Skip to main content
Journal cover image

Results of the cord blood transplantation study (COBLT): outcomes of unrelated donor umbilical cord blood transplantation in pediatric patients with lysosomal and peroxisomal storage diseases.

Publication ,  Journal Article
Martin, PL; Carter, SL; Kernan, NA; Sahdev, I; Wall, D; Pietryga, D; Wagner, JE; Kurtzberg, J
Published in: Biol Blood Marrow Transplant
February 2006

The Cord Blood Transplantation Study (COBLT), sponsored by the National Heart, Lung, and Blood Institute, is a phase II multicenter study designed to evaluate the use of cord blood in allogeneic transplantation. In this report, we evaluated the outcomes of cord blood transplantation in 69 patients with lysosomal and peroxisomal storage diseases. Patients with mucopolysaccharidoses I to III, mucolipidoses (ML) II (n = 36), adrenoleukodystrophy (n = 8), metachromatic leukodystrophy (n = 6), Krabbe disease (n = 16), and Tay-Sachs disease (n = 3) were enrolled between August 1999 and June 2004. All patients received the same preparative regimen, graft-versus-host disease (GVHD) prophylaxis, and supportive care. End points included survival, engraftment, GVHD, and toxicity. Sixty-nine patients (64% men; 81% white) with a median age of 1.8 years underwent transplantation with a median cell dose of 8.7 x 10(7)/kg. One-year survival was 72% (95% confidence interval, 61%-83%). The cumulative incidence of neutrophil engraftment by day 42 was 78% (95% confidence interval, 67%-87%) at a median of 25 days. Grade II to IV acute GVHD occurred in 36% of patients. Cord blood donors are readily available for rapid transplantation. Cord blood transplantation should be considered as frontline therapy for young patients with lysosomal and peroxisomal storage diseases.

Duke Scholars

Published In

Biol Blood Marrow Transplant

DOI

ISSN

1083-8791

Publication Date

February 2006

Volume

12

Issue

2

Start / End Page

184 / 194

Location

United States

Related Subject Headings

  • Transplantation, Homologous
  • Transplantation Conditioning
  • Survival Rate
  • Retrospective Studies
  • Male
  • Lysosomal Storage Diseases
  • Infant
  • Immunology
  • Humans
  • Graft vs Host Disease
 

Citation

APA
Chicago
ICMJE
MLA
NLM
Martin, P. L., Carter, S. L., Kernan, N. A., Sahdev, I., Wall, D., Pietryga, D., … Kurtzberg, J. (2006). Results of the cord blood transplantation study (COBLT): outcomes of unrelated donor umbilical cord blood transplantation in pediatric patients with lysosomal and peroxisomal storage diseases. Biol Blood Marrow Transplant, 12(2), 184–194. https://doi.org/10.1016/j.bbmt.2005.09.016
Martin, Paul L., Shelly L. Carter, Nancy A. Kernan, Indira Sahdev, Donna Wall, Daniel Pietryga, John E. Wagner, and Joanne Kurtzberg. “Results of the cord blood transplantation study (COBLT): outcomes of unrelated donor umbilical cord blood transplantation in pediatric patients with lysosomal and peroxisomal storage diseases.Biol Blood Marrow Transplant 12, no. 2 (February 2006): 184–94. https://doi.org/10.1016/j.bbmt.2005.09.016.
Martin, Paul L., et al. “Results of the cord blood transplantation study (COBLT): outcomes of unrelated donor umbilical cord blood transplantation in pediatric patients with lysosomal and peroxisomal storage diseases.Biol Blood Marrow Transplant, vol. 12, no. 2, Feb. 2006, pp. 184–94. Pubmed, doi:10.1016/j.bbmt.2005.09.016.
Martin PL, Carter SL, Kernan NA, Sahdev I, Wall D, Pietryga D, Wagner JE, Kurtzberg J. Results of the cord blood transplantation study (COBLT): outcomes of unrelated donor umbilical cord blood transplantation in pediatric patients with lysosomal and peroxisomal storage diseases. Biol Blood Marrow Transplant. 2006 Feb;12(2):184–194.
Journal cover image

Published In

Biol Blood Marrow Transplant

DOI

ISSN

1083-8791

Publication Date

February 2006

Volume

12

Issue

2

Start / End Page

184 / 194

Location

United States

Related Subject Headings

  • Transplantation, Homologous
  • Transplantation Conditioning
  • Survival Rate
  • Retrospective Studies
  • Male
  • Lysosomal Storage Diseases
  • Infant
  • Immunology
  • Humans
  • Graft vs Host Disease