Outcomes of transplantation using various hematopoietic cell sources in children with Hurler syndrome after myeloablative conditioning.
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.
Boelens, JJ; Aldenhoven, M; Purtill, D; Ruggeri, A; Defor, T; Wynn, R; Wraith, E; Cavazzana-Calvo, M; Rovelli, A; Fischer, A; Tolar, J; Prasad, VK; Escolar, M; Gluckman, E; O'Meara, A; Orchard, PJ; Veys, P; Eapen, M; Kurtzberg, J; Rocha, V; Eurocord, ; Inborn Errors Working Party of European Blood and Marrow Transplant group, ; Duke University Blood and Marrow Transplantation Program, ; Centre for International Blood and Marrow Research,
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