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A novel reduced-intensity conditioning regimen for unrelated umbilical cord blood transplantation in children with nonmalignant diseases.

Publication ,  Journal Article
Parikh, SH; Mendizabal, A; Benjamin, CL; Komanduri, KV; Antony, J; Petrovic, A; Hale, G; Driscoll, TA; Martin, PL; Page, KM; Flickinger, K ...
Published in: Biol Blood Marrow Transplant
March 2014

Reduced-intensity conditioning (RIC) regimens have the potential to decrease transplantation-related morbidity and mortality. However, engraftment failure has been prohibitively high after RIC unrelated umbilical cord blood transplantation (UCBT) in chemotherapy-naïve children with nonmalignant diseases (NMD). Twenty-two children with a median age of 2.8 years, many with severe comorbidities and prior viral infections, were enrolled in a novel RIC protocol consisting of hydroxyurea, alemtuzumab, fludarabine, melphalan, and thiotepa followed by single UCBT. Patients underwent transplantation for inherited metabolic disorders (n = 8), primary immunodeficiencies (n = 9), hemoglobinopathies (n = 4) and Diamond Blackfan anemia (n = 1). Most umbilical cord blood (UCB) units were HLA-mismatched with median infused total nucleated cell dose of 7.9 × 10(7)/kg. No serious organ toxicities were attributable to the regimen. The cumulative incidence of neutrophil engraftment was 86.4% (95% confidence interval [CI], 65% to 100%) in a median of 20 days, with the majority sustaining > 95% donor chimerism at 1 year. Cumulative incidence of acute graft-versus-host disease (GVHD) grades II to IV and III to IV by day 180 was 27.3% (95% CI, 8.7% to 45.9%) and 13.6% (95 CI, 0% to 27.6%), respectively. Cumulative incidence of extensive chronic GVHD was 9.1% (95% CI, 0% to 20.8%). The primary causes of death were viral infections (n = 3), acute GVHD (n = 1) and transfusion reaction (n = 1). One-year overall and event-free survivals were 77.3% (95% CI, 53.7% to 89.8%) and 68.2% (95% CI, 44.6% to 83.4%) with 31 months median follow-up. This is the first RIC protocol demonstrating durable UCB engraftment in children with NMD. Future risk-based modifications of this regimen could decrease the incidence of viral infections. (www.clinicaltrials.gov/NCT00744692).

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Published In

Biol Blood Marrow Transplant

DOI

EISSN

1523-6536

Publication Date

March 2014

Volume

20

Issue

3

Start / End Page

326 / 336

Location

United States

Related Subject Headings

  • Unrelated Donors
  • Transplantation, Homologous
  • Transplantation Conditioning
  • Transplantation Chimera
  • Survival Analysis
  • Metabolic Diseases
  • Male
  • Infant
  • Immunology
  • Humans
 

Citation

APA
Chicago
ICMJE
MLA
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Parikh, S. H., Mendizabal, A., Benjamin, C. L., Komanduri, K. V., Antony, J., Petrovic, A., … Szabolcs, P. (2014). A novel reduced-intensity conditioning regimen for unrelated umbilical cord blood transplantation in children with nonmalignant diseases. Biol Blood Marrow Transplant, 20(3), 326–336. https://doi.org/10.1016/j.bbmt.2013.11.021
Parikh, Suhag H., Adam Mendizabal, Cara L. Benjamin, Krishna V. Komanduri, Jeyaraj Antony, Aleksandra Petrovic, Gregory Hale, et al. “A novel reduced-intensity conditioning regimen for unrelated umbilical cord blood transplantation in children with nonmalignant diseases.Biol Blood Marrow Transplant 20, no. 3 (March 2014): 326–36. https://doi.org/10.1016/j.bbmt.2013.11.021.
Parikh SH, Mendizabal A, Benjamin CL, Komanduri KV, Antony J, Petrovic A, et al. A novel reduced-intensity conditioning regimen for unrelated umbilical cord blood transplantation in children with nonmalignant diseases. Biol Blood Marrow Transplant. 2014 Mar;20(3):326–36.
Parikh, Suhag H., et al. “A novel reduced-intensity conditioning regimen for unrelated umbilical cord blood transplantation in children with nonmalignant diseases.Biol Blood Marrow Transplant, vol. 20, no. 3, Mar. 2014, pp. 326–36. Pubmed, doi:10.1016/j.bbmt.2013.11.021.
Parikh SH, Mendizabal A, Benjamin CL, Komanduri KV, Antony J, Petrovic A, Hale G, Driscoll TA, Martin PL, Page KM, Flickinger K, Moffet J, Niedzwiecki D, Kurtzberg J, Szabolcs P. A novel reduced-intensity conditioning regimen for unrelated umbilical cord blood transplantation in children with nonmalignant diseases. Biol Blood Marrow Transplant. 2014 Mar;20(3):326–336.
Journal cover image

Published In

Biol Blood Marrow Transplant

DOI

EISSN

1523-6536

Publication Date

March 2014

Volume

20

Issue

3

Start / End Page

326 / 336

Location

United States

Related Subject Headings

  • Unrelated Donors
  • Transplantation, Homologous
  • Transplantation Conditioning
  • Transplantation Chimera
  • Survival Analysis
  • Metabolic Diseases
  • Male
  • Infant
  • Immunology
  • Humans