title: 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.
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.
authors.affiliation: Division of Pediatric BMT, Columbus Children's Hospital, 700 Children's Drive, Columbus, OH 43205, USA. sonis@pediatrics.ohio-state.edu
authors.forename: Sandeep
authors.initials: S
authors.lastname: Soni
authors.forename: Monica
authors.initials: M
authors.lastname: Hente
authors.forename: Nancy
authors.initials: N
authors.lastname: Breslin
authors.forename: Joseph
authors.initials: J
authors.lastname: Hersh
authors.forename: Chester
authors.initials: C
authors.lastname: Whitley
authors.forename: Alexandra
authors.initials: A
authors.lastname: Cheerva
authors.forename: Salvatore
authors.initials: S
authors.lastname: Bertolone
mesh_terms: D000917:Antibody Formation / Q000276:immunology*; D002675:Child, Preschool; D005500:Follow-Up Studies; D006801:Humans; D007068:Iduronidase / Q000097:blood / Q000276:immunology / Q000627:therapeutic use*; D008297:Male; D008059:Mucopolysaccharidosis I / Q000201:enzymology / Q000276:immunology / Q000628:therapy*; D011300:Preoperative Care / Q000379:methods*; D011994:Recombinant Proteins; D020127:Recovery of Function; D033581:Stem Cell Transplantation
chemical_list: D011994:Recombinant Proteins; D007068:Iduronidase