TY - JOUR
T1 - Outcomes of transplantation using various hematopoietic cell sources in children with Hurler syndrome after myeloablative conditioning
AU - Boelens, Jaap Jan
AU - Aldenhoven, Mieke
AU - Purtill, Duncan
AU - Ruggeri, Annalisa
AU - Defor, Todd
AU - Wynn, Robert
AU - Wraith, E.
AU - Cavazzana-Calvo, Marina
AU - Rovelli, Attilio
AU - Fischer, Alain
AU - Tolar, Jakub
AU - Prasad, Vinod K.
AU - Escolar, Maria
AU - Gluckman, Eliane
AU - O'Meara, Anne
AU - Orchard, Paul J.
AU - Veys, Paul
AU - Eapen, Mary
AU - Kurtzberg, Joanne
AU - Rocha, Vanderson
AU - Eurocord,
AU - Inborn Errors Working Party of European Blood, Errors Working Party of European Blood
AU - Duke University Blood, University Blood
AU - Centre for International Blood, for International Blood
N1 - Publisher Copyright:
© 2013 by The American Society of Hematology.
PY - 2013/5/9
Y1 - 2013/5/9
N2 - 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.
AB - 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.
UR - http://www.scopus.com/inward/record.url?scp=84880314796&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=84880314796&partnerID=8YFLogxK
U2 - 10.1182/blood-2012-09-455238
DO - 10.1182/blood-2012-09-455238
M3 - Article
C2 - 23493783
AN - SCOPUS:84880314796
SN - 0006-4971
VL - 121
SP - 3981
EP - 3987
JO - Blood
JF - Blood
IS - 19
ER -