In Hurler Syndrome patients early referral for HCT, with the best available HLA-matched donor offers the best "event free survival".
In Hurler syndrome patients HCT with a well matched unrelated cord blood unit is particularly attractive as the unit is readily available.
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.
- Submitted September 7, 2012.
- Accepted January 25, 2013.
- Copyright © 2005 American Society of Hematology