John E Wagner1, Mary Eapen, Shelly Carter, Yanli Wang, Kirk R Schultz, Donna A Wall, Nancy Bunin, Colleen Delaney, Paul Haut, David Margolis, Edward Peres, Michael R Verneris, Mark Walters, Mary M Horowitz, Joanne Kurtzberg. 1. From the University of Minnesota Medical School, Minneapolis (J.E.W., M.R.V.); Medical College of Wisconsin, Milwaukee (M.E., D.M., M.M.H.); EMMES Corporation, Rockville, MD (S.C., Y.W.); BC Children's Hospital, Vancouver (K.R.S.), and University of Manitoba, Winnipeg (D.A.W.) - both in Canada; Children's Hospital of Philadelphia, Philadelphia (N.B.); Fred Hutchinson Cancer Research Center, Seattle (C.D.); Indiana University, Indianapolis (P.H.); University of Michigan, Ann Arbor (E.P.); University of California San Francisco Benioff Children's Hospital Oakland, Oakland (M.W.); and Duke University, Durham, NC (J.K.).
Abstract
BACKGROUND: Umbilical-cord blood has been used as the source of hematopoietic stem cells in an estimated 30,000 transplants. The limited number of hematopoietic cells in a single cord-blood unit prevents its use in recipients with larger body mass and results in delayed hematopoietic recovery and higher mortality. Therefore, we hypothesized that the greater numbers of hematopoietic cells in two units of cord blood would be associated with improved outcomes after transplantation. METHODS:Between December 1, 2006, and February 24, 2012, a total of 224 patients 1 to 21 years of age with hematologic cancer were randomly assigned to undergo double-unit (111 patients) or single-unit (113 patients) cord-blood transplantation after a uniform myeloablative conditioning regimen and immunoprophylaxis for graft-versus-host disease (GVHD). The primary end point was 1-year overall survival. RESULTS:Treatment groups were matched for age, sex, self-reported race (white vs. nonwhite), performance status, degree of donor-recipient HLA matching, and disease type and status at transplantation. The 1-year overall survival rate was 65% (95% confidence interval [CI], 56 to 74) and 73% (95% CI, 63 to 80) among recipients of double and single cord-blood units, respectively (P=0.17). Similar outcomes in the two groups were also observed with respect to the rates of disease-free survival, neutrophil recovery, transplantation-related death, relapse, infections, immunologic reconstitution, and grade II-IV acute GVHD. However, improved platelet recovery and lower incidences of grade III and IV acute and extensive chronic GVHD were observed among recipients of a single cord-blood unit. CONCLUSIONS: We found that among children and adolescents with hematologic cancer, survival rates were similar after single-unit and double-unit cord-blood transplantation; however, a single-unit cord-blood transplant was associated with better platelet recovery and a lower risk of GVHD. (Funded by the National Heart, Lung, and Blood Institute and the National Cancer Institute; ClinicalTrials.gov number, NCT00412360.).
RCT Entities:
BACKGROUND: Umbilical-cord blood has been used as the source of hematopoietic stem cells in an estimated 30,000 transplants. The limited number of hematopoietic cells in a single cord-blood unit prevents its use in recipients with larger body mass and results in delayed hematopoietic recovery and higher mortality. Therefore, we hypothesized that the greater numbers of hematopoietic cells in two units of cord blood would be associated with improved outcomes after transplantation. METHODS: Between December 1, 2006, and February 24, 2012, a total of 224 patients 1 to 21 years of age with hematologic cancer were randomly assigned to undergo double-unit (111 patients) or single-unit (113 patients) cord-blood transplantation after a uniform myeloablative conditioning regimen and immunoprophylaxis for graft-versus-host disease (GVHD). The primary end point was 1-year overall survival. RESULTS: Treatment groups were matched for age, sex, self-reported race (white vs. nonwhite), performance status, degree of donor-recipient HLA matching, and disease type and status at transplantation. The 1-year overall survival rate was 65% (95% confidence interval [CI], 56 to 74) and 73% (95% CI, 63 to 80) among recipients of double and single cord-blood units, respectively (P=0.17). Similar outcomes in the two groups were also observed with respect to the rates of disease-free survival, neutrophil recovery, transplantation-related death, relapse, infections, immunologic reconstitution, and grade II-IV acute GVHD. However, improved platelet recovery and lower incidences of grade III and IV acute and extensive chronic GVHD were observed among recipients of a single cord-blood unit. CONCLUSIONS: We found that among children and adolescents with hematologic cancer, survival rates were similar after single-unit and double-unit cord-blood transplantation; however, a single-unit cord-blood transplant was associated with better platelet recovery and a lower risk of GVHD. (Funded by the National Heart, Lung, and Blood Institute and the National Cancer Institute; ClinicalTrials.gov number, NCT00412360.).
