Use of enzyme replacement therapy (Laronidase) before hematopoietic stem cell transplantation for mucopolysaccharidosis I: experience in 18 patients.
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.
Wynn
Robert F
RF
Blood and Marrow Transplant Unit, Royal Manchester Children's Hospital, Manchester, UK. robert.wynn@cmmc.nhs.uk
Mercer
Jean
J
Page
Joanne
J
Carr
Trevor F
TF
Jones
Simon
S
Wraith
J Edmond
JE
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
D007068:Iduronidase