title: Outcomes after allogeneic hematopoietic cell transplantation for childhood cerebral adrenoleukodystrophy: the largest single-institution cohort report.
abstract: Cerebral adrenoleukodystrophy (cALD) remains a devastating neurodegenerative disease; only allogeneic hematopoietic cell transplantation (HCT) has been shown to provide long-term disease stabilization and survival. Sixty boys undergoing HCT for cALD from 2000 to 2009 were analyzed. The median age at HCT was 8.7 years; conditioning regimens and allograft sources varied. At HCT, 50% demonstrated a Loes radiographic severity score ≥ 10, and 62% showed clinical evidence of neurologic dysfunction. A total of 78% (n = 47) are alive at a median 3.7 years after HCT. The estimate of 5-year survival for boys with Loes score < 10 at HCT was 89%, whereas that for boys with Loes score ≥ 10 was 60% (P = .03). The 5-year survival estimate for boys absent of clinical cerebral disease at HCT was 91%, whereas that for boys with neurologic dysfunction was 66% (P = .08). The cumulative incidence of transplantation-related mortality at day 100 was 8%. Post-transplantation progression of neurologic dysfunction depended significantly on the pre-HCT Loes score and clinical neurologic status. We describe the largest single-institution analysis of survival and neurologic function outcomes after HCT in cALD. These trials were registered at www.clinicaltrials.gov as #NCT00176904, #NCT00668564, and #NCT00383448.
authors.affiliation: Department of Pediatrics, Blood and Marrow Transplant Program, University of Minnesota Medical School, Minneapolis, MN 55455, USA. mill4991@umn.edu
authors.forename: Weston P
authors.initials: WP
authors.lastname: Miller
authors.forename: Steven M
authors.initials: SM
authors.lastname: Rothman
authors.forename: David
authors.initials: D
authors.lastname: Nascene
authors.forename: Teresa
authors.initials: T
authors.lastname: Kivisto
authors.forename: Todd E
authors.initials: TE
authors.lastname: DeFor
authors.forename: Richard S
authors.initials: RS
authors.lastname: Ziegler
authors.forename: Julie
authors.initials: J
authors.lastname: Eisengart
authors.forename: Kara
authors.initials: K
authors.lastname: Leiser
authors.forename: Gerald
authors.initials: G
authors.lastname: Raymond
authors.forename: Troy C
authors.initials: TC
authors.lastname: Lund
authors.forename: Jakub
authors.initials: J
authors.lastname: Tolar
authors.forename: Paul J
authors.initials: PJ
authors.lastname: Orchard
mesh_terms: D000293:Adolescent; D000326:Adrenoleukodystrophy / Q000473:pathology / Q000601:surgery* / Q000628:therapy*; D002648:Child; D002675:Child, Preschool; D015331:Cohort Studies; D018450:Disease Progression; D006086:Graft vs Host Disease / Q000209:etiology; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects; D006801:Humans; D007223:Infant; D008297:Male; D016019:Survival Analysis; D019172:Transplantation Conditioning / Q000009:adverse effects; D014184:Transplantation, Homologous; D016896:Treatment Outcome; D055815:Young Adult