Authors: J Kurtzberg; M Laughlin; M L Graham; C Smith; J F Olson; E C Halperin; G Ciocci; C Carrier; C E Stevens; P Rubinstein Journal: N Engl J Med Date: 1996-07-18 Impact factor: 91.245
Authors: A Ruggeri; G Sanz; H Bittencourt; J Sanz; A Rambaldi; F Volt; I Yakoub-Agha; J M Ribera; L Mannone; J Sierra; M Mohty; C Solano; S Nabhan; W Arcese; E Gluckman; M Labopin; V Rocha Journal: Leukemia Date: 2013-09-05 Impact factor: 11.528
Authors: E Gluckman; V Rocha; A Boyer-Chammard; F Locatelli; W Arcese; R Pasquini; J Ortega; G Souillet; E Ferreira; J P Laporte; M Fernandez; C Chastang Journal: N Engl J Med Date: 1997-08-07 Impact factor: 91.245
Authors: Juliet N Barker; Courtney E Byam; Nancy A Kernan; Sinda S Lee; Rebecca M Hawke; Kathleen A Doshi; Deborah S Wells; Glenn Heller; Esperanza B Papadopoulos; Andromachi Scaradavou; James W Young; Marcel R M van den Brink Journal: Biol Blood Marrow Transplant Date: 2010-08-25 Impact factor: 5.742
Authors: Andromachi Scaradavou; Claudio G Brunstein; Mary Eapen; Jennifer Le-Rademacher; Juliet N Barker; Nelson Chao; Corey Cutler; Colleen Delaney; Fangyu Kan; Luis Isola; Chatchada Karanes; Mary J Laughlin; John E Wagner; Elizabeth J Shpall Journal: Blood Date: 2012-12-09 Impact factor: 22.113
Authors: Thomas Bart; Michael Boo; Snejana Balabanova; Yvonne Fischer; Grazia Nicoloso; Lydia Foeken; Machteld Oudshoorn; Jakob Passweg; Andre Tichelli; Vincent Kindler; Joanne Kurtzberg; Thomas Price; Donna Regan; Elizabeth J Shpall; Rudolf Schwabe Journal: Transfus Med Hemother Date: 2013-01-07 Impact factor: 3.747
Authors: Joanne Kurtzberg; Vinod K Prasad; Shelly L Carter; John E Wagner; Lee Ann Baxter-Lowe; Donna Wall; Neena Kapoor; Eva C Guinan; Stephen A Feig; Elizabeth L Wagner; Nancy A Kernan Journal: Blood Date: 2008-08-21 Impact factor: 22.113
Authors: U Sobol; A Go; S Kliethermes; S Bufalino; T Rodriguez; S Smith; M Parthasarathy; P Stiff Journal: Bone Marrow Transplant Date: 2015-09-14 Impact factor: 5.483
Authors: Juliet N Barker; Jane Kempenich; Joanne Kurtzberg; Claudio G Brunstein; Colleen Delaney; Filippo Milano; Ioannis Politikos; Elizabeth J Shpall; Andromachi Scaradavou; Jason Dehn Journal: Blood Adv Date: 2019-04-23
Authors: Rohtesh S Mehta; Shernan G Holtan; Tao Wang; Michael T Hemmer; Stephen R Spellman; Mukta Arora; Daniel R Couriel; Amin M Alousi; Joseph Pidala; Hisham Abdel-Azim; Ibrahim Ahmed; Mahmoud Aljurf; Medhat Askar; Jeffery J Auletta; Vijaya Bhatt; Christopher Bredeson; Saurabh Chhabra; Shahinaz Gadalla; James Gajewski; Robert Peter Gale; Usama Gergis; Peiman Hematti; Gerhard C Hildebrandt; Yoshihiro Inamoto; Carrie Kitko; Pooja Khandelwal; Margaret L MacMillan; Navneet Majhail; David I Marks; Parinda Mehta; Taiga Nishihori; Richard F Olsson; Attaphol Pawarode; Miguel Angel Diaz; Tim Prestidge; Muna Qayed; Hemalatha Rangarajan; Olle Ringden; Ayman Saad; Bipin N Savani; Sachiko Seo; Ami Shah; Niketa Shah; Kirk R Schultz; Melhem Solh; Thomas Spitzer; Jeffrey Szer; Takanori Teshima; Leo F Verdonck; Kirsten M Williams; Baldeep Wirk; John Wagner; Jean A Yared; Daniel J Weisdorf Journal: Blood Adv Date: 2019-05-14