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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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
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spelling Real-World Data on the Efficacy and Safety of Osilodrostat in Patients with Cushing's Disease in SpainAraujo-Castro, MGarcía-Centeno, RGonzález, LHanzu, FAOrois, ACamara, RGarcía, MDOEcharri, AIGimeno, PGPascual-Corrales, EBiagetti, BCardona, AMolero, IGSimo-Servat, APérez, FGVillar-Taibo, RBernabéu, IFajardo-Montañana, CNovo-Rodríguez, CJimenéz, CTCalatayud, MRodríguez, MDMCordido, FCastro, AValero, LMFano, MPGoi, JAulinas, AAbellán, PIglesias, PSoto-Moreno, ACushing's syndromeCushing's diseasepituitaryosilodrostaturinary free cortisolObjective: 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.MDPI2025info:eu-repo/semantics/articleinfo:eu-repo/semantics/publishedVersionhttps://fisabio.portalinvestigacion.com/publicaciones/20112Journal of Clinical MedicineISSN: 20770383reponame:r-FISABIO. Repositorio Institucional de Producción Científicainstname:Fundación para el Fomento de la Investigación Sanitaria y Biomédica de la Comunitat Valenciana (FISABIO)Inglésinfo:eu-repo/semantics/openAccessoai:fisabio.fundanetsuite.com:p201122026-06-11T12:45:17Z
dc.title.none.fl_str_mv Real-World Data on the Efficacy and Safety of Osilodrostat in Patients with Cushing's Disease in Spain
title Real-World Data on the Efficacy and Safety of Osilodrostat in Patients with Cushing's Disease in Spain
spellingShingle Real-World Data on the Efficacy and Safety of Osilodrostat in Patients with Cushing's Disease in Spain
Araujo-Castro, M
Cushing's syndrome
Cushing's disease
pituitary
osilodrostat
urinary free cortisol
title_short Real-World Data on the Efficacy and Safety of Osilodrostat in Patients with Cushing's Disease in Spain
title_full Real-World Data on the Efficacy and Safety of Osilodrostat in Patients with Cushing's Disease in Spain
title_fullStr Real-World Data on the Efficacy and Safety of Osilodrostat in Patients with Cushing's Disease in Spain
title_full_unstemmed Real-World Data on the Efficacy and Safety of Osilodrostat in Patients with Cushing's Disease in Spain
title_sort Real-World Data on the Efficacy and Safety of Osilodrostat in Patients with Cushing's Disease in Spain
dc.creator.none.fl_str_mv 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
author Araujo-Castro, M
author_facet 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
author_role author
author2 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
author2_role author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
dc.subject.none.fl_str_mv Cushing's syndrome
Cushing's disease
pituitary
osilodrostat
urinary free cortisol
topic Cushing's syndrome
Cushing's disease
pituitary
osilodrostat
urinary free cortisol
description 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.
publishDate 2025
dc.date.none.fl_str_mv 2025
dc.type.none.fl_str_mv info:eu-repo/semantics/article
info:eu-repo/semantics/publishedVersion
format article
status_str publishedVersion
dc.identifier.none.fl_str_mv https://fisabio.portalinvestigacion.com/publicaciones/20112
url https://fisabio.portalinvestigacion.com/publicaciones/20112
dc.language.none.fl_str_mv Inglés
language_invalid_str_mv Inglés
dc.rights.none.fl_str_mv info:eu-repo/semantics/openAccess
eu_rights_str_mv openAccess
dc.publisher.none.fl_str_mv MDPI
publisher.none.fl_str_mv MDPI
dc.source.none.fl_str_mv Journal of Clinical Medicine
ISSN: 20770383
reponame:r-FISABIO. Repositorio Institucional de Producción Científica
instname:Fundación para el Fomento de la Investigación Sanitaria y Biomédica de la Comunitat Valenciana (FISABIO)
instname_str Fundación para el Fomento de la Investigación Sanitaria y Biomédica de la Comunitat Valenciana (FISABIO)
reponame_str r-FISABIO. Repositorio Institucional de Producción Científica
collection r-FISABIO. Repositorio Institucional de Producción Científica
repository.name.fl_str_mv
repository.mail.fl_str_mv
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score 15,812455

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