Cushing’s Disease: Long-Term Effectiveness and Safety of Osilodrostat in a Polish Group of Patients with Persistent Hypercortisolemia in the Experience of a Single Center

Author:

Dzialach Lukasz1ORCID,Sobolewska Joanna1ORCID,Respondek Wioleta2,Szamotulska Katarzyna3,Witek Przemysław1

Affiliation:

1. Department of Internal Medicine Endocrinology and Diabetes, Medical University of Warsaw, 03-242 Warsaw, Poland

2. Department of Internal Medicine Endocrinology and Diabetes, Mazovian Brodnowski Hospital, 03-242 Warsaw, Poland

3. Department of Epidemiology and Biostatistics, Institute of Mother and Child, 01-211 Warsaw, Poland

Abstract

Osilodrostat is a potent oral steroidogenesis inhibitor that has emerged as the new medical agent for patients with Cushing’s disease (CD) requiring long-term medical therapy for hypercortisolemia control. Its efficacy and safety have been assessed in clinical trials; however, real-world evidence is still scarce. This study aimed to investigate the long-term treatment (156 weeks) clinical and biochemical effect of osilodrostat in six patients with CD at a single center in Poland, initially participating in the LINC4 study. At week 36, all six patients met the key secondary endpoint of the LINC4 trial, achieving normalization of median urinary free cortisol. Osilodrostat treatment allowed for complete disease control in all patients and none of the patients was excluded due to the lack of treatment effectiveness in 156 weeks of follow-up. All patients demonstrated significant improvement from baseline on most metabolic and cardiovascular parameters, which was most evident at week 36 and sustained throughout the study period. This study supports and strengthens the role of osilodrostat as an effective long-term medical treatment in patients with CD. We also present three patient case histories in detail to highlight the clinical situations that endocrinologists might face during osilodrostat therapy.

Publisher

MDPI AG

Subject

General Biochemistry, Genetics and Molecular Biology,Medicine (miscellaneous)

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