ABSTRACT
OBJECTIVE: In the Brazilian public national healthcare system, botulinum toxin type A has traditionally been the sole treatment option for patients with dystonia. However, as of October 2022, deep brain stimulation (DBS) garnered positive recommendations for the condition. This study aims to assess the cost-effectiveness of DBS in treating adults with generalized and cervical dystonia within the Brazilian healthcare context, considering its recent inclusion. METHODS: A systematic review identified randomized controlled trials assessing DBS efficacy in treating adults with generalized and cervical dystonia. Two cost-utility analyses compared the cost-effectiveness of DBS plus the best clinical practice (BCP) to BCP alone. Markov models, which included 3 health states (no clinical improvement, clinical improvement, and death), employed a 1-year cycle and a lifetime horizon. The study used both 1-way and probabilistic sensitivity analyses. RESULTS: Two randomized controlled trials, one for each condition, revealed superior clinical improvement with DBS when compared to sham simulation. The incremental cost-utility ratio was $ 1121.66 for generalized dystonia and $4556.50 for cervical dystonia. Effectiveness discount rates and age at surgery were identified as influential parameters. In 1000 Monte Carlo simulations, 99.9% of the incremental cost-utility ratio values for generalized dystonia and 74.2% for cervical dystonia fell below the cost-effectiveness threshold in Brazil ($8146.64 per quality-adjusted life year). CONCLUSIONS: From the perspective of the Brazilian public health system, the combination of DBS and BCP appears to be cost-effective for the treatment of both generalized and cervical dystonia when compared to BCP alone.