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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.

Publication ,  Journal Article
Railey, MD; Lokhnygina, Y; Buckley, RH
Published in: J Pediatr
December 2009

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.

Duke Scholars

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Published In

J Pediatr

DOI

EISSN

1097-6833

Publication Date

December 2009

Volume

155

Issue

6

Start / End Page

834 / 840.e1

Location

United States

Related Subject Headings

  • Young Adult
  • Treatment Outcome
  • Transplantation Conditioning
  • Survival Rate
  • Severe Combined Immunodeficiency
  • Pediatrics
  • Male
  • Infant
  • Humans
  • Health Status
 

Citation

APA
Chicago
ICMJE
MLA
NLM
Railey, Mary Dell, Yuliya Lokhnygina, and Rebecca H. Buckley. “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.J Pediatr 155, no. 6 (December 2009): 834-840.e1. https://doi.org/10.1016/j.jpeds.2009.07.049.
Journal cover image

Published In

J Pediatr

DOI

EISSN

1097-6833

Publication Date

December 2009

Volume

155

Issue

6

Start / End Page

834 / 840.e1

Location

United States

Related Subject Headings

  • Young Adult
  • Treatment Outcome
  • Transplantation Conditioning
  • Survival Rate
  • Severe Combined Immunodeficiency
  • Pediatrics
  • Male
  • Infant
  • Humans
  • Health Status