The Journal of Pediatrics
Volume 154, Issue 1 , Pages 135-139, January 2009

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

  • Robert F. Wynn, MD

      Affiliations

    • Blood and Marrow Transplant Unit, Royal Manchester Children's Hospital, Manchester, UK
    • Corresponding Author InformationReprint requests: Dr Robert F. Wynn, BMTU Programme Director, Royal Manchester Children's Hospital, Manchester M27 4HA, UK
    • ,
  • Jean Mercer, RGN

      Affiliations

    • Willink Unit for Biochemical Genetics, Royal Manchester Children's Hospital, Manchester, UK
    • ,
  • Joanne Page, BSc

      Affiliations

    • Blood and Marrow Transplant Unit, Royal Manchester Children's Hospital, Manchester, UK
    • ,
  • Trevor F. Carr, PhD

      Affiliations

    • Blood and Marrow Transplant Unit, Royal Manchester Children's Hospital, Manchester, UK
    • ,
  • Simon Jones, MRCPCH

      Affiliations

    • Willink Unit for Biochemical Genetics, Royal Manchester Children's Hospital, Manchester, UK
    • ,
  • J. Edmond Wraith, FRCPCH

      Affiliations

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

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

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 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

The Journal of Pediatrics
Volume 154, Issue 1 , Pages 135-139, January 2009

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