative incidence of NRM of the UM171 CB and MUD PBSC cohorts.
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