Outcomes of transplantation using various hematopoietic cell sources in children with Hurler syndrome after myeloablative conditioning

• Published in: Blood Vol. 121; no. 19; pp. 3981 - 3987
• Main Authors: Boelens, Jaap Jan, Aldenhoven, Mieke, Purtill, Duncan, Ruggeri, Annalisa, DeFor, Todd, Wynn, Robert, Wraith, Ed, Cavazzana-Calvo, Marina, Rovelli, Attilio, Fischer, Alain, Tolar, Jakub, Prasad, Vinod K., Escolar, Maria, Gluckman, Eliane, O'Meara, Anne, Orchard, Paul J., Veys, Paul, Eapen, Mary, Kurtzberg, Joanne, Rocha, Vanderson
• Format: Journal Article
• Language: English
• Published: United States Elsevier Inc 09.05.2013; American Society of Hematology
• Subjects: Adolescent; Child; Child, Preschool; Female; Hematopoietic Stem Cell Transplantation - adverse effects; Hematopoietic Stem Cell Transplantation - methods; Hematopoietic Stem Cells - cytology; Histocompatibility Testing; Humans; Infant; Male; Mucopolysaccharidosis I - epidemiology; Mucopolysaccharidosis I - mortality; Mucopolysaccharidosis I - therapy; Myeloablative Agonists - adverse effects; Myeloablative Agonists - therapeutic use; Retrospective Studies; Tissue and Organ Procurement - methods; Tissue Donors; Transplantation; Transplantation Conditioning - adverse effects; Transplantation Conditioning - methods; Treatment Outcome; Young Adult
• ISSN: 0006-4971; 1528-0020; 1528-0020
• DOI: 10.1182/blood-2012-09-455238

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. •In HS, patients' early referral for HCT, with the best available HLA-matched donor offers the best event-free survival.•In HS, patients' HCT with a well-matched unrelated cord blood unit is particularly attractive because the unit is readily available.