Omidubicel vs standard myeloablative umbilical cord blood transplantation: results of a phase 3 randomized study
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- Mitchell E. Horwitz
- Duke University Medical Center, Durham, NC;
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- Patrick J. Stiff
- Loyola University Medical Center, Chicago, IL;
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- Corey Cutler
- Dana-Farber Cancer Institute, Boston MA;
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- Claudio Brunstein
- University of Minnesota, Minneapolis, MN;
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- Rabi Hanna
- Taussig Cancer Institute, Department of Hematology & Medical Oncology, Cleveland Clinic, Cleveland, OH;
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- Richard T. Maziarz
- Oregon Health and Science University, Portland, OR;
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- Andrew R. Rezvani
- Stanford University Cancer Institute, Palo Alto, CA;
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- Nicole A. Karris
- City of Hope, Duarte, CA;
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- Joseph McGuirk
- University of Kansas Medical Center, Westwood, KS;
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- David Valcarcel
- Department of Hematology, University Hospital Vall d’Hebron, Barcelona, Spain;
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- Gary J. Schiller
- David Geffen School of Medicine at University of California Los Angeles, Los Angeles, CA;
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- Caroline A. Lindemans
- Princes Maxima Children’s Hospital and the Department of Pediatrics, University Medical Center, University of Utrecht, Utrecht, The Netherlands;
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- William Y. K. Hwang
- Singapore General Hospital, Singapore and National Cancer Centre of Singapore, Singapore;
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- Liang Piu Koh
- National University Cancer Institute, National University Health System Singapore, Singapore;
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- Amy Keating
- Childrens Hospital Colorado, Aurora, CO;
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- Yasser Khaled
- Methodist University Hospital, Memphis, TN;
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- Nelson Hamerschlak
- Israelita Albert Einstein Hospital, Sao Paolo, Brazil;
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- Olga Frankfurt
- Northwestern University, Chicago, IL;
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- Tony Peled
- Gamida Cell, Jerusalem, Israel;
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- Irit Segalovich
- Gamida Cell, Jerusalem, Israel;
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- Beth Blackwell
- Emmes Corporation, Rockville, MD;
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- Stephen Wease
- Emmes Corporation, Rockville, MD;
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- Laurence S. Freedman
- Gertner Institute for Epidemiology and Health Policy Research, Sheba Medical Center, Tel Hashomer, Israel; and
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- Einat Galamidi-Cohen
- Gamida Cell, Jerusalem, Israel;
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- Guillermo Sanz
- Hematology Department, Hospital Universitari i Politècnic La Fe, Valencia, Spain
抄録
<jats:title>Abstract</jats:title> <jats:p>Omidubicel is an ex vivo expanded hematopoietic progenitor cell and nonexpanded myeloid and lymphoid cell product derived from a single umbilical cord blood unit. We report results of a phase 3 trial to evaluate the efficacy of omidubicel compared with standard umbilical cord blood transplantation (UCBT). Between January 2017 and January 2020, 125 patients age 13 to 65 years with hematologic malignancies were randomly assigned to omidubicel vs standard UCBT. Patients received myeloablative conditioning and prophylaxis with a calcineurin inhibitor and mycophenolate mofetil for graft-versus-host disease (GVHD). The primary end point was time to neutrophil engraftment. The treatment arms were well balanced and racially diverse. Median time to neutrophil engraftment was 12 days (95% confidence interval [CI], 10-14 days) for the omidubicel arm and 22 days (95% CI, 19-25 days) for the control arm (P < .001). The cumulative incidence of neutrophil engraftment was 96% for patients receiving omidubicel and 89% for patients receiving control transplants. The omidubicel arm had faster platelet recovery (55% vs 35% recovery by 42 days; P = .028), had a lower incidence of first grade 2 to 3 bacterial or invasive fungal infection (37% vs 57%; P = .027), and spent more time out of hospital during the first 100 days after transplant (median, 61 vs 48 days; P = .005) than controls. Differences in GVHD and survival between the 2 arms were not statistically significant. Transplantation with omidubicel results in faster hematopoietic recovery and reduces early transplant-related complications compared with standard UCBT. The results suggest that omidubicel may be considered as a new standard of care for adult patients eligible for UCBT. The trial was registered at www.clinicaltrials.gov as #NCT02730299.</jats:p>
収録刊行物
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- Blood
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Blood 138 (16), 1429-1440, 2021-10-21
American Society of Hematology