title: Use of enzyme replacement therapy (Laronidase) before hematopoietic stem cell transplantation for mucopolysaccharidosis I: experience in 18 patients.
abstract: 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.
authors.affiliation: Blood and Marrow Transplant Unit, Royal Manchester Children's Hospital, Manchester, UK. robert.wynn@cmmc.nhs.uk
authors.forename: Robert F
authors.initials: RF
authors.lastname: Wynn
authors.forename: Jean
authors.initials: J
authors.lastname: Mercer
authors.forename: Joanne
authors.initials: J
authors.lastname: Page
authors.forename: Trevor F
authors.initials: TF
authors.lastname: Carr
authors.forename: Simon
authors.initials: S
authors.lastname: Jones
authors.forename: J Edmond
authors.initials: JE
authors.lastname: Wraith
mesh_terms: D005260:Female; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007068:Iduronidase / Q000008:administration & dosage*; D007223:Infant; D008297:Male; D008059:Mucopolysaccharidosis I / Q000401:mortality / Q000601:surgery*; D020360:Neoadjuvant Therapy; D016019:Survival Analysis; D019172:Transplantation Conditioning; D016896:Treatment Outcome
chemical_list: D007068:Iduronidase