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Endocrine Abstracts (2024) 102 163 | DOI: 10.1530/endoabs.102.163

EYES2024 ESE Young Endocrinologists and Scientists (EYES) 2024 Adrenals and Neuroendocrine Tumors (17 abstracts)

Osilodrostat as a safe and effective treatment for cushing disease in adolescent patient

Mari Minasyan 1,


1Endocrinology, Oncological Endocrinology, and Nuclear Medicine Department, University Hospital, Krakow, Poland; 2Chair and Department of Endocrinology, Jagiellonian University Medical College, Krakow, Poland


Osilodrostat as an inhibitor of adrenal 11B-hydroxylas, is an effective medication used in the management of endogenous hypercortisolism. There are limited data regarding usage of osilodrostat in adolescent patients. Case presents a 16-year-old female who was admitted to Endocrinology Department due to suspicion of Cushing Syndrome. Patient’s medical history was significant for newly diagnosed hypertension, diabetes mellitus type 2, menstrual irregularities, insomnia for few months. Physical examination was prominent for obesity (BMI 37 kg/m2), acanthosis nigricans, red striae, buffalo hump, face plethora. Hormonal evaluation indicated ACTH dependent Cushing Syndrome. It showed impaired cortisol circadian rhythm (serum morning cortisol: 15.5 mg/dl, serum midnight cortisol 15 mg/dl), elevated ACTH level (146 pg/ml), elevated late night salivary cortisol (2.7 × ULN), elevated urinary free cortisol (2.8 × ULN). Desmopressin test showed cortisol rise by 25% and ACTH rise by 69%. Pituitary MRI visualized microadenoma. Additional laboratory evaluations showed uncontrolled diabetes mellitus (HBA1C 7.10%), elevated liver enzymes (ALT 7 × ULN, GGTP 3 × ULN, ALP 1.7 ULN). Abdominal USG showed liver steatosis. Densitometry indicated normal bone density. Osilodrostat treatment was introduced with initial daily dosage of 2 mg, followed by titration to 5 mg daily with good clinical tolerance. The dosing was adjusted based on cortisol level in urine, late night cortisol in saliva, patient’s clinical symptoms. During 2 months of treatment with osilodrostat, patient showed biochemical (normalization of late-night saliva cortisol and urinary free cortisol) and clinical control (better control of diabetes mellitus and hypertension, sleep pattern normalization, 4% weight loss). After initial preparation with osilodrostat, patient is planned to undergo transsphenoidal pituitary adenoma removal. Clinical trials of osilodrostat in children and adolescents are lacking. This case describes its potential role in these populations.

Volume 102

ESE Young Endocrinologists and Scientists (EYES) 2024

European Society of Endocrinology 

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