title: Outcome of children with severe acquired aplastic anemia treated with rabbit antithymocyte globulin and cyclosporine A.
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.
authors.affiliation: 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.
authors.forename: Marlene Pereira
authors.initials: MP
authors.lastname: Garanito
authors.affiliation: Instituto da Criança, Hospital das Clínicas, Faculdade de Medicina, Universidade de São Paulo (USP), São Paulo, SP, Brazil.
authors.forename: Jorge David Aivazoglou
authors.initials: JD
authors.lastname: Carneiro
authors.affiliation: Instituto da Criança, Hospital das Clínicas, Faculdade de Medicina, Universidade de São Paulo (USP), São Paulo, SP, Brazil.
authors.forename: Vicente
authors.initials: V
authors.lastname: Odone Filho
authors.affiliation: Hospital Beneficência Portuguesa, São Paulo, SP, Brazil.
authors.forename: Phillip
authors.initials: P
authors.lastname: Scheinberg
mesh_terms: D000293:Adolescent; D000741:Anemia, Aplastic / Q000401:mortality* / Q000628:therapy; D000818:Animals; D000961:Antilymphocyte Serum / Q000627:therapeutic use*; D001938:Brazil / Q000453:epidemiology; D002648:Child; D002675:Child, Preschool; D060965:Clonal Evolution; D016572:Cyclosporine / Q000627:therapeutic use*; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D007223:Infant; D008297:Male; D011817:Rabbits; D012008:Recurrence; D012189:Retrospective Studies; D015996:Survival Rate; D016896:Treatment Outcome
chemical_list: D000961:Antilymphocyte Serum; D007166:Immunosuppressive Agents; D016572:Cyclosporine
keywords: Anemia aplástica; Anti-thymocyte globulin; Aplastic anemia; Clonal evolution; Evolução clonal; Globulina antitimocítica; Immunosuppressive therapy; Pancitopenia; Pancytopenia; Recorrência; Relapse; Terapia imunossupressora