Use of enzyme replacement therapy (Laronidase) before hematopoietic stem cell transplantation for mucopolysaccharidosis I: experience in 18 patients

Robert F Wynn; Jean Mercer; Joanne Page; Trevor F Carr; Simon Jones; J Edmond Wraith

doi:10.1016/j.jpeds.2008.07.004

Use of enzyme replacement therapy (Laronidase) before hematopoietic stem cell transplantation for mucopolysaccharidosis I: experience in 18 patients

J Pediatr. 2009 Jan;154(1):135-9. doi: 10.1016/j.jpeds.2008.07.004.

Authors

Robert F Wynn¹, Jean Mercer, Joanne Page, Trevor F Carr, Simon Jones, J Edmond Wraith

Affiliation

¹ Blood and Marrow Transplant Unit, Royal Manchester Children's Hospital, Manchester, UK. robert.wynn@cmmc.nhs.uk

PMID: 19187736
DOI: 10.1016/j.jpeds.2008.07.004

Abstract

We describe the use of enzyme replacement therapy in conjunction with hematopoietic stem cell transplantation in 18 consecutive patients with severe mucopolysaccharidosis type I. The survival and engraftment rate was 89% overall and 93% for the 15 patients who received full-intensity conditioning.

Publication types

Research Support, Non-U.S. Gov't

MeSH terms

Female
Hematopoietic Stem Cell Transplantation*
Humans
Iduronidase / administration & dosage*
Infant
Male
Mucopolysaccharidosis I / mortality
Mucopolysaccharidosis I / surgery*
Neoadjuvant Therapy*
Survival Analysis
Transplantation Conditioning
Treatment Outcome

Substances

Iduronidase