Navigating the Present, Shaping the Future
Mônica R Gadelha, Luiz Eduardo Wildemberg, Nelma Veronica Marques, Leandro Kasuki
Endocrine Reviews, First published on July 11, 2025, bnaf020
https://doi.org/10.1210/endrev/bnaf020
Acromegaly is a chronic systemic disease associated with considerable morbidity due to its many complications, which lead to increased mortality rates. Adequate treatment is essential to reduce the development of long-term complications and to restore mortality rates to those of the general population. Three modalities of treatment are currently available (surgery, medical therapy, and radiotherapy). Surgery is considered the first-line treatment and achieves disease cure in approximately 50% of patients in reference centers. Three drug classes are currently available (somatostatin receptor ligands, dopamine agonists, and growth hormone receptor antagonists); however, disease control is not achieved in a considerable proportion of patients (∼40%). Despite substantial advances in medical therapy over the last few decades, the treatment burden is still high, and quality of life is not fully restored in many patients, even when biochemical control is achieved. Therefore, the development of medications with greater efficacy and/or easier and more convenient administration is an unmet need in the treatment of acromegaly. In addition, shifting from the current trial-and-error approach to a more precise treatment strategy guided by biomarkers predictive of the response to different medical therapies will optimize and improve patient outcomes. In this review, current treatments as well as new drugs in different phases of development are detailed, and the role of precision medicine in the treatment of acromegaly is discussed.
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