Outcome of children with severe acquired aplastic anemia treated with rabbit antithymocyte globulin and cyclosporine A
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 wi...
| Main Authors: | , , , |
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| Format: | Article |
| Language: | English |
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Elsevier
2014-09-01
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| Series: | Jornal de Pediatria |
| Online Access: | http://www.sciencedirect.com/science/article/pii/S0021755714000655 |
| _version_ | 1828346736474587136 |
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| author | Marlene Pereira Garanito Jorge David Aivazoglou Carneiro Vicente Odone Filho Phillip Scheinberg |
| author_facet | Marlene Pereira Garanito Jorge David Aivazoglou Carneiro Vicente Odone Filho Phillip Scheinberg |
| author_sort | Marlene Pereira Garanito |
| collection | DOAJ |
| description | 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. Resumo: Objetivo: Avaliar o resultado de crianças com anemia aplástica grave adquirida tratadas com globulina antitimocítica de coelho e ciclosporina como tratamento inicial em nosso instituto. Métodos: Análise retrospectiva de 26 pacientes pediátricos com anemia aplástica tratados entre 1996 e 2011 com globulina antitimocítica de coelho e ciclosporina. Resultados: A taxa de resposta geral em seis meses foi de 34,6% (9/26), e a incidência acumulada de recorrência foi de 26,5% (intervalo de confiança [IC] de 95%,1,4%-66%) em cinco anos. A incidência acumulada de evolução clonal após a terapia imunossupressora foi de 8,3% (IC 95%, 0,001%-53,7%) em cinco anos, com ambas as evoluções clonais em pacientes sem resposta que adquiriram o cariótipo com monossomia 7. A sobrevida geral em cinco anos foi de 73,6% (IC 95%, 49,2%-87,5%). Conclusões: Nossos resultados confirmam a baixa taxa de resposta com globulina antitimocítica de coelho como terapia inicial em pacientes pediátricos, da mesma forma como relatado para pacientes de todas as idades. Essa confirmação é problemática em nosso país devido à falta de globulina antitimocítica de cavalo em muitos mercados fora dos Estados Unidos, incluindo o Brasil. Keywords: Aplastic anemia, Immunosuppressive therapy, Anti-thymocyte globulin, Pancytopenia, Relapse, Clonal evolution, Palavras-chave: Anemia aplástica, Terapia imunossupressora, Globulina antitimocítica, Pancitopenia, Recorrência, Evolução clonal |
| first_indexed | 2024-04-14T00:30:46Z |
| format | Article |
| id | doaj.art-067c80971f134cf7a9721d2fa5364e40 |
| institution | Directory Open Access Journal |
| issn | 0021-7557 |
| language | English |
| last_indexed | 2024-04-14T00:30:46Z |
| publishDate | 2014-09-01 |
| publisher | Elsevier |
| record_format | Article |
| series | Jornal de Pediatria |
| spelling | doaj.art-067c80971f134cf7a9721d2fa5364e402022-12-22T02:22:33ZengElsevierJornal de Pediatria0021-75572014-09-01905523527Outcome of children with severe acquired aplastic anemia treated with rabbit antithymocyte globulin and cyclosporine AMarlene Pereira Garanito0Jorge David Aivazoglou Carneiro1Vicente Odone Filho2Phillip Scheinberg3Instituto da Criança, Hospital das Clínicas, Faculdade de Medicina, Universidade de São Paulo (USP), São Paulo, SP, Brazil; Corresponding author.Instituto da Criança, Hospital das Clínicas, Faculdade de Medicina, Universidade de São Paulo (USP), São Paulo, SP, BrazilInstituto da Criança, Hospital das Clínicas, Faculdade de Medicina, Universidade de São Paulo (USP), São Paulo, SP, BrazilHospital Beneficência Portuguesa, São Paulo, SP, BrazilObjective: 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. Resumo: Objetivo: Avaliar o resultado de crianças com anemia aplástica grave adquirida tratadas com globulina antitimocítica de coelho e ciclosporina como tratamento inicial em nosso instituto. Métodos: Análise retrospectiva de 26 pacientes pediátricos com anemia aplástica tratados entre 1996 e 2011 com globulina antitimocítica de coelho e ciclosporina. Resultados: A taxa de resposta geral em seis meses foi de 34,6% (9/26), e a incidência acumulada de recorrência foi de 26,5% (intervalo de confiança [IC] de 95%,1,4%-66%) em cinco anos. A incidência acumulada de evolução clonal após a terapia imunossupressora foi de 8,3% (IC 95%, 0,001%-53,7%) em cinco anos, com ambas as evoluções clonais em pacientes sem resposta que adquiriram o cariótipo com monossomia 7. A sobrevida geral em cinco anos foi de 73,6% (IC 95%, 49,2%-87,5%). Conclusões: Nossos resultados confirmam a baixa taxa de resposta com globulina antitimocítica de coelho como terapia inicial em pacientes pediátricos, da mesma forma como relatado para pacientes de todas as idades. Essa confirmação é problemática em nosso país devido à falta de globulina antitimocítica de cavalo em muitos mercados fora dos Estados Unidos, incluindo o Brasil. Keywords: Aplastic anemia, Immunosuppressive therapy, Anti-thymocyte globulin, Pancytopenia, Relapse, Clonal evolution, Palavras-chave: Anemia aplástica, Terapia imunossupressora, Globulina antitimocítica, Pancitopenia, Recorrência, Evolução clonalhttp://www.sciencedirect.com/science/article/pii/S0021755714000655 |
| spellingShingle | Marlene Pereira Garanito Jorge David Aivazoglou Carneiro Vicente Odone Filho Phillip Scheinberg Outcome of children with severe acquired aplastic anemia treated with rabbit antithymocyte globulin and cyclosporine A Jornal de Pediatria |
| title | Outcome of children with severe acquired aplastic anemia treated with rabbit antithymocyte globulin and cyclosporine A |
| title_full | Outcome of children with severe acquired aplastic anemia treated with rabbit antithymocyte globulin and cyclosporine A |
| title_fullStr | Outcome of children with severe acquired aplastic anemia treated with rabbit antithymocyte globulin and cyclosporine A |
| title_full_unstemmed | Outcome of children with severe acquired aplastic anemia treated with rabbit antithymocyte globulin and cyclosporine A |
| title_short | Outcome of children with severe acquired aplastic anemia treated with rabbit antithymocyte globulin and cyclosporine A |
| title_sort | outcome of children with severe acquired aplastic anemia treated with rabbit antithymocyte globulin and cyclosporine a |
| url | http://www.sciencedirect.com/science/article/pii/S0021755714000655 |
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