ArticlePituitary2021
Corticotroph tumor progression during long-term therapy with osilodrostat in a patient with persistent Cushing's disease.
Article in Pituitary, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT02180217 (Phase III, Multi-center, Double-blind, Randomized Withdrawal Study of LCI699 Following a 24 Week, Single-arm, Open-label Dose Titration and Treatment Period to Evaluate the Safety and Efficacy of LCI699 for the Treatment of Patients With Cushing's Disease), which is not on this map. Cited by 16 papers.
What it found
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The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Phase III, Multi-center, Double-blind, Randomized Withdrawal Study of LCI699 Following a 24 Week, Single-arm, Open-label Dose Titration and Treatment Period to Evaluate the Safety and Efficacy of LCI699 for the Treatment of Patients With Cushing's Disease
Who cites it
16 citing papers in PubMed, 27 citations in OpenAlex.
- Levoketoconazole treatment in endogenous Cushing's syndrome: extended evaluation of clinical, biochemical, and radiologic outcomes.European journal of endocrinology · 2022Trial
- Use of osilodrostat in a block-and-replace and titration strategy for cushing's syndrome: a position paper by French experts.Reviews in endocrine & metabolic disorders · 2026Article
- Article
- Management of Osilodrostat Therapy in Patients With Cushing's Syndrome: A Modified Delphi Consensus Panel.Journal of the Endocrine Society · 2025Article
- Real-World Experience with Pasireotide-LAR in Cushing's Disease: Single-Center 12-Month Observational Study.Journal of clinical medicine · 2025Article
- Medical Treatment of Cushing's Syndrome.Endocrinology and metabolism (Seoul, Korea) · 2025Review
- Cushing syndrome.Nature reviews. Disease primers · 2025Review
- Medical treatment of functional pituitary adenomas, trials and tribulations.Journal of neuro-oncology · 2024Review
- Cushing's Disease: Long-Term Effectiveness and Safety of Osilodrostat in a Polish Group of Patients with Persistent Hypercortisolemia in the Experience of a Single Center.Biomedicines · 2023Article
- An individualized approach to the management of Cushing disease.Nature reviews. Endocrinology · 2023Review
- Clinical Utility of Osilodrostat in Cushing's Disease: Review of Currently Available Literature.Drug design, development and therapy · 2023Review
- Treatment of Cushing's syndrome with osilodrostat: practical applications of recent studies with case examples.Pituitary · 2022Review
- Management of Nelson's Syndrome.Medicina (Kaunas, Lithuania) · 2022Review
- Long-term outcomes of osilodrostat in Cushing's disease: LINC 3 study extension.European journal of endocrinology · 2022Article
- The Role of Glucocorticoid Receptor in the Pathophysiology of Pituitary Corticotroph Adenomas.International journal of molecular sciences · 2022Review
- Consensus on diagnosis and management of Cushing's disease: a guideline update.The lancet. Diabetes & endocrinology · 2021Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
5 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
purposeCorticotroph tumor progression (CTP) or Nelson's syndrome (NS) can occur in patients with Cushing's disease (CD) following bilateral adrenalectomy. It has rarely been observed in patients treated with long-term medical therapy for persistent CD. Osilodrostat (LCI699) is a new steroidogenesis inhibitor of 11β-hydroxylase (CYP11β1) that induced remission of hypercortisolism in 86% of patients with refractory CD in the randomized placebo-controlled trial LINC-3 (NCT02180217).
methodsA 40-year-old woman with persistent CD following transsphenoidal surgery was treated with osilodrostat in the LINC-3 trial and was followed with regular hormonal assessments and imaging of residual corticotroph tumor.
resultsUnder oral therapy with osilodrostat 10 mg twice daily, urinary free cortisol (UFC) normalized and clinical signs of CD regressed during therapy. However after 4 years of treatment, ACTH levels increased from 73 to 500 pmol/L and corticotroph tumor size increased rapidly from 3 to 14 mm, while UFCs remained well controlled. Surgical resection of an atypical tumor with weak ACTH expression and increased proliferative index (Ki-67 ≥ 8%) resulted in current remission but will require close follow-up.
conclusionThis case highlights the importance of monitoring ACTH and corticotroph tumor size in patients with persistent CD, either under effective treatment with steroidogenesis inhibitors or after bilateral adrenalectomy.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.