title: Outcomes of transplantation using various hematopoietic cell sources in children with Hurler syndrome after myeloablative conditioning.
abstract: We report transplantation outcomes of 258 children with Hurler syndrome (HS) after a myeloablative conditioning regimen from 1995 to 2007. Median age at transplant was 16.7 months and median follow-up was 57 months. The cumulative incidence of neutrophil recovery at day 60 was 91%, acute graft-versus-host disease (GVHD) (grade II-IV) at day 100 was 25%, and chronic GVHD and 5 years was 16%. Overall survival and event-free survival (EFS) at 5 years were 74% and 63%, respectively. EFS after HLA-matched sibling donor (MSD) and 6/6 matched unrelated cord blood (CB) donor were similar at 81%, 66% after 10/10 HLA-matched unrelated donor (UD), and 68% after 5/6 matched CB donor. EFS was lower after transplantation in 4/6 matched unrelated CB (UCB) (57%; P = .031) and HLA-mismatched UD (41%; P = .007). Full-donor chimerism (P = .039) and normal enzyme levels (P = .007) were higher after CB transplantation (92% and 98%, respectively) compared with the other grafts sources (69% and 59%, respectively). In conclusion, results of allogeneic transplantation for HS are encouraging, with similar EFS rates after MSD, 6/6 matched UCB, 5/6 UCB, and 10/10 matched UD. The use of mismatched UD and 4/6 matched UCB was associated with lower EFS.
authors.affiliation: University Medical Center Utrecht, Pediatric Blood and Marrow Transplantation Program, Utrecht, The Netherlands. j.j.boelens@umcutrecht.nl
authors.forename: Jaap Jan
authors.initials: JJ
authors.lastname: Boelens
authors.forename: Mieke
authors.initials: M
authors.lastname: Aldenhoven
authors.forename: Duncan
authors.initials: D
authors.lastname: Purtill
authors.forename: Annalisa
authors.initials: A
authors.lastname: Ruggeri
authors.forename: Todd
authors.initials: T
authors.lastname: Defor
authors.forename: Robert
authors.initials: R
authors.lastname: Wynn
authors.forename: Ed
authors.initials: E
authors.lastname: Wraith
authors.forename: Marina
authors.initials: M
authors.lastname: Cavazzana-Calvo
authors.forename: Attilio
authors.initials: A
authors.lastname: Rovelli
authors.forename: Alain
authors.initials: A
authors.lastname: Fischer
authors.forename: Jakub
authors.initials: J
authors.lastname: Tolar
authors.forename: Vinod K
authors.initials: VK
authors.lastname: Prasad
authors.forename: Maria
authors.initials: M
authors.lastname: Escolar
authors.forename: Eliane
authors.initials: E
authors.lastname: Gluckman
authors.forename: Anne
authors.initials: A
authors.lastname: O'Meara
authors.forename: Paul J
authors.initials: PJ
authors.lastname: Orchard
authors.forename: Paul
authors.initials: P
authors.lastname: Veys
authors.forename: Mary
authors.initials: M
authors.lastname: Eapen
authors.forename: Joanne
authors.initials: J
authors.lastname: Kurtzberg
authors.forename: Vanderson
authors.initials: V
authors.lastname: Rocha
mesh_terms: D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects / Q000379:methods*; D006412:Hematopoietic Stem Cells / Q000166:cytology*; D006650:Histocompatibility Testing; D006801:Humans; D007223:Infant; D008297:Male; D008059:Mucopolysaccharidosis I / Q000453:epidemiology / Q000401:mortality / Q000628:therapy*; D019653:Myeloablative Agonists / Q000009:adverse effects / Q000627:therapeutic use*; D012189:Retrospective Studies; D014019:Tissue Donors; D009927:Tissue and Organ Procurement / Q000379:methods; D019172:Transplantation Conditioning / Q000009:adverse effects / Q000379:methods*; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D019653:Myeloablative Agonists