Clinical characteristics of clonal evolution after immunosuppressive therapy in children with severe/very severe aplastic anemia / 中国当代儿科杂志
Chinese Journal of Contemporary Pediatrics
; (12): 27-33, 2017.
Artigo
em Chinês
| WPRIM (Pacífico Ocidental)
| ID: wpr-351407
Biblioteca responsável:
WPRO
ABSTRACT
<p><b>OBJECTIVE</b>To evaluate the clinical characteristics and risk factors of clonal evolution after immunosuppressive therapy (IST) in children with severe/very severe aplastic anemia (SAA/VSAA).</p><p><b>METHODS</b>The clinical data of 231 children with newly-diagnosed SAA/VSAA who received IST were retrospectively studied. The incidence and risk factors of clonal evolution after IST were analyzed.</p><p><b>RESULTS</b>The 5-year overall survival rate of the 231 patients was 82.7%. Except for 18 cases of early deaths, 213 patients were evaluated for IST efficacy. Among the 231 patients, cytogenetic abnormalities for at least two chromosome metaphase were detectable in 14 (7.4%) patients, and PNH clones were detectable in either peripheral red blood cells or neutrophils for 95 patients. Among the 213 patients evaluated for IST efficacy, 15 patients experienced clonal evolution after IST. Five patients had PNH and trisomy 8 which were defined as favorable progressions, and ten patients experienced monosomy 7 and MDS/AML as unfavorable progressions. The 5-year accumulative incidence of favorable and unfavorable progression were (2.2±2.2)% and (4.8±3.3)%, respectively. Until the last follow-up, 100% (5/5) of patients with favorable progressions and 50% (5/10) of patients with unfavorable progressions survived. WBC>3.5×10/L, CD3T cell percentage>80%, dosage of antithymocyte globulin >3.0 mg/(kg·d) and no response to IST were related to unfavorable progressions by univariate analysis. Cox multivariate analysis revealed that an increased CD3T cell percentage (>80%) and no response to IST were independent risk factors for unfavorable progressions.</p><p><b>CONCLUSIONS</b>The children with SAA/VSAA who have an increased CD3T cell percentage at diagnosis or have no response to IST are in high risks of unfavorable progressions.</p>
Texto completo:
Disponível
Contexto em Saúde:
ODS3 - Saúde e Bem-Estar
/
ODS3 - Meta 3.4 Reduzir as mortes prematuras devido doenças não transmissíveis
Problema de saúde:
Meta 3.2: Reduzir as mortes de recém nascidos e crianças com menos de 5 anos
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Anomalias Congênitas e Cromossômicas
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Outras Doenças Sanguíneas
Base de dados:
WPRIM (Pacífico Ocidental)
Assunto principal:
Modelos de Riscos Proporcionais
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Estudos Retrospectivos
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Mortalidade
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Aberrações Cromossômicas
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Usos Terapêuticos
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Tratamento Farmacológico
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Alergia e Imunologia
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Evolução Clonal
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Genética
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Imunossupressores
Tipo de estudo:
Estudo observacional
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Estudo prognóstico
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Fatores de risco
Limite:
Adolescente
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Criança
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Criança, pré-escolar
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Feminino
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Humanos
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Lactente
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Masculino
Idioma:
Chinês
Revista:
Chinese Journal of Contemporary Pediatrics
Ano de publicação:
2017
Tipo de documento:
Artigo