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Cord blood transplantation for nonmalignant disorders: early functional immunity and high survival.
Martinez, Caridad; Aguayo-Hiraldo, Paibel; Chaimowitz, Natalia; Forbes, Lisa; Rider, Nicholas; Nicholas, Sarah; Seeborg, Filiz; Chinen, Javier; Chinn, Ivan; Davis, Carla; Roseblatt, Howard; Noroski, Lenora; Omer, Bilal; John, Tami; Yassine, Khaled; Naik, Swati; Craddock, John; Bhar, Saleh; Allen, Carl; Ahmed, Nabil; Sasa, Ghadir; Steffin, David; Doherty, Erin; George, Anil; Salem, Baheyeldin; Friend, Brian; Hegde, Meenakshi; Brenner, Malcolm K; Heslop, Helen E; Leen, Ann; Peña, Amanda; Wu, Mengfen; Hanson, I Celine; Krance, Robert A.
Affiliation
  • Martinez C; Center for Cell and Gene Therapy, Bayor College of Medicine, Texas Children's Hospital, Houston, TX.
  • Aguayo-Hiraldo P; Center for Cell and Gene Therapy, Bayor College of Medicine, Texas Children's Hospital, Houston, TX.
  • Chaimowitz N; Immunology, Allergy and Retrovirology, Baylor College of Medicine, Texas Children's Hospital, Houston, TX.
  • Forbes L; Immunology, Allergy and Retrovirology, Baylor College of Medicine, Texas Children's Hospital, Houston, TX.
  • Rider N; Immunology, Allergy and Retrovirology, Baylor College of Medicine, Texas Children's Hospital, Houston, TX.
  • Nicholas S; Immunology, Allergy and Retrovirology, Baylor College of Medicine, Texas Children's Hospital, Houston, TX.
  • Seeborg F; Immunology, Allergy and Retrovirology, Baylor College of Medicine, Texas Children's Hospital, Houston, TX.
  • Chinen J; Immunology, Allergy and Retrovirology, Baylor College of Medicine, Texas Children's Hospital, Houston, TX.
  • Chinn I; Immunology, Allergy and Retrovirology, Baylor College of Medicine, Texas Children's Hospital, Houston, TX.
  • Davis C; Immunology, Allergy and Retrovirology, Baylor College of Medicine, Texas Children's Hospital, Houston, TX.
  • Roseblatt H; Immunology, Dell Children's Medical Center and SFC, Austin, TX.
  • Noroski L; Immunology, Allergy and Retrovirology, Baylor College of Medicine, Texas Children's Hospital, Houston, TX.
  • Omer B; Center for Cell and Gene Therapy, Bayor College of Medicine, Texas Children's Hospital, Houston, TX.
  • John T; Center for Cell and Gene Therapy, Bayor College of Medicine, Texas Children's Hospital, Houston, TX.
  • Yassine K; Center for Cell and Gene Therapy, Bayor College of Medicine, Texas Children's Hospital, Houston, TX.
  • Naik S; Center for Cell and Gene Therapy, Bayor College of Medicine, Texas Children's Hospital, Houston, TX.
  • Craddock J; Center for Cell and Gene Therapy, Bayor College of Medicine, Texas Children's Hospital, Houston, TX.
  • Bhar S; Center for Cell and Gene Therapy, Bayor College of Medicine, Texas Children's Hospital, Houston, TX.
  • Allen C; Center for Cell and Gene Therapy, Bayor College of Medicine, Texas Children's Hospital, Houston, TX.
  • Ahmed N; Center for Cell and Gene Therapy, Bayor College of Medicine, Texas Children's Hospital, Houston, TX.
  • Sasa G; Center for Cell and Gene Therapy, Bayor College of Medicine, Texas Children's Hospital, Houston, TX.
  • Steffin D; Center for Cell and Gene Therapy, Bayor College of Medicine, Texas Children's Hospital, Houston, TX.
  • Doherty E; Center for Cell and Gene Therapy, Bayor College of Medicine, Texas Children's Hospital, Houston, TX.
  • George A; Center for Cell and Gene Therapy, Bayor College of Medicine, Texas Children's Hospital, Houston, TX.
  • Salem B; Center for Cell and Gene Therapy, Bayor College of Medicine, Texas Children's Hospital, Houston, TX.
  • Friend B; Center for Cell and Gene Therapy, Bayor College of Medicine, Texas Children's Hospital, Houston, TX.
  • Hegde M; Center for Cell and Gene Therapy, Bayor College of Medicine, Texas Children's Hospital, Houston, TX.
  • Brenner MK; Center for Cell and Gene Therapy, Bayor College of Medicine, Texas Children's Hospital, Houston, TX.
  • Heslop HE; Center for Cell and Gene Therapy, Bayor College of Medicine, Texas Children's Hospital, Houston, TX.
  • Leen A; Center for Cell and Gene Therapy, Bayor College of Medicine, Texas Children's Hospital, Houston, TX.
  • Peña A; Center for Cell and Gene Therapy, Bayor College of Medicine, Texas Children's Hospital, Houston, TX.
  • Wu M; Biostatistics Shared Resource, Dan L. Duncan Cancer Center, Baylor College of Medicine, Houston, TX.
  • Hanson IC; Immunology, Allergy and Retrovirology, Baylor College of Medicine, Texas Children's Hospital, Houston, TX.
  • Krance RA; Center for Cell and Gene Therapy, Bayor College of Medicine, Texas Children's Hospital, Houston, TX.
Blood Adv ; 7(9): 1823-1830, 2023 05 09.
Article in En | MEDLINE | ID: mdl-36453638
There is no consensus on the best donor for children with nonmalignant disorders and immune deficiencies in the absence of a matched related donor (MRD). We evaluated the 2-year overall survival (OS) after umbilical cord blood transplantation (UCBT) in patients with nonmalignant disorders from 2009 to 2020 enrolled in a prospective clinical trial using either 5/6 or 6/6 UCB as the cell source. Patients receive a fully ablative busulfan, cyclophosphamide, and fludarabine without serotherapy. Fifty-five children were enrolled, median age 5 months (range, 1-111 months); primary immune deficiency (45), metabolic (5), hemophagocytic lymphohistiocytosis (1), and hematologic disorders (4). Twenty-six patients had persistent infections before transplant. Nineteen of them (34%) were 6/6 matched, and 36 (66%) were 5/6 human leukocyte antigen-matched. The OS at 2 years was 91% (95% cumulative incidence, 79-96), with a median follow-up of 4.3 years. The median time to neutrophil and platelet recovery were 17 days (range, 5-39 days) and 37 days (range, 20-92 days), respectively. All but one evaluable patient achieved full donor chimerism. The cumulative incidence of acute GVHD grades 2-4 on day 100 was 16% (n = 9). All patients with viral infections at the time of transplant cleared the infection at a median time of 54 days (range, 44-91 days). All evaluable patients underwent correction of their immune or metabolic defects. We conclude that in the absence of MRD, UCBT following myeloablative conditioning without serotherapy is an excellent curative option in young children with nonmalignant disorders. This trial has been registered at www.clinicaltrials.gov as NCT00950846.
Subject(s)

Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Hematopoietic Stem Cell Transplantation / Cord Blood Stem Cell Transplantation Limits: Child / Child, preschool / Humans / Infant Language: En Journal: Blood Adv Year: 2023 Document type: Article Country of publication:

Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Hematopoietic Stem Cell Transplantation / Cord Blood Stem Cell Transplantation Limits: Child / Child, preschool / Humans / Infant Language: En Journal: Blood Adv Year: 2023 Document type: Article Country of publication: