Opicapone in Parkinson’s disease: a real-world, multicenter, retrospective study to identify patient characteristics for sustained treatment benefit

Fluctuations often complicate the course of Parkinson's disease (PD). The third-generation catechol-O-methyltransferase inhibitor, opicapone, is safe and effective in patients with PD motor fluctuations, whether long-standing or earlier onset. Real-world data are needed to identify the most appropriate candidates for initiating and sustaining therapy with opicapone. To evaluate the long-term outcomes and identify predictors for safer and prolonged use of opicapone. This real-world, longitudinal, multicenter, retrospective study collected data on the demographics, medical history, motor subtype, motor severity, cognitive and psychiatric disorders at baseline when opicapone was introduced as add-on therapy and at the last follow-up. Discontinuation rates and main causes were recorded. Clinical features of patients who continued/discontinued were compared to identify predictors for drug discontinuation and define successful long-term outcomes. Of the 178 enrolled patients, 85% (35% of them female) continued opicapone for a mean of 30.2 ± 19.64 months. The remaining 15% (13.4% female) discontinued opicapone within 8.9 ± 11.30 months due to disabling dyskinesia, hypotension, gastrointestinal disorders, or psychiatric disturbances. Continuers were younger at baseline than those who discontinued, but clinical-demographic features did not predict continuing opicapone therapy. Continuers had significantly lower Hoehn and Yahr scale scores and fewer night-time OFF periods compared with discontinuers, whereas levodopa equivalent daily dose and burden of motor and neuropsychiatric complications were similar. Continued opicapone therapy was associated with less motor impairment and reduced night-time OFF. Safety and long-term tolerability were higher in younger patients with a tendency to milder motor severity.