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<p>Osilodrostat is a new oral steroidogenesis inhibitor, which has been recently approved for the treatment of adult patients with Cushing’s syndrome, including Cushing’s disease and paraneoplastic secretions. Scientific evidence provided by multicentre phase II and III clinical trials and real world studies shows that osilodrostat reduces effectively 24-hours urinary cortisol with significant improvement of all the features due to hypercortisolism. Caution should be taken to avoid or prevent adrenal insufficiency. Regimens can vary and should be tailored to the patient and their underlying disease. Here we will review the clinical trials and real world studies on the use of osilodrostat in the different settings.</p>

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Uso dell’osilodrostat: dagli studi LINC alla real world nelle sindromi di Cushing da ACTH ectopico

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2025年4月22日

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  • Filippo Ceccato,
  • Rossana Richiusa,
  • Dario Sardone,
  • Stella Bernardi

摘要

Osilodrostat is a new oral steroidogenesis inhibitor, which has been recently approved for the treatment of adult patients with Cushing’s syndrome, including Cushing’s disease and paraneoplastic secretions. Scientific evidence provided by multicentre phase II and III clinical trials and real world studies shows that osilodrostat reduces effectively 24-hours urinary cortisol with significant improvement of all the features due to hypercortisolism. Caution should be taken to avoid or prevent adrenal insufficiency. Regimens can vary and should be tailored to the patient and their underlying disease. Here we will review the clinical trials and real world studies on the use of osilodrostat in the different settings.