Treatment Continuation and Long-Term Outcomes of Perampanel in Primary Orthostatic Tremor; A Cohort Study

Primary orthostatic tremor (POT) is a rare hyperkinetic movement disorder with limited and largely ineffective treatment options. Perampanel has been suggested as a therapy, but has so far only been investigated in small studies with short duration.

To (1) describe longitudinal perampanel use and its association with key clinical outcomes and baseline characteristics in a well-defined cohort of POT, and (2) evaluate factors possibly associated with treatment continuation.

A prospective observational cohort study in POT patients who started perampanel treatment between November 2018 and November 2022, and who completed five self-report scales prior to treatment start and during clinical follow-up were included. Scales assessed daily functioning and well-being, and included the Dutch OT-questionnaire, Hospital Anxiety and Depression Scale, (instrumental) Activities of Daily Living, and the Short Form-36.

Thirty one POT patients were included, and followed for 32 months on average. Seventeen patients continued taking perampanel, demonstrating improvements in social functioning, fewer emotional role limitations, better mental health, and reduced pain compared to those who discontinued. Fourteen patients discontinued perampanel due to lack of effect and/or adverse effects, which were also common and persistent in those who continued. Men were more likely to continue treatment. Patients who discontinued had lower baseline functioning and well-being scores than those continuing, which persisted over time.

In around half of patients, continued use of perampanel is associated with better clinical outcomes, also on the long term, but adverse effects are common and persistent.