This review explores the evolving landscape of treatments for hypercortisolism, highlighting both established and emerging therapies. Although surgery remains the cornerstone of management, medical therapies play a crucial and expanding role, especially in cases of persistent, recurrent, or severe hypercortisolism. We discuss the effectiveness and limitations of steroidogenesis inhibitors, pituitary-directed drugs, glucocorticoid receptor antagonists, and experimental drugs targeting novel molecular pathways that have been implicated in the pathogenesis of hypercortisolism. Despite advancements, significant unmet needs persist, underscoring the importance of personalized treatment approaches and the development of targeted therapies. Ongoing and future clinical trials are crucial for validating the safety and efficacy of these innovative treatments in Cushing disease management.
New Approaches to the Treatment of Hypercortisolism / Pofi, Riccardo; DE ALCUBIERRE, Dario; Dong, Jiawen; W Tomlinson, Jeremy. - In: ANNUAL REVIEW OF MEDICINE. - ISSN 0066-4219. - (2024). [10.1146/annurev-med-071723-044849]
New Approaches to the Treatment of Hypercortisolism.
Riccardo Pofi
Primo
Conceptualization
;Dario De AlcubierreSecondo
Writing – Original Draft Preparation
;
2024
Abstract
This review explores the evolving landscape of treatments for hypercortisolism, highlighting both established and emerging therapies. Although surgery remains the cornerstone of management, medical therapies play a crucial and expanding role, especially in cases of persistent, recurrent, or severe hypercortisolism. We discuss the effectiveness and limitations of steroidogenesis inhibitors, pituitary-directed drugs, glucocorticoid receptor antagonists, and experimental drugs targeting novel molecular pathways that have been implicated in the pathogenesis of hypercortisolism. Despite advancements, significant unmet needs persist, underscoring the importance of personalized treatment approaches and the development of targeted therapies. Ongoing and future clinical trials are crucial for validating the safety and efficacy of these innovative treatments in Cushing disease management.I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.