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

Sandeep Soni, Monica Hente, Nancy Breslin, Joseph Hersh, Chester B Whitley, Alexandra Cheerva, Salvatore Bertolone

Research output: Contribution to journalArticlepeer-review

10 Scopus citations

Abstract

Combined enzyme replacement therapy (ERT) and stem cell transplant (SCT) were done for a two year old boy with severe Hurler syndrome(HS) with the aim to decrease transplant related complications. He tolerated both the procedures well without any major complications. Urine glycosaminoglycans (GAGs) decreased post-transplant and child has improved clinically and neurologically. Insignificant titers of the anti-iduronidase antibodies which developed post-transplant did not affect the transplant outcome or the endogenous recovery of the alpha-L-iduronidase enzyme.

Original languageEnglish (US)
Pages (from-to)563-567
Number of pages5
JournalPediatric transplantation
Volume11
Issue number5
DOIs
StatePublished - Aug 1 2007

Keywords

  • Alpha-L-iduronidase (Aldurazyme)
  • Glycosaminoglycans
  • Mucopolysaccharidosis
  • Stem cell transplantation

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