How Do Oral Medications, Botulinum Toxin Use, and Treatment Burden Change after Deep Brain Stimulation for Dystonia?

Deep brain stimulation (DBS) is an established therapy for dystonia; however, the long-term trajectory of adjunctive treatment burden, including oral medications and botulinum toxin (BoNT) injections, and the likelihood of achieving medication-free status remain poorly understood, despite important implications for adverse effects, injection burden, and quality of life.

Determine long-term changes in adjunctive medication burden and the durability of medication-free status among dystonia patients undergoing DBS.

We retrospectively examined the proportion of dystonia patients requiring oral medications and BoNT therapy after DBS, tracking postoperative changes at 6 months, 1, 3, 5, and 10 years. Factors influencing overall medication burden were evaluated, and the proportion of patients achieving treatment-free status was determined.

Among 100 patients in this cohort (56% generalized dystonia), baseline use of oral medications and BoNT was 85% and 43%, respectively. The proportion requiring oral medications and BoNT declined to ~60% and ~ 20-25% as DBS benefits emerged, with reductions maintained throughout follow-up. Among those continuing BoNT, dosing remained largely unchanged. The overall medication burden score, calculated by combining oral medications and BoNT, was significantly reduced at all follow-up intervals, with body distribution influencing the magnitude of reduction. Around one-third of patients did not require any medications at 10-year follow-up.

DBS leads to sustained reductions in adjunctive oral medications and BoNT use. At 10-year follow-up, around 20% of dystonia patients continued to require BoNT injections, but 30% were completely medication-free. These data highlight evolving long-term benefits and inform patient expectations.