Journal
JOURNAL OF PEDIATRICS
Volume 155, Issue 6, Pages 834-U297Publisher
MOSBY-ELSEVIER
DOI: 10.1016/j.jpeds.2009.07.049
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Funding
- National Institute of Allergy and Infectious Diseases [AI042951, AI47605]
- National Center for Research Resources (NCRR), a component of the National Institutes of Health (NIH) [1UL1 RR024128-01]
- NIH Roadmap for Medical Research
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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. Study design 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.
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