Metyrapone-associated improvements in health-related quality of life in Cushing's syndrome
Authors' conclusion
Metyrapone treatment is associated with meaningful, progressive HRQoL improvements across multiple symptom domains over 36 weeks, with a pattern consistent with rapid cortisol-mediated relief (eating, mood), delayed recovery in social and sexual domains, and persistent residual burden in symptom clusters likely driven by incomplete phenotypic reversal within the study timeframe.
Study context
- Population
- Adults with confirmed endogenous CSevaluable
- Participants
- 49 adults
Methodological detailslongitudinal analysis of patient-reported outcomes, prospective, open-label, multicountry, item-level analysis
- Longitudinal analysis of patient-reported outcomes
- Prospective
- Open-label
- Multicountry
- Item-level analysis
Primary result
As reported in the abstract
Change in health-related quality of life (HRQoL) · week 36
≥10-point CushingQoL improvement
44.7%
Further findings
Timing of domain improvement
- Early responses (by week 12) in eating behaviour and depression symptoms
- Delayed improvements (consolidating at weeks 24-36) in sexual activity, environment and social domains
Domains without improvement · over the study period
Points
- Bodily restrictions, P = .203
- −4.3
- Cognition, P = .143
- −6.8
Persistent symptom burden (item level) · throughout 36 weeks
- Cluster of symptoms, predominantly psychological health anxiety and physical appearance concerns, remained elevated despite cortisol normalization
Original publication
Metyrapone-associated improvements in health-related quality of life in Cushing's syndrome. Eur J Endocrinol. Published 31 August 2026. doi:10. 1093/ ejendo/ lvag164
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