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

<b>Objective</b>: To evaluate the efficacy and safety of osilodrostat in patients with Cushing's disease (CD). <b>Methods:</b> 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). <b>Results:</b> 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, <i>p</i> = 0.006) and the maintenance doses (r = 0.440, <i>p</i> = 0.011). The initial doses of osilodrostat were a predictor of complete response (vs. partial) (Odds ratio [OR] 2.82, <i>p</i> = 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% (<i>n</i> = 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. <b>Conclusions:</b> 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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Journal
Journal of Clinical Medicine
Published
2025-10-25
DOI
https://doi.org/10.3390/jcm14217575
Citations
2
Primary Topic
Pituitary Gland Disorders and Treatments
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article
Field-Weighted Citation Impact
2.59
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article

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

Cristina Novo‐Rodríguez, Miguel Paja, Rocío Villar‐Taibo, Luis Candil Valero et al.
2 citations
Journal of Clinical Medicine
Pituitary Gland Disorders and Treatments
2.59
article

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

Cristina Novo‐Rodríguez, Miguel Paja, Rocío Villar‐Taibo, Luis Candil Valero, Fernando Cordido, Andrés Cardona, Rosa Cámara, Anna Aulinas, Paola Gracia Gimeno, Laura González Fernández, Ana Irigaray, Andreu Simó-Servat, Pedro Iglesias, Marta Araujo‐Castro, Ana Castro, Alfonso Soto‐Moreno, Ignacio Bernabéu, Fernando Pérez-Sánz, Inmaculada González Molero, Rogelio García-Centeno, Betina Biagetti, Felicia Alexandra Hanzu, María Calatayud, Pablo Abellán Galiana, Aida Orois, Eider Pascual‐Corrales, Carmen Tenorio-Jiménez, Carmen Fajardo, María Dolores Moure Rodríguez, María Dolores Ollero García, Jessica Goi
article en
2 citations

Abstract

<b>Objective</b>: To evaluate the efficacy and safety of osilodrostat in patients with Cushing's disease (CD). <b>Methods:</b> 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). <b>Results:</b> 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, <i>p</i> = 0.006) and the maintenance doses (r = 0.440, <i>p</i> = 0.011). The initial doses of osilodrostat were a predictor of complete response (vs. partial) (Odds ratio [OR] 2.82, <i>p</i> = 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% (<i>n</i> = 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. <b>Conclusions:</b> 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.

Journal of Clinical MedicineVol. 14(21)
Universidade da Coruña (ES), University of the Basque Country (ES), Instituto Cajal (ES), Hospital de Sant Pau (ES), Bellvitge University Hospital (ES), ProMedica Toledo Hospital (US), Hospital General Universitario Gregorio Marañón (ES), Hospital Universitari i Politècnic La Fe (ES), Hospital de Basurto (ES), Hospital Terrassa (ES), Hospital de La Ribera (ES), Hospital Clínico Universitario de Valencia (ES), Complejo Hospitalario Universitario de Santiago (ES), Complejo Hospitalario de Navarra (ES), Hospital Universitario 12 De Octubre (ES), Hospital Royo Villanova (ES), Hospital Universitario Puerta de Hierro Majadahonda (ES), Hospital Regional Universitario de Málaga (ES), Hospital Clínic de Barcelona (ES), Hospital Universitario Virgen de las Nieves (ES), Leitat Technological Center (ES), Instituto Ramón y Cajal de Investigación Sanitaria (ES), Vall d'Hebron Hospital Universitari (ES), Instituto de Biomedicina de Sevilla (ES), Hospital de Cruces (ES), Complexo Hospitalario Universitario A Coruña (ES), Hospital Universitario Virgen del Rocío (ES), Hospital Universitario Ramón y Cajal (ES), Complejo Hospitalario Universitario de Toledo (ES), Instituto de Investigación Sanitaria La Fe (ES), Universidad de Navarra (ES)
Openalex Percentile: Top 8%
Pituitary Gland Disorders and Treatments
2.59
DOIOpen Access PDFOpenAlex
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