Stymulacja nerwu błędnego u pacjentów z zespołem stwardnienia guzowatego i oporną na leki epilepsją: badanie prospektywne

OBJECTIVE: Tuberous sclerosis complex (TSC) is a genetic multisystem disorder with a high prevalence of drug-resistant epilepsy (DRE), significantly affecting patients' quality of life. For patients with TSC lacking localizing epileptogenic tuber, resective surgery is often not feasible. Vagus nerve stimulation (VNS) has been proposed as an alternative surgical therapy, although robust evidence in TSC-related DRE remains limited. METHODS: A prospective cohort study was conducted between December 2019 and July 2024 across multiple epilepsy centers in China. Seventy patients with TSC-related DRE were enrolled, all lacking resectable epileptogenic tubers on multidisciplinary evaluation and ineligible for resective surgery. Participants were divided into VNS (n = 20) and medication-only (n = 50) groups based on actual treatment, and followed for up to 3 years. Seizure outcomes, anti-seizure medication use, IQ, and quality of life were evaluated longitudinally. RESULTS: The VNS group demonstrated significantly higher response rates at 1-, 2-, and 3-year follow-ups (65%-75%) compared to the medical treatment group (31%-36%). Median and average seizure frequency reductions were also greater in the VNS group. VNS was associated with significantly higher treatment response than medication, with the relative risk (RR) of response being 1.81 (95% confidence interval [CI]: 1.22-2.67), 2.21 (95% CI: 1.42-3.44), and 2.42 (95% CI: 1.49-3.93) at 1-, 2-, and 3-year follow-ups (p < 0.01). Seizure recurrence was significantly lower in the VNS group (p < 0.01). Moreover, VNS treatment led to significant reductions in anti-seizure medication (ASM) use and improvement in intelligence quotient and quality of life at the 2-year follow-up. Treatment modality was the only independent predictor of clinical response. VNS was well tolerated, with only mild surgical complications and no permanent adverse events reported. SIGNIFICANCE: VNS significantly improves seizure control, cognitive outcomes, and quality of life, with a favorable safety profile. These findings support VNS as an effective and safe alternative for this specific TSC population. Future randomized controlled trials are warranted to further validate these results.