title: Long-term clinical outcome of patients with severe combined immunodeficiency who received related donor bone marrow transplants without pretransplant chemotherapy or post-transplant GVHD prophylaxis.
abstract: OBJECTIVE
  To determine long-term health benefits of nonablative bone marrow transplantation for severe combined immunodeficiency (SCID), we investigated our cohort of 161 related donor bone marrow-transplanted patients with SCID. Only 16 (10%) had HLA-identical donors.
  
  
  METHODS
  All 124 survivors were sent questionnaires about their current clinical statuses. Details from clinic visits were also compiled. One hundred eleven patients (90%) were reached. We compared outcomes of patients transplanted before and after 3.5 months of life and by molecular defect.
  
  
  RESULTS
  The overall survival rate was 77%, but the rate for the 48 infants transplanted in the first 3.5 months of life was 94%, compared with 70% for the 113 transplanted after 3.5 months (P = .002). Twenty-eight (76%) of the 37 deceased patients died of viral infections present at diagnosis. One or more clinical problems were reported to have been present in the past 2 years in 71 (64%) of the survivors, although 95 (86%) were considered healthy by their families.
  
  
  CONCLUSIONS
  Most patients with SCID transplanted with related donor marrow without pretransplant chemotherapy have done well in the long term, but those transplanted at <3.5 months of age had a superior survival rate, a lower rate of clinical problems, less need for booster transplants, and better nutritional status.
authors.affiliation: Department of Pediatrics, Duke University Medical Center, Durham, NC 27710, USA.
authors.forename: Mary Dell
authors.initials: MD
authors.lastname: Railey
authors.forename: Yuliya
authors.initials: Y
authors.lastname: Lokhnygina
authors.forename: Rebecca H
authors.initials: RH
authors.lastname: Buckley
mesh_terms: D000293:Adolescent; D000328:Adult; D000367:Age Factors; D016026:Bone Marrow Transplantation; D002648:Child; D002675:Child, Preschool; D015331:Cohort Studies; D005260:Female; D006086:Graft vs Host Disease / Q000517:prevention & control; D006304:Health Status; D006801:Humans; D007223:Infant; D008297:Male; D016511:Severe Combined Immunodeficiency / Q000235:genetics / Q000401:mortality* / Q000601:surgery*; D015996:Survival Rate; D019172:Transplantation Conditioning; D016896:Treatment Outcome; D055815:Young Adult