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J Allergy Clin Immunol. 2014 Oct;134(4):935-943.e15. doi: 10.1016/j.jaci.2014.06.021. Epub 2014 Aug 7.

Comparison of outcomes of hematopoietic stem cell transplantation without chemotherapy conditioning by using matched sibling and unrelated donors for treatment of severe combined immunodeficiency.

Author information

  • 1Division of Pediatric Allergy, Immunology, and Blood and Marrow Transplant, Benioff Children's Hospital, University of California San Francisco, San Francisco, Calif. Electronic address:
  • 2Centre for Immunodeficiency, Molecular Immunology Unit, UCL Institute of Child Health, London, United Kingdom.
  • 3Institute of Cellular Medicine, Newcastle University, Newcastle upon Tyne, United Kingdom.
  • 4Department of Pediatrics, University Medical Center, Ulm, Germany.
  • 5Department of Pediatrics, Leiden University Medical Center, Leiden, The Netherlands.
  • 6Departments of Pediatrics and Immunology, Duke University Medical Center, Durham, NC.
  • 7Division of Hematology and Hematologic Malignancies, Primary Children's Hospital, University of Utah School of Medicine/Huntsman Cancer Institute, Salt Lake City, Utah.
  • 8Hematology/Oncology/BMT Program, British Columbia Children's Hospital, Vancouver, British Columbia, Canada.
  • 9University Children's Hospital, Stem Cell Transplantation Department, Zurich, Switzerland.
  • 10Oncology Department, The Children's Hospital at Westmead, Westmead, Australia.
  • 11Department of Pediatrics, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio.
  • 12Division of Oncology, Department of Pediatrics, Children's Hospital of Philadelphia, Philadelphia, Pa.
  • 13Department of Pediatric Hematology and Oncology, Teaching Hospital Motol, Prague, Czech Republic.
  • 14Division of Pediatric Hematology-Oncology, University of Michigan, Ann Arbor, Mich.
  • 15Department of Pediatrics, University of Oklahoma, Oklahoma City, Okla.
  • 16Division of Immunology and The Manton Center for Orphan Disease Research, Children's Hospital Boston, Harvard Medical School, Boston, Mass.
  • 17Division of Hematology and Oncology, Boston Children's Hospital, and the Department of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, Mass.
  • 18Division of Pediatric Allergy, Immunology, and Blood and Marrow Transplant, Benioff Children's Hospital, University of California San Francisco, San Francisco, Calif.



Patients with severe combined immunodeficiency disease who have matched sibling donors (MSDs) can proceed to hematopoietic cell transplantation (HCT) without conditioning chemotherapy.


We sought to determine whether the results of HCT without chemotherapy-based conditioning from matched unrelated donors (URDs), either from volunteer adults or umbilical cord blood, are comparable with those from MSDs.


We performed a multicenter survey of severe combined immunodeficiency transplantation centers in North America, Europe, and Australia to compile retrospective data on patients who have undergone unconditioned HCT from either URDs (n = 37) or MSDs (n = 66).


Most patients undergoing URD HCT (92%) achieved donor T-cell engraftment compared with 97% for those with MSDs; however, estimated 5-year overall and event-free survival were worse for URD recipients (71% and 60%, respectively) compared with MSD recipients (92% and 89%, respectively; P < .01 for both). URD recipients who received pre-HCT serotherapy had similar 5-year overall survival (100%) to MSD recipients. The incidences of grade II to IV acute and chronic graft-versus-host disease were higher in URD (50% and 39%, respectively) compared with MSD (22% and 5%, respectively) recipients (P < .01 for both). In the surviving patients there was no difference in T-cell reconstitution at the last follow-up between the URD and MSD recipients; however, MSD recipients were more likely to achieve B-cell reconstitution (72% vs 17%, P < .001).


Unconditioned URD HCT achieves excellent rates of donor T-cell engraftment similar to that seen in MSD recipients, and reconstitution rates are adequate. However, only a minority will have myeloid and B-cell reconstitution, and attention must be paid to graft-versus-host disease prophylaxis. This approach might be safer in children ineligible for intense regimens to spare the potential complications of chemotherapy.

Copyright © 2014 American Academy of Allergy, Asthma & Immunology. Published by Elsevier Inc. All rights reserved.


Severe combined immunodeficiency; conditioning; hematopoietic cell transplantation; serotherapy; sibling donors; umbilical cord blood; unrelated donors

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