Hematopoietic stem cell transplantation for children with high risk acutelymphoblastic leukemia in first complete remission: a report from theAIEOP registry.

Children with high-risk acute lymphoblastic leukemia in first complete remission can benefit from allogeneic hematopoietic stem cell transplantation. We analyzed the outcome of 211 children with high-risk acute lymphoblastic leukemia in first complete remission given allogeneic transplant from 1990 to 2008; outcome of patients who, despite having the indication to transplantation and a suitable donor, did not receive the allograft for different reasons in the same time period was not analyzed. Sixty-nine patients (33%) were transplanted between 1990 and 1999, 58 (27%) between 2000 and 2005, and 84 (40%) between 2005 and 2008. A matched family donor was employed in 138 patients (65%) and an unrelated donor in 73 (35%). The 10-year probability of overall and disease-free survival was 63.4% and 61%, respectively. The 10-year cumulative incidence of transplantation-related mortality and relapse was 15% and 24%, respectively. After 1999, no differences in terms of both disease-free survival and transplantation-related mortality were observed in patients transplanted from unrelated or matched family donor. In multivariate analysis, grade IV acute graft-versus-host disease was an independent factor associated with worse disease-free survival. By contrast, grade I acute graft-versus-host disease and age at diagnosis between 1 and 9 years were favorable prognostic variables. Our study, not intended to evaluate whether transplantation is superior to chemotherapy for children with acute lymphoblastic leukemia in first complete remission and high-risk features, shows that the allograft cured more than 60% of these patients; in the most recent period, the outcome of matched family and unrelated donor recipients is comparable.