Patient-Reported Burden of Illness in Endogenous Cushing’s Syndrome

Capture the burden of illness and health-related quality of life (HRQoL) associated with endogenous Cushing’s syndrome (CS) using validated patient reported outcome (PRO) measures.

Methods:

A cross-sectional survey of patients with confirmed endogenous CS was conducted in 2021. Patients were ≥21 years old and had received ≥1 pharmacotherapy for the treatment of their endogenous CS prior to study enrollment. Data were collected via a web-enabled instrument including a screener, questionnaire, and five validated PRO measures: CushingQoL, Pain Visual Analog Scale (VAS), Brief Fatigue Inventory (BFI), PROMIS Sleep Disturbance, and PROMIS Anxiety SF 8a. Data are reported as mean ± standard deviation (SD) unless otherwise noted.

Results:

Of the 55 patients surveyed, the majority were female (85%). The average age was 43.4±12.3 years; mean body mass index was 36.9±6.4 kg/m2. The majority (80%) of the cohort had received surgical treatment for CS. Patients reported a mean lag time of 9.9 years between the first occurrence of CS symptoms and diagnosis. Weight gain was the most frequently reported symptom at three unique timepoints including first occurrence of symptoms (76%), time of diagnosis (75%) and time of the survey (42%). Muscle fatigue and weakness together were also frequently reported at these time points (49%, 44%, and 27% respectively). From first occurrence to the time of the survey, most symptoms improved with treatment, but did not fully resolve. At the time of the survey, patients reported that CS had a moderate impact on their activities of daily living (1=no impact to 7=very significant impact), with sexual activity level (5.1±1.4) and self-confidence (4.9±1.7) being the most impacted. CushingQoL scores demonstrated moderate HRQoL impairment (55.8±20.4) on a scale of 0=worst to 100=best HRQoL. Mild pain intensity was reported on the Pain VAS (3.6±1.9) (scale of 0=no pain to 10=severe pain), with 89% of patients taking over-the-counter pain medications. Both sleep disturbance and anxiety were slightly worse in the study cohort than the US general population, as reported on the two PROMIS scales. Additionally, 70% of patients had moderate-severe levels of fatigue as reported on the BFI.

Discussion/Conclusion:

The patient-reported burden of illness associated with endogenous CS is multi-factorial and includes inadequate symptom control despite treatment, as demonstrated by the impact of CS on HRQoL. Diagnostic delays and treatments that do not sufficiently address patient symptoms may play a role in long-term patient burden. There is an unmet need for interventions that provide better symptom control and improve HRQoL in patients with endogenous CS.