Use of Enzyme Replacement Therapy (Laronidase) before Hematopoietic Stem Cell Transplantation for Mucopolysaccharidosis I: Experience in 18 Patients

Received 11 February 2008; received in revised form 16 June 2008; accepted 2 July 2008.

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.

Abbreviations: ERT, Enzyme replacement therapy, GVHD, Graft-versus-host disease, HSCT, Hematopoietic stem cell transplantation, MPS I, Mucopolysaccharidosis type I

a Blood and Marrow Transplant Unit, Royal Manchester Children's Hospital, Manchester, UK

b Willink Unit for Biochemical Genetics, Royal Manchester Children's Hospital, Manchester, UK

Reprint requests: Dr Robert F. Wynn, BMTU Programme Director, Royal Manchester Children's Hospital, Manchester M27 4HA, UK

Dr Wraith has undertaken paid and unpaid consultancy work for, and has been a principle investigator in, studies sponsored by Genzyme.

PII: S0022-3476(08)00577-5

doi:10.1016/j.jpeds.2008.07.004

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