Physician Perceptions on the Treatment and Health-Related Quality of Life Burden of Endogenous Cushing's Syndrome


Page Wilson G1, Oak B2, Silber A2, Meyer J3, O'Hara M2, Geer EB4
1Columbia University Irving Medical Center, New York, NY, USA, 2Trinity Life Sciences, Waltham, MA, USA, 3Xeris Pharmaceuticals, Inc, Chicago, IL, USA, 4Memorial Sloan Kettering Cancer Center, New York, NY, USA

OBJECTIVES: Endogenous Cushing’s syndrome (CS) results from chronic hypercortisolism and is associated with symptoms and complications that adversely impact morbidity. Research indicates that a health-related quality of life (HRQoL) burden persists despite treatment. This study evaluated physician perceptions on treatments and their rationale behind treatment decisions.

METHODS: A cross-sectional, web-enabled, 45-minute questionnaire was used to collect data from qualified physicians who were board-certified or board-eligible in endocrinology in the US and in-practice for 3+ years post-residency. Physicians were asked about perceptions of endogenous CS treatments, disease burden, and attributes associated with treatments.

RESULTS: Sixty-nine physicians were recruited. Respondents were in practice for a mean of 17±7.6 (SD) years and managed a median (IQR) of 20 (6-40) endogenous CS patients during the past 6 months. Regarding treatment perceptions, when evaluating treatments for endogenous CS, HRQoL improvement was rated as the most important attribute (7.8±1.2) on a scale from 1 (Not at all important) to 9 (Extremely important). Physicians were more satisfied with surgical intervention efficacy (7.2±1.6) and safety (6.3±1.4), than pharmacotherapy efficacy (5.9±1.6) and safety (5.9±1.4). The most important attributes for prescribing therapy were efficacy post-surgery (4.0±1.2) and potential use as a combination therapy (3.7±1.2) on a scale from 1 (least important) to 5 (most important). Most (83%) agreed that CS patients “can have reduced ability to function at work/school due to their condition” and 79% completely agreed that patients “feel the impact of their condition every day”.

CONCLUSIONS: Physicians recognize that endogenous CS patients suffer from a debilitating condition with a high HRQoL burden. Overall, improvement in HRQoL was the key treatment attribute influencing treatment choices. This study provides valuable information on the physician perspective of unmet needs and treatment goals for CS patients. Future research is needed to develop a treatment paradigm that alleviates disease burden in endogenous CS.

Conference/Value in Health Info

2022-11, ISPOR Europe 2022, Vienna, Austria

Value in Health, Volume 25, Issue 12S (December 2022)




SDC: Rare & Orphan Diseases

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