Novas terapias para a ptta (púrpura trombocitopênica trombótica adquirida)
Data
2024-08-20
Autores
Yama, Juliana Sayuri Ishikawa 
Orientadores
Fonseca, Fernando Luiz Affonso
Tipo
Trabalho de conclusão de curso
Título da Revista
ISSN da Revista
Título de Volume
Resumo
A Púrpura Trombocitopênica Trombótica (PTT) é uma doença rara e fatal, se não for diagnosticada e tratada precocemente. É caracterizada pela presença de multímeros do fator de von Willebrand, ocasionando seu acúmulo e, consequentemente, a agregação plaquetária. É ocasionada devido à deficiência em uma enzima sintetizada no fígado, a ADAMTS13. A literatura possui um amplo consenso acerca da urgência de um reconhecimento breve ou imediato para obter sucesso no tratamento da doença. Este trabalho teve como objetivo apresentar os avanços alcançados com os tratamentos para a púrpura trombocitopênica trombótica adquirida no período de 2016 a 2024 e avaliar as vantagens e possíveis desvantagens da sua utilização. Uma revisão sistemática de artigos científicos resultou em 14 artigos encontrados em inglês que descreveu dois novos medicamentos: o rituximabe e o caplacizumabe. Os estudos avaliaram a eficácia, segurança e protocolo de utilização destes medicamentos no tratamento da doença e ambos apresentaram boa performance tanto para o tratamento, quanto para redução de tempo de internação e refratoriedade. Bem como o custo benefício da utilização destes medicamentos.
Thrombotic Thrombocytopenic Purpura (TTP) is a rare and fatal disease and is not detected and treated early. It is characteristic of the presence of multimers of von Willebrand factor, causing its accumulation and, consequently, platelet aggregation. And it is caused due to deficiency in an enzyme synthesized in the liver, with a motif of thrombospondin type 1 member 13 (ADAMTS13). The literature has a broad consensus on the urgency of brief or immediate recognition to achieve successful treatment of the disease. This work aimed to present the improved advances with treatments for acquired thrombotic thrombocytopenic purpura in the period from 2016 to 2024 and evaluate the advantages and possibilities of their use. A systematic review of scientific articles searched for 14 articles found in English that describe two new drugs: rituximab and caplacizumab. The studies evaluated the efficacy, safety and protocol for using medications in the treatment of the disease and both evaluated good performance both for treatment and for reducing hospitalization time and refractoriness. Additionally, the cost-effectiveness of using these medications was assessed.
Thrombotic Thrombocytopenic Purpura (TTP) is a rare and fatal disease and is not detected and treated early. It is characteristic of the presence of multimers of von Willebrand factor, causing its accumulation and, consequently, platelet aggregation. And it is caused due to deficiency in an enzyme synthesized in the liver, with a motif of thrombospondin type 1 member 13 (ADAMTS13). The literature has a broad consensus on the urgency of brief or immediate recognition to achieve successful treatment of the disease. This work aimed to present the improved advances with treatments for acquired thrombotic thrombocytopenic purpura in the period from 2016 to 2024 and evaluate the advantages and possibilities of their use. A systematic review of scientific articles searched for 14 articles found in English that describe two new drugs: rituximab and caplacizumab. The studies evaluated the efficacy, safety and protocol for using medications in the treatment of the disease and both evaluated good performance both for treatment and for reducing hospitalization time and refractoriness. Additionally, the cost-effectiveness of using these medications was assessed.