title: Long-term outcome of non-ablative booster BMT in patients with SCID.
abstract: 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.
authors: Departments of Pediatrics and Immunology, Duke University Medical Center, Durham, NC 27710, USA.
authors: C L
authors: CL
authors: Teigland
authors: R E
authors: RE
authors: Parrott
authors: R H
authors: RH
authors: Buckley
mesh_terms: D016026:Bone Marrow Transplantation / Q000009:adverse effects / Q000379:methods*; D005260:Female; D006086:Graft vs Host Disease / Q000209:etiology / Q000276:immunology; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D008297:Male; D011446:Prospective Studies; D012189:Retrospective Studies; D016511:Severe Combined Immunodeficiency / Q000235:genetics / Q000276:immunology / Q000601:surgery*; D013601:T-Lymphocytes / Q000276:immunology; D018183:Transplantation Chimera / Q000276:immunology; D016896:Treatment Outcome
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