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

Jaap Jan Boelens, Mieke Aldenhoven, Duncan Purtill, Annalisa Ruggeri, Todd Defor, Robert Wynn, E. Wraith, Marina Cavazzana-Calvo, Attilio Rovelli, Alain Fischer, Jakub Tolar, Vinod K. Prasad, Maria Escolar, Eliane Gluckman, Anne O'Meara, Paul J. Orchard, Paul Veys, Mary Eapen, Joanne Kurtzberg, Vanderson RochaEurocord, Errors Working Party of European Blood Inborn Errors Working Party of European Blood, University Blood Duke University Blood, for International Blood Centre for International Blood

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125 Scopus citations

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 5 .031) and HLA-mismatched UD (41%; P 5 .007). Full-donor chimerism (P 5 .039) and normal enzyme levels (P 5 .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.

Original languageEnglish (US)
Pages (from-to)3981-3987
Number of pages7
JournalBlood
Volume121
Issue number19
DOIs
StatePublished - May 9 2013

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