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Leucemia linfócitica crónica. diagnóstico e terapêutica: novas abordagens
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A leucemia linfocítica crónica é uma neoplasia hematológica linfo proliferativa que resulta da proliferação e acumulação de linfócitos B monoclonais. É a doença hematológica mais comum nas sociedades ocidentais. Os doentes podem ser divididos em diferentes estadios, determinados com o objetivo de avaliar a extensão da doença, orientar o tratamento e estabelecer um prognóstico. O conhecimento emergente sobre esta doença permitiu a descoberta de novos marcadores de prognóstico, o que possibilita a distinção dos doentes com um curso clínico indolente daqueles que apresentam doença de elevado risco.
Dada a heterogeneidade da leucemia linfocítica crónica, a necessidade de investigação de novos meios de diagnóstico e de alvos terapêuticos tem vindo a ser crescente. Aquando do diagnóstico, a maioria dos doentes apresenta-se assintomática. Devido à inespecificidade, ou ausência de sintomas relacionados com a doença, é necessário realizar o diagnóstico diferencial dos pacientes com base nas características imunofenotípicas das células B e na sua análise morfológica.
Com o surgimento das técnicas moleculares, que permitiram a descoberta das anomalias citogenéticas por detrás da leucemia linfocítica crónica, é possível cada vez mais direcionar os esquemas terapêuticos e desenvolver novos fármacos.
Os doentes com prognóstico mais desfavorável e que possuem indicação para iniciar terapêutica, podem ser tratados com terapêuticas standard, tais como os regimes de quimioimunoterapia. Se apresentarem comorbilidades significativas, outros esquemas menos agressivos têm vindo a ser aplicados, como os inibidores dos recetores das células B. Apesar do sucesso desta terapêutica, muito ainda tem que ser investigado, sobretudo no campo da leucemia refratária ou recidiva. Por isso, novos fármacos como o acalabrutinib e o venetoclax receberam aprovação com base em estudos recentes e muitos outros encontram-se ainda em investigação exaustiva.
Chronic lymphocytic leukemia is a lymphoproliferative hematological neoplasia that results from the proliferation and accumulation of monoclonal B lymphocytes. This is the most common hematological disease in Western societies. Patients can be divided into different stages, determined with the aim of assessing the extent of the disease, guiding treatment and establishing a prognosis. The emerging knowledge allowed the discovery of new prognostic markers, which makes possible to distinguish patients with an indolent clinical course from those who have high risk disease. Relatively to the heterogeneity of this pathology, the need to investigate new means of diagnosis and therapeutic targets has been growing. At diagnosis, most patients are asymptomatic. Due to the non-specificity or absence of symptoms related to the disease, it is necessary to make the differential diagnosis of patients based on the immunophenotypic characteristics of B cells, as well as their morphological analysis. With the emergence of molecular techniques that allowed the discovery of cytogenetic abnormalities behind this pathology, is possible to increase target therapeutic schemes and develop new drugs. Patients with a unfavorable prognosis are indicated to start therapy, they can be treated with standard therapies such as chemoimmunotherapy regimens. However, if they have significant comorbidities, other less aggressive regimens have been applied, such as inhibitors of B cell receptors. Despite the success of this therapy, much remains to be investigated, especially the refractory or relapsing leukemia. Therefore, new drugs such as acalabrutinib and venetoclax have received approval based on recent studies and many others are still under exhaustive research.
Chronic lymphocytic leukemia is a lymphoproliferative hematological neoplasia that results from the proliferation and accumulation of monoclonal B lymphocytes. This is the most common hematological disease in Western societies. Patients can be divided into different stages, determined with the aim of assessing the extent of the disease, guiding treatment and establishing a prognosis. The emerging knowledge allowed the discovery of new prognostic markers, which makes possible to distinguish patients with an indolent clinical course from those who have high risk disease. Relatively to the heterogeneity of this pathology, the need to investigate new means of diagnosis and therapeutic targets has been growing. At diagnosis, most patients are asymptomatic. Due to the non-specificity or absence of symptoms related to the disease, it is necessary to make the differential diagnosis of patients based on the immunophenotypic characteristics of B cells, as well as their morphological analysis. With the emergence of molecular techniques that allowed the discovery of cytogenetic abnormalities behind this pathology, is possible to increase target therapeutic schemes and develop new drugs. Patients with a unfavorable prognosis are indicated to start therapy, they can be treated with standard therapies such as chemoimmunotherapy regimens. However, if they have significant comorbidities, other less aggressive regimens have been applied, such as inhibitors of B cell receptors. Despite the success of this therapy, much remains to be investigated, especially the refractory or relapsing leukemia. Therefore, new drugs such as acalabrutinib and venetoclax have received approval based on recent studies and many others are still under exhaustive research.
Descrição
Trabalho Final de Mestrado Integrado, Ciências Farmacêuticas, 2020, Universidade de Lisboa, Faculdade de Farmácia.
Palavras-chave
Leucemia linfócitica crónica Fisiopatologia Prognóstico Diagnóstico Novos agentes terapêuticos Mestrado integrado - 2020