Metyrapone-associated improvements in health-related quality of life in Cushing's syndrome

Mezôsi, Emese; Nieman, Lynnette; Scaroni, Carla; Boscaro, Marco; Deutschbein, Timo; Driessens, Natacha; Georgescu, Carmen; Motyka, Marcin; Hubalewska-Dydejczyk, Alicja; Jarzab, Barbara; Maiter, Dominique; Reincke, Martin; Loli, Paola; Zampetti, Benedetta; Atmaca, Ayşegül; Badiu, Corin; Beckers, Albert; Bolanowski, Marek; Cavagnini, Francesco; Unger, Nicole; Giordano, Roberta; Hanzu, Felicia; Terzolo, Massimo; Domenech, Arnau; Nader, Myriam; Sinaii, Ninet; Tőke, Judit; Tóth, Miklós

doi:10.1093/ejendo/lvag164

Metyrapone-associated improvements in health-related quality of life in Cushing's syndrome

Mezôsi E., Nieman L. K., Scaroni C., Boscaro M., Deutschbein T., Driessens N., ...Daha Fazla

European Journal of Endocrinology, cilt.195, sa.3, ss.431-442, 2026 (SCI-Expanded, Scopus)

Yayın Türü: Makale / Tam Makale
Cilt numarası: 195 Sayı: 3
Basım Tarihi: 2026
Doi Numarası: 10.1093/ejendo/lvag164
Dergi Adı: European Journal of Endocrinology
Derginin Tarandığı İndeksler: Science Citation Index Expanded (SCI-EXPANDED), Scopus, BIOSIS, Chemical Abstracts Core, EMBASE, MEDLINE, Academic Search Ultimate (EBSCO)
Sayfa Sayıları: ss.431-442
Anahtar Kelimeler: Cushing's syndrome, CushingQoL, longitudinal, metyrapone, patient-reported outcomes, PROMPT study, quality of life, Tübingen CD-25
Ondokuz Mayıs Üniversitesi Adresli: Evet

Özet

Objective: This analysis describes the evolution of health-related quality of life (HRQoL) over 36 weeks of metyrapone treatment in the PROMPT study, identifying patterns of early, delayed improvement, or persistence using item-level resolution. Design and Methods: Longitudinal analysis of patient-reported outcomes from PROMPT (NCT02297945), a prospective, open-label, multicountry study of metyrapone in 49 evaluable adults with confirmed endogenous CS. HRQoL was assessed with the CushingQoL (0–100; higher = better QoL) and Tübingen CD-25 (0–100; higher = worse QoL) at weeks 4, 12, 24, and 36. Item-level analyses characterized the pattern and timing of improvement. Within-patient changes were assessed using the Wilcoxon signed-rank test. Results: Both questionnaires demonstrated significant overall improvement by week 36: CushingQoL increased by 10.4 points (P < .001; 44.7% with ≥10-point improvement), and Tübingen CD-25 decreased by −7.8 points (P = .01). Early responses (by week 12) involved eating behaviour and depression symptoms. Delayed improvements (consolidating at weeks 24-36) were observed in sexual activity, environment, and social domains. Bodily restrictions (−4.3 points, P = .203) and cognition (−6.8 points, P = .143) did not show improvement over the study period. Item-level analysis identified a cluster of symptoms, predominantly psychological health anxiety and physical appearance concerns, where burden remained elevated throughout the 36 weeks despite cortisol normalization. Conclusions: 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.