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Cord Blood Transplants for SCID: Better B-cell Engraftment?

Chan, Wan-Yin MD, MS; Roberts, Robert Lloyd MD, PhD; Moore, Theodore B. MD; Stiehm, E. Richard MD

Journal of Pediatric Hematology/Oncology: January 2013 - Volume 35 - Issue 1 - p e14–e18
doi: 10.1097/MPH.0b013e31824e15b8
Online Articles: Clinical and Laboratory Observations

Hematopoietic stem-cell transplantation is the treatment of choice for severe combined immunodeficiency (SCID). Despite successful T-cell engraftment in transplanted patients, B-cell function is not always achieved; up to 58% of patients require immunoglobulin therapy after receiving haploidentical transplants. We report 2 half-sibling males with X-linked γ-chain SCID treated with different sources of stem cells. Sibling 1 was transplanted with T-cell–depleted haploidentical maternal bone marrow and sibling 2 was transplanted with 7/8 human leukocyte antigen-matched unrelated umbilical cord blood. Both patients received pretransplant conditioning and posttransplant graft-versus-host-disease prophylaxis. B-cell engraftment and function was achieved in sibling 1 but not in sibling 2. This disparate result is consistent with a review of 19 other SCID children who received cord blood transplants. B-cell function, as indicated by no need for immunoglobulin therapy, was restored in 42% of patients given haploidentical transplants and in 68% of patients given matched unrelated donor transplants compared with 80% of patients given cord blood transplants. Cord blood is an alternative source of stem cells for transplantation in children with SCID and has a higher likelihood of B-cell reconstitution.

*Department of Pediatrics, Division of Immunology, Allergy, and Rheumatology

Division of Hematology/Oncology, University of California, Los Angeles, CA

The authors declare no conflict of interest.

Reprints: Wan-Yin Chan, MD, MS, Department of Pediatrics, Division of Immunology, Allergy, and Rheumatology, University of California, 10833 Le Conte Ave., Room 12-430 MDCC, Los Angeles, CA 90095 (e-mail:

Received October 21, 2011

Accepted February 1, 2012

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