Objective: To assess the efficacy and side-effects of TBZ in Huntington disease (HD), vascular chorea, tics, dystonia, oromandibular dyskinesia and other tardive syndromes (TS).

Background: Tetrabenazine (TBZ) is commonly used as a treatment in a wide spectrum of hyperkinetic movement disorders.

Methods: Qualitative review of clinical response was used to estimate TBZ efficacy. TBZ-induced adverse events (AE) frequency and subsequent discontinuation rate were used for estimate TBZ safety. Tolerability profile was measure through the TBZ minimal dose and exposure time required to elicit AEs.

Results: We enrolled 108 patients. 87% had a clinically meaningful improvement sustained over a period of 40 months. The number of responders ranged from 100% in HD to 62.5% and 77.1% in tics disorders and oromandibular dyskinesia (p<0.001). TBZ associated AE were found in 48.1% of patients and ranged from 40.9% in other TS and 41.7%, in vascular chorea and HD, to 60% in oromandibular dyskinesia (p<0.001). The most common AE were parkinsonism (51.8%), followed by psychiatric disorders (25%) other movement disorders (3.6%) and other AEs (19.6%). TBZ discontinuation rate did not differ between diagnostic categories after AE emergence. AE eliciting dose differed according to the diagnosis (from 18.8 mg in tics disorders to 75 mg in HD, p=0.003).

Conclusions: TBZ is moderately effective in a wide spectrum of hyperkinetic movement disorders, with excellent results in the HD, which was related to different tolerability profiles. Somnolence was the most frequent AE during titration period and parkinsonism during maintenance period.

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MDS Abstracts - https://www.mdsabstracts.org/abstract/efficacy-and-safety-of-tetrabenazine-in-hyperkinetic-movements-disorders-an-observational-study/