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Publication
Osilodrostat no tratamento de síndrome de Cushing
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Abstract(s)
O osilodrostat é um potente inibidor da enzima 11ß-hidroxilase (CYP11B1), que desempenha
um papel importante na síntese de cortisol. Este é utilizado no tratamento da síndrome de
Cushing endógena, uma condição rara caraterizada pela produção excessiva de cortisol,
maioritariamente causada por tumores na hipófise ou nas glândulas suprarrenais.
Este fármaco foi aprovado em 2020 pela FDA (Administração de Alimentos e Medicamentos
dos Estados Unidos da América) e pela EMA (Agência Europeia de Medicamentos) para o
tratamento da síndrome de Cushing, como uma opção terapêutica para pacientes adultos que
não são elegíveis para cirurgia ou que apresentam recidiva após o tratamento cirúrgico.
A terapia mostrou uma redução rápida e sustentada na produção de cortisol, com a maioria
dos pacientes mantendo os níveis controlados a longo prazo. Além disso, apresenta um perfil
de segurança aceitável, apesar de potenciais efeitos colaterais, como insuficiência suprarrenal,
hipertensão arterial e prolongamento do intervalo QT, que requerem uma monitorização
contínua durante o tratamento.
Osilodrostat tem se mostrado uma opção eficaz para pacientes com síndrome de Cushing que
não podem realizar ou não obtiveram sucesso com a cirurgia, proporcionando uma nova
abordagem terapêutica para o controlo dos níveis de cortisol.
Osilodrostat is a potent inhibitor of the 11ß-hydroxylase enzyme (CYP11B1), which plays an important role in cortisol synthesis. It is used in the treatment of endogenous Cushing's syndrome, a rare condition characterized by excessive cortisol production, mostly caused by tumors in the pituitary or adrenal glands. This drug was approved in 2020 by the FDA (U.S. Food and Drug Administration) and the EMA (European Medicines Agency) for the treatment of Cushing's syndrome as a therapeutic option for adult patients who are not eligible for surgery or who have experienced a recurrence after surgical treatment. The therapy has shown a rapid and sustained reduction in cortisol production, with most patients maintaining controlled levels in the long term. Moreover, it presents an acceptable safety profile despite potential side effects, such as adrenal insufficiency, hypertension, and QT interval prolongation, which require continuous monitoring during treatment. Osilodrostat has proven to be an effective option for patients with Cushing's syndrome who cannot undergo or have not succeeded with surgery, providing a new therapeutic approach to control cortisol levels.
Osilodrostat is a potent inhibitor of the 11ß-hydroxylase enzyme (CYP11B1), which plays an important role in cortisol synthesis. It is used in the treatment of endogenous Cushing's syndrome, a rare condition characterized by excessive cortisol production, mostly caused by tumors in the pituitary or adrenal glands. This drug was approved in 2020 by the FDA (U.S. Food and Drug Administration) and the EMA (European Medicines Agency) for the treatment of Cushing's syndrome as a therapeutic option for adult patients who are not eligible for surgery or who have experienced a recurrence after surgical treatment. The therapy has shown a rapid and sustained reduction in cortisol production, with most patients maintaining controlled levels in the long term. Moreover, it presents an acceptable safety profile despite potential side effects, such as adrenal insufficiency, hypertension, and QT interval prolongation, which require continuous monitoring during treatment. Osilodrostat has proven to be an effective option for patients with Cushing's syndrome who cannot undergo or have not succeeded with surgery, providing a new therapeutic approach to control cortisol levels.
Description
Keywords
Cortisol Inibição da 11ß-
Hidroxilase Osilodrostat Síndrome de Cushing Tratamento Farmacológico