Cord blood transplantation for nonmalignant disorders: early functional immunity and high survival

Caridad Martinez, Paibel Aguayo-Hiraldo, Natalia Chaimowitz, Lisa Forbes, Nicholas Rider, Sarah Nicholas, Filiz Seeborg, Javier Chinen, Ivan Chinn, Carla Davis, Howard Roseblatt, Lenora Noroski, Bilal Omer, Tami John, Khaled Yassine, Swati Naik, John Craddock, Saleh Bhar, Carl Allen, Nabil AhmedGhadir Sasa, David Steffin, Erin Doherty, Anil George, Baheyeldin Salem, Brian Friend, Meenakshi Hegde, Malcolm K. Brenner, Helen E. Heslop, Ann Leen, Amanda Peña, Mengfen Wu, I. Celine Hanson, Robert A. Krance

Research output: Contribution to journalArticlepeer-review

4 Scopus citations


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 as NCT00950846.

Original languageEnglish (US)
Pages (from-to)1823-1830
Number of pages8
JournalBlood Advances
Issue number9
StatePublished - May 9 2023


  • Child
  • Child, Preschool
  • Humans
  • Infant
  • Busulfan
  • Cord Blood Stem Cell Transplantation
  • Cyclophosphamide/therapeutic use
  • Hematopoietic Stem Cell Transplantation
  • Prospective Studies

ASJC Scopus subject areas

  • Hematology


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