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- Title
Outcome of children with severe acquired aplastic anemia treated with rabbit antithymocyte globulin and cyclosporine A.
- Authors
Garanito, Marlene Pereira; Aivazoglou Carneiro, Jorge David; Filho, Vicente Odone; Scheinberg, Phillip
- 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 [Cl]: 1.4%-66%) at 5 years. The cumulative incidence of clonal evolution after immunosuppressive therapy was 8.3% (95% Cl: 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% Cl: 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.
- Subjects
APLASTIC anemia treatment; TREATMENT effectiveness; IMMUNOSUPPRESSIVE agents; CYCLOSPORINE; GLOBULINS; THYMOCYTES; DISEASE relapse; ANEMIA in children; THERAPEUTICS
- Publication
Jornal de Pediatria, 2014, Vol 90, Issue 5, p523
- ISSN
0021-7557
- Publication type
Article
- DOI
10.1016/j.jped.2014.02.004