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 ,Revised 16 June 2008 ,Accepted 2 July 2008.

References 

  1. Neufeld EF, Muenzer J. The mucopolysaccharidoses. In:  Scriver C,  Beaudet A,  Sly W,  Valle D,  Childs B,  Kinzler K editor. The Metabolic and Molecular Bases of Inherited Disease. New York: McGraw-Hill; 2001;p. 3421–3452
  2. Peters C, Steward CG. Hematopoietic cell transplantation for inherited metabolic diseases: an overview of outcomes and practice guidelines. Bone Marrow Transplant. 2003;31:229–239
  3. Boelens JJ. Trends in haematopoietic cell transplantation for inborn errors of metabolism. J Inherit Metab Dis. 2006;29:413–420
  4. Boelens JJ, Wynn RF, O'Meara A, Veys P, Bertrand Y, Souillet G, et al. Outcomes of hematopoietic stem cell transplantation for Hurler's syndrome in Europe: a risk factor analysis for graft failure. Bone Marrow Transplant. 2007;40:225–233
  5. Kakkis ED, Muenzer J, Tiller GE, Waber L, Belmont J, Passage M, et al. Enzyme-replacement therapy in mucopolysaccharidosis I. N Engl J Med. 2001;344:182–188
  6. Wraith JE, Clarke LA, Beck M, Kolodny EH, Pastores GM, Muenzer J, et al. Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human alpha-L-iduronidase (laronidase). J Pediatr. 2004;144:581–588
  7. Wraith JE, Beck M, Lane R, Van der Ploeg AT, Shapiro E, Xue Y, et al. Enzyme replacement therapy in patients who have mucopolysaccharidosis I and are younger than 5 years: results of a multinational study of recombinant human alpha- L-iduronidase (laronidase). Pediatrics. 2007;120:e37–e46
  8. Cox-Brinkman J, Boelens JJ, Wraith JE, O'Meara A, Veys P, Wijburg FA, et al. Haematopoietic cell transplantation (HCT) in combination with enzyme replacement therapy (ERT) in patients with Hurler syndrome. Bone Marrow Transplant. 2006;38:17–21
  9. Hirth A, Berg A, Greve G. Successful treatment of severe heart failure in an infant with Hurler syndrome. J Inherit Metab Dis. 2007;30:820
  10. Tolar J, Grewal SS, Bjoraker KJ, Whitley CB, Shapiro EG, Charnas L. Combination of enzyme replacement and hematopoietic stem cell transplantation as therapy for Hurler syndrome. Bone Marrow Transplant. 2007;41:531–535
  11. Grewal SS, Wynn R, Abdenur JE, Burton BK, Gharib M, Haase C, et al. Safety and efficacy of enzyme replacement therapy in combination with hematopoietic stem cell transplantation in Hurler syndrome. Genet Med. 2005;7:143–146
  12. Soni S, Hente M, Breslin N, Hersh J, Whitley C, Cheerva A, et al. Pre-stem cell transplantation enzyme replacement therapy in Hurler syndrome does not lead to significant antibody formation or delayed recovery of the endogenous enzyme post-transplant: a case report. Pediatr Transplant. 2007;11:563–567
  13. Vassal G, Michel G, Esperou H, Gentet JC, Valteau-Couanet D, Doz F, et al. Prospective validation of a novel IV busulfan fixed dosing for paediatric patients to improve therapeutic AUC targeting without drug monitoring. Cancer Chemother Pharmacol. 2008;61:113–123
  14. Tran HT, Madden T, Petropoulos D, Worth LL, Felix EA, Sprigg-Saenz HA, et al. Individualizing high-dose oral busulfan: prospective dose adjustment in a pediatric population undergoing allogeneic stem cell transplantation for advanced hematologic malignancies. Bone Marrow Transplant. 2000;26:463–470
  15. Staba SL, Escolar ML, Poe M, Kim Y, Martin PL, Szabolcs P, et al. Cord-blood transplants from unrelated donors in patients with Hurler's syndrome. N Engl J Med. 2004;350:1960–1969

 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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