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Purpose <p>To evaluate the efficacy and safety of osilodrostat in patients with Ectopic Cushing syndrome (ECS).</p> Methods <p>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] &lt; the upper limit of normal [ULN] or adrenal insufficiency development).</p> Results <p>A total of 17 patients with ECS were identified. Most of the cases (88.2%, <i>n</i> = 15) were classified as severe Cushing´s syndrome (UFC &gt; 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&#xa0;mg/day and the maximum doses between 4 and 60&#xa0;mg/day. Response to osilodrostat was evaluated in 16 patients because one patient died few days (&lt; 30) after the initiation of the treatment. We found that 88% (<i>n</i> = 14/16) were complete responders while 2 patients had partial response (UFC reduction &gt; 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.</p> Conclusion <p>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.</p>

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

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2025年12月3日

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  • Marta Araujo-Castro,
  • Rogelio Garcia-Centeno,
  • Laura González Fernández,
  • Alfonso Soto-Moreno,
  • Rosa Camara,
  • María Dolores Ollero García,
  • Ana Irigaray Echarri,
  • Paola Gracia,
  • Eider Pascual-Corrales,
  • Betina Biagetti,
  • Andrés Cardona,
  • Inmaculada González Molero,
  • Andreu Simo-Servat,
  • Fernando Guerrero-Perez,
  • Rocío Villar-Taibo,
  • Ignacio Bernabéu,
  • Carmen Fajardo-Montañana,
  • Cristina Novo-Rodríguez,
  • Carmen Tenorio-Jimenéz,
  • María Calatayud,
  • María Dolores Moure Rodríguez,
  • Fernando Cordido,
  • Ana Castro,
  • Lucía Manzano Valero,
  • Miguel Paja,
  • Jessica Goi,
  • Anna Aulinas,
  • Pablo Abellán,
  • Pedro Iglesias,
  • Felicia Alexandra Hanzu

摘要

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 Cushing´s 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.