Authors:
Jaap Jan Boelens, Mieke Aldenhoven, Duncan Purtill, Annalisa Ruggeri, Todd DeFor, Robert Wynn, Ed 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, and Vanderson Rocha
Summary:
We report transplantation outcomes on 258 children with Hurler syndrome (HS) after a myelo-ablative conditioning regimen from 1995 to 2007. Median age at transplant was 16.7 months and median follow-up was 57 months. The cumulative incidence (CI) of day-60 neutrophil recovery was 91%, day-100 acute-GvHD (grade II-IV), 25%, and 5-year chronic-GvHD, 16%. Overall survival (OS) 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) were similar at 81%, 66% after 10/10 HLA matched unrelated donor (UD) and 68% after 5/6 matched CB. EFS was lower after transplantation of 4/6 matched unrelated CB (57%; p=0.031) and HLA mismatched UD (41%; p=0.007). Full donor chimerism (p=0.039) and normal enzyme levels (p=0.007) were higher after CB transplantation (92% and 98%, respectively) compared to 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 unrelated-CB, 5/6-unrelated-CB and 10/10 MUD. The use of mismatched UD and 4/6 matched-unrelated CB was associated with lower EFS.
Source:
Blood; (03/14/13)