Patient-reported responses to medical treatment in primary dystonia

In the absence of pathogenesis-targeted therapy in most types of primary dystonia, the current management strategy is largely symptomatic. Our aim was to comparatively evaluate the patients' perception of symptomatic benefits with the medical treatment of primary dystonia. We reviewed the medical records of 206 patients who received medical treatment upon diagnosis of primary dystonia. The patients were prescribed five different dystonia medications: clonazepam, trihexyphenidyl, nortriptyline, baclofen, and levodopa. Patients tried one type of medicine during each following week and whether each medication was beneficial was recorded in a binary fashion. Subgroups analysis was performed according to the body distribution, duration, ages at onset and treatment of dystonia. A total of 172 patients were included in the analysis. The majority (84%) had focal dystonia, most frequently cervical dystonia and blepharospasm. Clonazepam received the most favorable response (40%), followed by baclofen (20%) and trihexyphenidyl (20%). Patients with focal limb dystonia gave higher rate of positive responses to levodopa (24%) compared to other focal dystonia subgroups. Clonazepam, followed by baclofen and trihexyphenidyl is a useful pharmacologic option for primary dystonia. Levodopa can be considered for isolated limb dystonia.