Real-World Data on the Efficacy and Safety of Osilodrostat in Patients with Cushing's Disease in Spain
Por:
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
Publicada:
25 oct 2025
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.
Filiaciones:
Araujo-Castro, M:
Hosp Univ Ramon y Cajal, Endocrinol & Nutr Dept, Colmenar Viejo St Km 9, Madrid 28034, Spain
García-Centeno, R:
Hosp Univ Gregorio Maranon, Endocrinol & Nutr Dept, Madrid 28007, Spain
González, L:
Hosp Univ Gregorio Maranon, Endocrinol & Nutr Dept, Madrid 28007, Spain
Hanzu, FA:
Hosp Clin Barcelona, Endocrinol & Nutr Dept, Barcelona 08036, Spain
Orois, A:
Hosp Clin Barcelona, Endocrinol & Nutr Dept, Barcelona 08036, Spain
Camara, R:
Hosp La Fe, Endocrinol & Nutr Dept, Valencia 46600, Spain
García, MDO:
Hosp Univ Navarra, Endocrinol & Nutr Dept, Pamplona 31008, Spain
Echarri, AI:
Hosp Univ Navarra, Endocrinol & Nutr Dept, Pamplona 31008, Spain
Gimeno, PG:
Hosp Univ Royo Villanova, Endocrinol & Nutr Dept, Zaragoza 50015, Spain
Pascual-Corrales, E:
Hosp Univ Ramon y Cajal, Endocrinol & Nutr Dept, Colmenar Viejo St Km 9, Madrid 28034, Spain
Biagetti, B:
Hosp Univ Vall Hebron, Endocrinol & Nutr Dept, Barcelona 08035, Spain
CIBERER Grp 747, Barcelona 08035, Spain
Cardona, A:
Hosp Univ Vall Hebron, Endocrinol & Nutr Dept, Barcelona 08035, Spain
CIBERER Grp 747, Barcelona 08035, Spain
Molero, IG:
Hosp Reg Univ Malaga, Endocrinol & Nutr Dept, Malaga 29010, Spain
Simo-Servat, A:
Hosp Terrassa, Dept Plast Surg, Barcelona 08227, Spain
Pérez, FG:
Hosp Univ Bellvitge, Endocrinol & Nutr Dept, LHospitalet De Llobregat 08907, Cataluna, Spain
Villar-Taibo, R:
Hosp Univ Santiago De Compostela, Endocrinol & Nutr Dept, Galicia 15706, Spain
Bernabéu, I:
Hosp Univ Santiago De Compostela, Endocrinol & Nutr Dept, Galicia 15706, Spain
Fajardo-Montañana, C:
Hosp Univ La Ribera, Endocrinol & Nutr Dept, Valencia 46600, Spain
Novo-Rodríguez, C:
Hosp Univ Virgen Nieves, Endocrinol & Nutr Dept, Granada 18014, Spain
Jimenéz, CT:
Hosp Univ Virgen Nieves, Endocrinol & Nutr Dept, Granada 18014, Spain
Calatayud, M:
Hosp Univ 12 Octubre, Endocrinol & Nutr Dept, Madrid 28041, Spain
Rodríguez, MDM:
Hosp Univ Cruces, Endocrinol & Nutr Dept, Bilbao 48903, Spain
Cordido, F:
Hosp Univ Coruna, Endocrinol & Nutr Dept, La Coruna 15006, Spain
Univ A Coruna, La Coruna 15006, Spain
Castro, A:
Hosp Univ Toledo, Endocrinol & Nutr Dept, Toledo 45007, Spain
Valero, LM:
Hosp Univ Toledo, Endocrinol & Nutr Dept, Toledo 45007, Spain
Fano, MP:
Hosp Univ Basurto, Endocrinol & Nutr Dept, OSI Bilbao Basurto, Bilbao 48013, Spain
Univ Basque Country UPV EHU, Med Dept, Bilbao 48013, Spain
Goi, J:
Hosp Univ Ramon y Cajal, Endocrinol & Nutr Dept, Colmenar Viejo St Km 9, Madrid 28034, Spain
Aulinas, A:
Hosp Santa Creu i St Pau, Endocrinol & Nutr Dept, Irsant PAU, CIBERER U747 ISCIII, Barcelona 08025, Spain
Abellán, P:
Hosp Univ Valencia, Endocrinol & Nutr Dept, Valencia 46010, Spain
Iglesias, P:
Hosp Univ Puerta Hierro, Endocrinol & Nutr Dept, Madrid 28222, Spain
Soto-Moreno, A:
Univ Seville, Hosp Univ Virgen Rocio, Unidad Gest Endocrinol & Nutr, Inst Biomed Sevilla IBiS,CSIC, Cordoba, Spain
Inst Invest Biomed Ramon Y Cajal IRYCIS, Colmenar Viejo St Km 9, Madrid 28034, Spain
Green Accepted, Green Submitted, gold
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