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Unrelated donor umbilical cord blood transplantation for inherited metabolic disorders in 159 pediatric patients from a single center: influence of cellular composition of the graft on transplantation outcomes.

Publication ,  Journal Article
Prasad, VK; Mendizabal, A; Parikh, SH; Szabolcs, P; Driscoll, TA; Page, K; Lakshminarayanan, S; Allison, J; Wood, S; Semmel, D; Escolar, ML ...
Published in: Blood
October 1, 2008

Outcomes of 159 young patients with inherited metabolic disorders (IMDs) undergoing transplantation with partially HLA-mismatched unrelated donor umbilical cord blood were studied to investigate the impact of graft and patient characteristics on engraftment, overall survival (OS), and graft-versus-host disease (GVHD). Patients received myeloablative chemotherapy (busulfan, cyclophosphamide, ATG) and cyclosporine-based GVHD prophylaxis. Infused cell doses were high (7.57 x 10(7)/kg) because of the patients' young age (median, 1.5 years) and small size (median, 12 kg). Median follow-up was 4.2 years (range, 1-11 years). The cumulative incidences of neutrophil and platelet engraftment were 87.1% (95% confidence interval [CI], 81.8%-92.4%) and 71.0% (95% CI, 63.7%-78.3%). A total of 97% achieved high (> 90%) donor chimerism. Serum enzyme normalized in 97% of patients with diseases for which testings exist. Grade III/IV acute GVHD occurred in 10.3% (95% CI, 5.4%-15.2%) of patients. Extensive chronic GVHD occurred in 10.8% (95% CI, 5.7%-15.9%) of patients by 1 year. OS at 1 and 5 years was 71.8% (95% CI, 64.7%-78.9%) and 58.2% (95% CI, 49.7%-66.6%) in all patients and 84.5% (95% CI, 77.0%-92.0%) and 75.7% (95% CI, 66.1%-85.3%) in patients with high (80-100) performance score. In multivariate analysis, favorable factors for OS were high pretransplantation performance status, matched donor/recipient ethnicity, and higher infused colony forming units.

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Published In

Blood

DOI

EISSN

1528-0020

Publication Date

October 1, 2008

Volume

112

Issue

7

Start / End Page

2979 / 2989

Location

United States

Related Subject Headings

  • United States
  • Treatment Outcome
  • Tissue Donors
  • Survival Rate
  • Neutrophils
  • Multivariate Analysis
  • Metabolism, Inborn Errors
  • Kaplan-Meier Estimate
  • Infant
  • Immunology
 

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Prasad, V. K., Mendizabal, A., Parikh, S. H., Szabolcs, P., Driscoll, T. A., Page, K., … Kurtzberg, J. (2008). Unrelated donor umbilical cord blood transplantation for inherited metabolic disorders in 159 pediatric patients from a single center: influence of cellular composition of the graft on transplantation outcomes. Blood, 112(7), 2979–2989. https://doi.org/10.1182/blood-2008-03-140830
Prasad, Vinod K., Adam Mendizabal, Suhag H. Parikh, Paul Szabolcs, Timothy A. Driscoll, Kristin Page, Sonali Lakshminarayanan, et al. “Unrelated donor umbilical cord blood transplantation for inherited metabolic disorders in 159 pediatric patients from a single center: influence of cellular composition of the graft on transplantation outcomes.Blood 112, no. 7 (October 1, 2008): 2979–89. https://doi.org/10.1182/blood-2008-03-140830.
Prasad VK, Mendizabal A, Parikh SH, Szabolcs P, Driscoll TA, Page K, Lakshminarayanan S, Allison J, Wood S, Semmel D, Escolar ML, Martin PL, Carter S, Kurtzberg J. Unrelated donor umbilical cord blood transplantation for inherited metabolic disorders in 159 pediatric patients from a single center: influence of cellular composition of the graft on transplantation outcomes. Blood. 2008 Oct 1;112(7):2979–2989.

Published In

Blood

DOI

EISSN

1528-0020

Publication Date

October 1, 2008

Volume

112

Issue

7

Start / End Page

2979 / 2989

Location

United States

Related Subject Headings

  • United States
  • Treatment Outcome
  • Tissue Donors
  • Survival Rate
  • Neutrophils
  • Multivariate Analysis
  • Metabolism, Inborn Errors
  • Kaplan-Meier Estimate
  • Infant
  • Immunology