TY - JOUR
T1 - Long-Term Outcomes of Hematopoietic Stem Cell Transplantation for Severe Treatment-Resistant Autoimmune Cytopenia in Children
AU - Rabusin, Marco
AU - Snowden, J. A.
AU - Veys, Paul
AU - Quartier, Pierre
AU - Dalle, Jean Hughes
AU - Dhooge, Catharina
AU - Di Bartolomeo, Paolo
AU - Gonzalez-Vicent, Maria
AU - Gibson, Brenda
AU - Iriondo, Arturo
AU - Juergens, Herbert
AU - Lisukov, Igor
AU - Messina, Chiara
AU - Mialou, Valerie
AU - Steward, Colin Graham
AU - Urban, Christian
AU - Renard, Marleen
AU - Giurici, Nagua
AU - Peters, Christina
AU - Badoglio, Manuela
AU - Ronfani, Luca
AU - Dini, Giorgio
AU - Farge, Dominique
AU - Saccardi, Riccardo
PY - 2013/4
Y1 - 2013/4
N2 - We analyzed the long-term outcomes of pediatric patients registered in the European Group for Blood and Marrow Transplantation database who underwent hematopoietic stem cell transplantation (HSCT) for severe treatment refractory autoimmune cytopenia. With a median follow-up of 100 months, event-free survival was 54% overall, with no significant difference between allogeneic HSCT (n = 15) and autologous HSCT (n = 7) recipients (58% versus 42%; P = .50). Despite a trend toward failure of response or relapse after autologous HSCT compared with allogeneic HSCT, the difference was not significant (43% versus 13%; P = .30). Treatment-related mortality was high in both HSCT groups (29% and 16%; P = .09). Based on the limited numbers of subjects in this retrospective analysis, both allogeneic and autologous HSCT may induce complete and persistent responses in approximately one-half of pediatric patients with severe refractory autoimmune cytopenia, although treatment-related toxicity is high.
AB - We analyzed the long-term outcomes of pediatric patients registered in the European Group for Blood and Marrow Transplantation database who underwent hematopoietic stem cell transplantation (HSCT) for severe treatment refractory autoimmune cytopenia. With a median follow-up of 100 months, event-free survival was 54% overall, with no significant difference between allogeneic HSCT (n = 15) and autologous HSCT (n = 7) recipients (58% versus 42%; P = .50). Despite a trend toward failure of response or relapse after autologous HSCT compared with allogeneic HSCT, the difference was not significant (43% versus 13%; P = .30). Treatment-related mortality was high in both HSCT groups (29% and 16%; P = .09). Based on the limited numbers of subjects in this retrospective analysis, both allogeneic and autologous HSCT may induce complete and persistent responses in approximately one-half of pediatric patients with severe refractory autoimmune cytopenia, although treatment-related toxicity is high.
KW - Allogeneic transplant
KW - Childhood
KW - Evans syndrome
KW - HSCT
KW - Refractory cytopenia
UR - http://www.scopus.com/inward/record.url?scp=84875514124&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=84875514124&partnerID=8YFLogxK
U2 - 10.1016/j.bbmt.2012.12.008
DO - 10.1016/j.bbmt.2012.12.008
M3 - Article
C2 - 23253561
AN - SCOPUS:84875514124
VL - 19
SP - 666
EP - 669
JO - Biology of Blood and Marrow Transplantation
JF - Biology of Blood and Marrow Transplantation
SN - 1083-8791
IS - 4
ER -