Real-World Data on the Efficacy and Safety of Osilodrostat in Patients with Cushing’s Disease in Spain

שמור ב:
מידע ביבליוגרפי
הוצא לאור ב:Journal of Clinical Medicine vol. 14, no. 21 (2025), p. 7575-7588
מחבר ראשי: Araujo-Castro, Marta
מחברים אחרים: García-Centeno Rogelio, González, Laura, Hanzu, Felicia A, Orois Aida, Camara, Rosa, García María Dolores Ollero, Echarri Ana Irigaray, Gracia Gimeno Paola, Pascual-Corrales Eider, Biagetti Betina, Cardona Andrés, González Molero Inmaculada, Simo-Servat Andreu, Guerrero Pérez Fernando, Villar-Taibo Rocío, Bernabéu Ignacio, Fajardo-Montañana Carmen, Novo-Rodríguez, Cristina, Tenorio, Jimenéz Carmen, Calatayud María, Moure Rodríguez María Dolores, Cordido Fernando, Castro, Ana, Valero Lucía Manzano, Paja Fano Miguel, Goi Jessica, Aulinas, Anna, Abellán Pablo, Iglesias, Pedro, Soto-Moreno, Alfonso
יצא לאור:
MDPI AG
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גישה מקוונת:Citation/Abstract
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Resumen:Objective: To evaluate the efficacy and safety of osilodrostat in patients with Cushing’s disease (CD). Methods: A retrospective, multicenter, real-world study of patients with CD. The main efficacy endpoint was the proportion of patients who were complete responders (urinary free cortisol [UFC] < the upper limit of normal and/or with adrenal insufficiency development). Results: Thirty-seven CD patients were enrolled. There were 33 patients who initially received osilodrostat in monotherapy and 4 in combination. However, 3 patients of the monotherapy group were switched to combination therapy. The median duration of osilodrostat treatment was 5 months (range 1–93). All the patients were classified as responders: 33 (89.2%) had complete response and 4 partial response. A positive correlation was detected between the percentage of UFC decrease and the maximum (r = 0.481, p = 0.006) and the maintenance doses (r = 0.440, p = 0.011). The initial doses of osilodrostat were a predictor of complete response (vs. partial) (Odds ratio [OR] 2.82, p = 0.030). The median time to UFC normalization in the group of complete responders was 4 weeks (range 1–20) and UFC normalized before or at month 1 in 67% (n = 20/30) of the patients. Osilodrostat led to a significant decrease in systolic and diastolic blood pressure in parallel with a reduction of antihypertensive medications. Conclusions: Osilodrostat leads to a complete UFC normalization in up to 90% of the patients with CD, in parallel with an improvement in the cardiometabolic profile. A proper titration of osilodrostat is important to achieve a complete response since a positive correlation between the doses and the UFC reduction was observed.
ISSN:2077-0383
DOI:10.3390/jcm14217575
Fuente:Health & Medical Collection