Objective: To characterize real-world treatment patterns in adults with isolated dystonia in the United States.

Background: Isolated dystonia is a rare, complex movement disorder characterized by involuntary, sustained or intermittent muscle contractions that cause abnormal posturing, twisting, or repetitive movements. Real-world data on patterns of medication and therapy used in adults with isolated dystonia remain limited.

Method: This retrospective study used US-linked claims and electronic medical records from the IQVIA PharMetrics Plus database to identify a cohort of individuals with newly diagnosed isolated dystonia between January 2017 and December 2024. The study included adults (N=20,951) who had 12 months of continuous medical and pharmacy benefits enrolled before (baseline) and after (follow-up) the index date (date of first diagnosis). Treatment use at baseline and follow-up was assessed for botulinum toxin (BoNT), deep brain stimulation (DBS), pain and psychiatric medications, muscle relaxants, dystonia-related medications, and rehabilitative therapies.

Results: BoNT utilization at 12 months after diagnosis was 17.7%. In adults with 3-year follow-up, BoNT utilization was 27%. Use of pain medications significantly increased from baseline to follow-up (NSAIDs, 25% to 42%; opioids, 22% to 36%; gabapentin/pregabalin, 10% to 16%). Significant increases were also observed for benzodiazepines (13% to 22%) and baclofen (3% to 7%). Use of dystonia-related medications significantly increased from baseline to follow-up (anticholinergics, 6% to 12%), as did use of muscle relaxants (cyclobenzaprine, 9% to 18%; methocarbamol, 3% to 7%; tizanidine, 4% to 7%). During the follow-up period, use of VMAT2 inhibitors (0.1%), dopamine agonists (2.0%), levodopa or carbidopa (0.8%), and DBS (0.2%) was negligible. Rehabilitative therapies (eg, physical therapy, occupational therapy, and chiropractic care) also showed substantial increases from baseline to follow-up.

Conclusion: Adults with isolated dystonia showed increased reliance on medications for pain and psychiatric disorders that carry a risk of dependence following diagnosis. Use of BoNT was 17% at 12 months after diagnosis and was used by <30% of adults with 3-year follow-up. These findings suggest an unmet need for systemic treatments that effectively target the root cause of movement dysfunction and reduce the use of higher-risk off-label medicines.

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MDS Abstracts - https://www.mdsabstracts.org/abstract/treatment-patterns-in-adults-with-isolated-dystonia-in-the-united-states/