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Medical treatment of functional pituitary adenomas, trials and tribulations

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Abstract

Context

Functioning pituitary adenomas (FPAs) include most frequently prolactinomas, somatotroph or corticotroph adenomas, while thyrotroph and gonadotroph adenomas are very rare. Despite their benign histological nature (aggressive tumors are rare and malignant ones exceptional), FPAs could cause significant morbidity and increased mortality due to complications associated with hormonal excess syndromes and/or mass effect leading to compression of adjacent structures. This mini review will focus on the increasing role of medical therapy in the multimodal treatment, which also includes transsphenoidal surgery (TSS) and radiotherapy.

Evidence synthesis

Most patients with prolactinomas are treated only with medications, but surgery could be considered for some patients in a specialized pituitary center, if higher chances of cure. Dopamine agonists, especially cabergoline, are efficient in reducing tumor size and normalizing prolactin. TSS is the first-line treatment for all other FPAs, but most patients require complex adjuvant treatment, including a combination of therapeutic approaches. Medical therapy is the cornerstone of treatment in all patients after unsuccessful surgery or when surgery cannot be offered and includes somatostatin receptor ligands and dopamine agonists (almost all FPAs), growth hormone receptor antagonists (acromegaly), adrenal steroidogenesis inhibitors and glucocorticoid receptor blockers (Cushing’s disease). Novel medical treatments, especially for acromegaly and Cushing’s disease are under research.

Conclusions

An enlarged panel of effective drugs available with increased knowledge of predictive factors for response and/or adverse effects will enhance the possibility to offer a more individualized treatment. This would not only improve disease control and prognosis, but also quality of life.

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Correspondence to Maria Fleseriu.

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M.F. has received grants to the institution from Crinetics, Ionis, Recordati, Sparrow, and Xeris and occasional consulting fees from Amryt, Camurus, Crinetics, Ipsen, Recordati, Sparrow, and Xeris.

C.C. has received occasional speaker and consulting fees from Pfizer and Ipsen.

FAH has received grants to the institution from HRPharma and Camurus and occasional consulting fees from Recordati, Pfizer, Ipsen and HRPharma.

JMHA has received occasional speaker and consulting fees from Ipsen.

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Capatina, C., Hanzu, F.A., Hinojosa-Amaya, J.M. et al. Medical treatment of functional pituitary adenomas, trials and tribulations. J Neurooncol 168, 197–213 (2024). https://doi.org/10.1007/s11060-024-04670-x

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