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

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...

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Detalles Bibliográficos
Autores: Araujo-Castro, M, García-Centeno, R, González, L, Hanzu, FA, Orois, A, Camara, R, García, MDO, Echarri, AI, Gimeno, PG, Pascual-Corrales, E, Biagetti, B, Cardona, A, Molero, IG, Simo-Servat, A, Pérez, FG, Villar-Taibo, R, Bernabéu, I, Fajardo-Montañana, C, Novo-Rodríguez, C, Jimenéz, CT, Calatayud, M, Rodríguez, MDM, Cordido, F, Castro, A, Valero, LM, Fano, MP, Goi, J, Aulinas, A, Abellán, P, Iglesias, P, Soto-Moreno, A
Tipo de recurso: artículo
Estado:Versión publicada
Fecha de publicación:2025
País:España
Institución:Fundación para el Fomento de la Investigación Sanitaria y Biomédica de la Comunitat Valenciana (FISABIO)
Repositorio:r-FISABIO. Repositorio Institucional de Producción Científica
OAI Identifier:oai:fisabio.fundanetsuite.com:p20112
Acceso en línea:https://fisabio.portalinvestigacion.com/publicaciones/20112
Access Level:acceso abierto
Palabra clave:Cushing's syndrome
Cushing's disease
pituitary
osilodrostat
urinary free cortisol
Descripción
Sumario: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.

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