Marlene Pereira Garanito1, Jorge David Aivazoglou Carneiro2, Vicente Odone Filho2, Phillip Scheinberg3. 1. Instituto da Criança, Hospital das Clínicas, Faculdade de Medicina, Universidade de São Paulo (USP), São Paulo, SP, Brazil. Electronic address: marlene.garanito@hc.fm.usp.br. 2. Instituto da Criança, Hospital das Clínicas, Faculdade de Medicina, Universidade de São Paulo (USP), São Paulo, SP, Brazil. 3. Hospital Beneficência Portuguesa, São Paulo, SP, Brazil.
Abstract
OBJECTIVE: To evaluate the outcome of children with severe acquired aplastic anemia treated with rabbit antithymocyte globulin and cyclosporine as first-line treatment at this institution. METHODS: Retrospective analysis of 26 pediatric patients with aplastic anemia, treated between 1996 and 2011 with rabbit antithymocyte globulin plus cyclosporine. RESULTS: The overall response rate at six months was 34.6% (9/26), and the cumulative incidence of relapse was 26.5% (95% confidence interval [CI]: 1.4%-66%) at 5 years. The cumulative incidence of clonal evolution after immunosuppressive therapy was 8.3% (95% CI: 0.001%-53.7%) at five years with both clonal evolutions in non -responders who acquired monosomy 7 karyotype. The overall survival at five years was 73.6% (95% CI: 49.2%-87.5%). CONCLUSIONS: The present results confirm the poor response rate with rabbit antithymocyte globulin as first therapy in pediatrics patients, similar to what has been reported for patients of all ages. This confirmation is problematic in Brazil, given the lack of horse antithymocyte globulin in many markets outside the United States.
OBJECTIVE: To evaluate the outcome of children with severe acquired aplastic anemia treated with rabbit antithymocyte globulin and cyclosporine as first-line treatment at this institution. METHODS: Retrospective analysis of 26 pediatric patients with aplastic anemia, treated between 1996 and 2011 with rabbit antithymocyte globulin plus cyclosporine. RESULTS: The overall response rate at six months was 34.6% (9/26), and the cumulative incidence of relapse was 26.5% (95% confidence interval [CI]: 1.4%-66%) at 5 years. The cumulative incidence of clonal evolution after immunosuppressive therapy was 8.3% (95% CI: 0.001%-53.7%) at five years with both clonal evolutions in non -responders who acquired monosomy 7 karyotype. The overall survival at five years was 73.6% (95% CI: 49.2%-87.5%). CONCLUSIONS: The present results confirm the poor response rate with rabbit antithymocyte globulin as first therapy in pediatrics patients, similar to what has been reported for patients of all ages. This confirmation is problematic in Brazil, given the lack of horse antithymocyte globulin in many markets outside the United States.
Authors: Zora R Rogers; Taizo A Nakano; Timothy S Olson; Alison A Bertuch; Winfred Wang; Alfred Gillio; Thomas D Coates; Anjulika Chawla; Paul Castillo; Peter Kurre; Christopher Gamper; Carolyn M Bennett; Sarita Joshi; Amy E Geddis; Jessica Boklan; Grzegorz Nalepa; Jennifer A Rothman; James N Huang; Gary M Kupfer; Michaela Cada; Bertil Glader; Kelly J Walkovich; Alexis A Thompson; Rabi Hanna; Adrianna Vlachos; Maggie Malsch; Edie A Weller; David A Williams; Akiko Shimamura Journal: Haematologica Date: 2019-04-04 Impact factor: 9.941