Efficacy and Safety of Osilodrostat in Paraneoplastic Cushing Syndrome: A Real-World Multicenter Study in France
Authors' conclusion
Osilodrostat is a rapidly efficient therapy for PNCS/EAS with severe/intense hypercortisolism. Osilodrostat was generally well tolerated; adrenal insufficiency was the main side effect.
Study context
- Population
- Patients with paraneoplastic Cushing syndrome/ectopic ACTH syndrome (PNCS/EAS) with intense/severe hypercortisolism, France, May 2019–March 2022
- Participants
- 33 patients
- Exposure
- First-line monotherapy vs Second-line monotherapy vs Combination
- First-line monotherapyn = 11
- Second-line monotherapyn = 13
- Combinationn = 9
- Outcome
- Median 24h-UFC (× ULN), before → on osilodrostat
Methodological detailsmulticenter, real-world, Median initial dose 4 mg/day (range 1-60); maximum dose 20 mg/day (4-100), Regimens: titration (n = 6), block and replace (n = 16), titration then block and replace (n = 11)
- Multicenter
- Real-world
- Median initial dose 4 mg/day (range 1-60); maximum dose 20 mg/day (4-100)
- Regimens: titration (n = 6), block and replace (n = 16), titration then block and replace (n = 11)
Main finding
As reported in the abstract
- First-line monotherapy: 24h-UFC from 26 × ULN (2.9-659) to 0.11 × ULN (0.08-14.9)
- P < .001
- Second-line monotherapy: 24h-UFC from 2.6 × ULN (1.1-144) to 0.22 × ULN (0.12-0.66)
- P < .01
- Combination: 24h-UFC from 11.8 × ULN (0.3-247) to 0.43 × ULN (0.33-2.4)
- P < .01
Observational data: associations, not effects.
Further findings
- 24h-UFC normalization, first-line monotherapy
- 9 · events
- 24h-UFC normalization, first-line monotherapy — Median time to normalization
- 2 weeks
Clinical symptoms/comorbidities
- Major symptoms/comorbidities improved dramatically (blood pressure, hyperglycemia, hypokalemia), allowing discontinuation or dose reduction of treatments
Safety
- Adrenal insufficiency (grade 3-4)
- 8 of 33 patients
Original publication
Efficacy and Safety of Osilodrostat in Paraneoplastic Cushing Syndrome: A Real-World Multicenter Study in France. J Clin Endocrinol Metab. Published 6 December 2022. doi:10.
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