title: Enzyme replacement is associated with better cognitive outcomes after transplant in Hurler syndrome.
abstract: OBJECTIVE
  To investigate whether intravenous enzyme replacement therapy (ERT) benefits cognitive function in patients with mucopolysaccharidosis type IH (Hurler syndrome) undergoing hematopoietic cell transplantation (HCT).
  
  
  METHODS
  Data were obtained for 9 children treated with HCT + ERT (ERT group) and 10 children treated with HCT only (no-ERT group) from neuropsychologic evaluations before HCT and at 1-year and 2-year post-HCT follow-up.
  
  
  RESULTS
  At 2 years after HCT, children in the ERT group lost 9.19 fewer IQ points per year compared with children in the no-ERT group (P = .031). Furthermore, the ERT group improved in nonverbal problem solving and processing, whereas the no-ERT group declined, resulting in a difference of 9.44 points per year between the 2 groups (P < .001).
  
  
  CONCLUSIONS
  ERT in association with HCT enhances cognitive outcomes, providing new evidence that ERT is a valuable addition to the standard transplantation protocol. Although the mechanism responsible for this improved outcome is unknown, both direct benefits and indirect effects must be considered.
authors.affiliation: Department of Pediatrics, Division of Pediatric Clinical Neuroscience, University of Minnesota, Minneapolis, MN, USA. jbeisengart@yahoo.com
authors.forename: Julie B
authors.initials: JB
authors.lastname: Eisengart
authors.forename: Kyle D
authors.initials: KD
authors.lastname: Rudser
authors.forename: Jakub
authors.initials: J
authors.lastname: Tolar
authors.forename: Paul J
authors.initials: PJ
authors.lastname: Orchard
authors.forename: Teresa
authors.initials: T
authors.lastname: Kivisto
authors.forename: Richard S
authors.initials: RS
authors.lastname: Ziegler
authors.forename: Chester B
authors.initials: CB
authors.lastname: Whitley
authors.forename: Elsa G
authors.initials: EG
authors.lastname: Shapiro
mesh_terms: D003071:Cognition; D056947:Enzyme Replacement Therapy; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007223:Infant; D008297:Male; D008059:Mucopolysaccharidosis I / Q000601:surgery*; D011446:Prospective Studies; D016896:Treatment Outcome