Efficacy and safety of vigabatrin as preventive therapy for children with tuberous sclerosis complex: A systematic review and meta-analysis.
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PURPOSE: Tuberous sclerosis complex (TSC) is associated with early-onset epilepsy, often leading to drug-resistant epilepsy (DRE) and developmental impairment. Preventive therapy with vigabatrin (VGB) has been proposed as a strategy to modify disease progression, but its efficacy and safety remain uncertain. This systematic review and meta-analysis aimed to evaluate the impact of preventive VGB therapy on seizure occurrence [including infantile epileptic spasms syndrome (IESS) and DRE], neurocognitive outcomes, and adverse events in infants with TSC. METHODS: We performed a systematic search of MEDLINE, EMBASE, Scopus, and Web of Science. Studies were eligible if they enrolled infants with TSC without prior seizures and compared preventive VGB to standard treatment. Risk of bias was assessed using the ROB 2.0 tool for randomized trials and the Newcastle-Ottawa Scale for observational studies. Meta-analyses were performed using a random-effects model, with results expressed as risk ratios (RR) or standardized mean differences (SMD) and 95 % confidence intervals (CI). RESULTS: Three studies with 149 children were included. There was reduced occurrence of seizures (including IESS and DRE) in the preventive therapy group (39/68 vs 64/81). However, the risk ratios were not statistically significant for occurrence of seizures (RR: 0.72; 95 % CI: 0.47-1.10), IESS (RR: 0.23; 95 % CI: 0.04-1.25), and DRE (RR: 0.74; 95 % CI: 0.49-1.12). Neurocognitive outcomes did not differ significantly between the two groups (SMD: 0.35; 95 % CI: -0.21- 0.91). Preventive vigabatrin was generally well-tolerated, with few adverse events and rare treatment discontinuation reported. CONCLUSION: Preventive vigabatrin therapy may prevent the development of epilepsy, including IESS and DRE, in children with TSC, with an acceptable safety profile. Although statistical significance was not achieved, the favorable trend highlights the potential clinical benefits of early intervention with VGB. Larger, high-quality randomized trials are warranted to confirm these findings and explore the long-term neurodevelopmental outcomes.