Long-Term Outcome of Pediatric Patients with Severe Aplastic Anemia Treated with Antithymocyte Globulin and Cyclosporine

Objective To determine the long-term outcomes in children with severe aplastic anemia (SAA) treated with antithymocyte globulin (ATG) and cyclosporine (CsA) through a retrospective analysis of the pediatric patients treated at our institution in all protocols that included horse ATG (h-ATG) and CsA. Study design Between 1989 and 2006, a total of 406 patients. 20% of whom were children under age 18 years, received an initial course of immunosuppressive therapy (IST) at our institution. Here we report the outcome of 77 children who were treated with all h-ATG plus CsA-based regimen during this period. Results The overall response rate at 6 months was 74% (57/77); the cumulative incidence. of relapse at 10 years was 33%. and the median time to relapse was 558 days. The cumulative incidence of evolution after IST was 8.5%; all 3 such events occurred in partial responders. Overall, there were 13 deaths (17%). with 4 occurring within the 3 months after IST in patients who had a pretreatment absolute neutrophil count of < 100/mu L and the other 9 occurring more than 6 months after initiation of IST. The median time to death was 570 days. The overall 10-year survival for the entire cohort was 80%; long-term survival in the children who responded to IST was 89%. Conclusions The long-term survival in pediatric patients who respond to IST is excellent, at about 90%. IST remains a good alternative in pediatric patients who lack an HLA-matched sibling donor and should be offered as initial therapy before possible hematopoietic stem cell transplantation from an unrelated donor. (J Pediatr 2008;153:814-9)