G. Page-Wilsona (Dr), B. Oakb (Dr), A. Silberb (Ms), J. Meyerc (Mr), M. O'Harab (Mr), E. Geer*d (Dr)

a Columbia University Irving Medical Center , New York City, UNITED STATES ; b Trinity Life Sciences, Waltham, UNITED STATES ; c Xeris Pharmaceuticals, Inc., Chicago, UNITED STATES ; d Memorial Sloan Kettering Cancer Center - Multidisciplinary Pituitary and Skull Base Tumor Center, New York City, UNITED STATES

* geere@mskcc.org

Objectives: Endogenous Cushing’s syndrome (CS) is characterized by chronic cortisol overproduction. The objective of this study was to evaluate the overall burden of endogenous CS as experienced by treated patients.

Methods: This study included a retrospective review of medical data from treated patients with a confirmed diagnosis of endogenous CS, abstracted by board-certified or board-eligible endocrinologists in practice for 3+ years post-residency in the US. A cross-sectional survey of the endocrinologists managing these patients was conducted concomitantly. The survey included a web-enabled questionnaire to capture physician-level data and a case report form capturing patient medical chart data. Results are reported as mean ± standard deviation unless otherwise noted.

Results: Sixty-nine physicians, who each saw a median (interquartile range) of 20 (6-40) endogenous CS patients during the past 6 months, abstracted data from 273 unique charts. Mean patient age was 46.5±13.4 years, with a 60:40 (female: male) gender split. The mean duration of endogenous CS amongst patients was 4.1 years. The majority (66%) of patients received pharmacotherapy, with or without prior surgery. The most frequently reported symptoms at diagnosis were fatigue (47%), weight gain (37%), acne (36%), and muscle weakness (32%); the occurrence of almost all symptoms declined post-treatment, but none were eliminated (37%, 19%, 36%, 26% respectively). Overall, 79% of patients continued to experience 1 to 6 symptoms, and 21% experienced 7 symptoms post-treatment. Patients required 1.0±1.4 hospitalizations, on average, annually for the treatment of or due to complications resulting from endogenous CS. The mean inpatient length of stay was 4.3±3.1 days per hospitalization incident. Patients required a mean of 4.3±6.3 outpatient visits or consultations annually due to complications resulting from endogenous CS.

Conclusion: Patients with endogenous CS have a high symptomatic burden at diagnosis and continue to experience symptoms after receiving treatment. The clinical burden of endogenous CS results in a direct economic burden in the form of healthcare resource utilization. Further research should focus on addressing the long-term burden CS patients experience despite treatment.

Funding: This study was funded by Strongbridge Biopharma plc, a wholly owned subsidiary of Xeris BioPharma Holdings, Inc.

The author has declared the following conflict(s) of interest:

This study was funded by Strongbridge Biopharma plc, a wholly owned subsidiary of Xeris BioPharma Holdings, Inc.