Long-term outcome of non-ablative booster BMT in patients with SCID

Journal Article

SCID is a fatal syndrome caused by mutations in at least 13 different genes. It is characterized by the absence of T cells. Immune reconstitution can be achieved through nonablative related donor BMT. However, the first transplant may not provide sufficient immunity. In these cases, booster transplants may be helpful. A prospective/retrospective study was conducted of 49 SCID patients (28.7% of 171 SCIDs transplanted over 30 years) who had received booster transplants to define the long-term outcome, factors contributing to a need for a booster and factors that predicted success. Of the 49 patients, 31 (63%) are alive for up to 28 years. Age at initial transplantation was found to have a significant effect on outcome (mean of 194 days old for patients currently alive, versus a mean of 273 days old for those now deceased, P=0.0401). Persistent viral infection was present in most deceased booster patients. In several patients, the use of two parents as sequential donors resulted in striking T-and B-cell immune reconstitution. A majority of the patients alive today have normal or adequate T-cell function and are healthy. Nonablative booster BMT can be lifesaving for SCID. © 2013 Macmillan Publishers Limited. All rights reserved.

Full Text

Duke Authors

Cited Authors

  • Teigland, CL; Parrott, RE; Buckley, RH

Published Date

  • 2013

Published In

Volume / Issue

  • 48 / 8

Start / End Page

  • 1050 - 1055

International Standard Serial Number (ISSN)

  • 0268-3369

Digital Object Identifier (DOI)

  • 10.1038/bmt.2013.6