Efficacy and safety of osilodrostat in patients with ectopic Cushings syndrome. a real-world study in Spain

Purpose To evaluate the efficacy and safety of osilodrostat in patients with Ectopic Cushing syndrome (ECS). Methods A retrospective, multicenter, real-world study of patients with ECS treated with osilodrostat. The main efficacy endpoint was the proportion of patients who were complete responders (...

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Detalles Bibliográficos
Autores: Araujo-Castro, M, Garcia-Centeno, R, Fernández, LG, Soto-Moreno, A, Camara, R, García, MDO, Echarri, AI, Gracia, P, Pascual-Corrales, E, Biagetti, B, Cardona, A, Molero, IG, Simo-Servat, A, Guerrero-Perez, F, Villar-Taibo, R, Bernabéu, I, Fajardo-Montañana, C, Novo-Rodríguez, C, Tenorio-Jimenéz, C, Calatayud, M, Rodríguez, MDM, Cordido, F, Castro, A, Valero, LM, Paja, M, Goi, J, Aulinas, A, Abellán, P, Iglesias, P, Hanzu, FA
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:p20109
Acceso en línea:https://fisabio.portalinvestigacion.com/publicaciones/20109
Access Level:acceso abierto
Palabra clave:Cushings syndrome
Ectopic cushing syndrome
Osilodrostat
Urinary free cortisol
Descripción
Sumario:Purpose To evaluate the efficacy and safety of osilodrostat in patients with Ectopic Cushing syndrome (ECS). Methods A retrospective, multicenter, real-world study of patients with ECS treated with osilodrostat. The main efficacy endpoint was the proportion of patients who were complete responders (urinary free cortisol [UFC] < the upper limit of normal [ULN] or adrenal insufficiency development). Results A total of 17 patients with ECS were identified. Most of the cases (88.2%, n = 15) were classified as severe Cushings syndrome (UFC > 5 ULN). Two patients received osilodrostat as first-line therapy, 9 as second line and 6 as a third line. Fourteen patients were treated with osilodrostat in monotherapy and 3 in combination with other treatments. The initial doses of osilodrostat ranged between 4 and 30 mg/day and the maximum doses between 4 and 60 mg/day. Response to osilodrostat was evaluated in 16 patients because one patient died few days (< 30) after the initiation of the treatment. We found that 88% (n = 14/16) were complete responders while 2 patients had partial response (UFC reduction > 50% but with no normalization). The median time to achieve hypercortisolism control was 4.5 weeks (range 1-12), and 40% of the cases had normal UFC after 1 month of treatment. Six patients developed adverse events associated with the use of osilodrostat: 3 had adrenal insufficiency, 1 QT prolongation and 1 deterioration of blood pressure control. Conclusion Overall, osilodrostat controls hypercortisolism in approximately 90% of the patients with ECS and severe hypercortisolism and with normalization of UFC in 40% of cases after just 4 weeks of treatment. Therefore, osilodrostat should be considered as first-line treatment in patients with ECS, especially in patients with severe hypercortisolism.

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