Title: Comparative Effectiveness of Vigabatrin Versus Alternative Therapies in Infantile Spasms: A Meta-analysis of PubMed Studies (P8-11.007)
Authors: Ana Leticia Fornari Caprara, Jamir Pitton Rissardo, Salman Assad, Evren Burakgazi-Dalkilic, and Eric P. Nagele
Conference: 2026 AAN, Chicago, IL
Objective
To evaluate the effectiveness of vigabatrin (VGB) compared to hormonal therapy (HRT), placebo, ketogenic diet, or combination therapy in achieving seizure freedom in infantile spasms (IS).
Background
VGB is widely used for IS, but its comparative efficacy remains debated. We synthesized evidence from randomized controlled trials (RCTs) and observational studies (OS) to clarify its role in clinical practice.
Design/Methods
We conducted a meta-analysis of 23 PubMed-indexed studies (n ≈ 2,000) reporting seizure freedom in IS patients without tuberous sclerosis complex. Random-effects models (DerSimonian-Laird) were applied using the Mantel-Haenszel method to pool risk ratios (RR) with 95% confidence intervals (CI). Heterogeneity was assessed with I² and Cochran’s Q. Publication bias was evaluated using Egger’s and Begg’s tests, trim-and-fill, and fail-safe N analyses.
Results
The random-effects model demonstrated a significant benefit of VGB (RR 0.92, 95% CI 0.84 to 0.9994). Prediction interval ranged from 0.48 to 1.50. Subgroup analyses showed consistent effects: RCTs comparing VGB vs HRT (RR 0.79; 95% CI 0.69 to 0.90) and observational studies (RR 0.76; 95% CI 0.64 to 0.90). Combination therapy (VGB + HRT) showed no clear advantage (RR 1.25; 95% CI 1.09 to 1.45). Egger’s test indicated small-study effects (p = 0.019), and trim-and-fill suggested six missing studies, reducing the effect size by 66%. Fail-safe N was 12, indicating susceptibility to publication bias.
Conclusions
VGB significantly improves seizure freedom compared to other therapies, with consistent effects across RCTs and observational studies. However, evidence of small-study effects and potential publication bias warrants cautious interpretation. Further large-scale trials are needed to confirm these findings.
Citation
Caprara AL, Rissardo JP, Assad S, Burakgazi-Dalkilic E, Nagele EP. Comparative Effectiveness of Vigabatrin Versus Alternative Therapies in Infantile Spasms: A Meta-analysis of PubMed Studies (P8-11.007). Neurology 2026;106(11_suppl):690. doi: 10.1212/WNL.0000000000213062.
Figure 1. Forest plot shows vigabatrin improves seizure freedom versus comparators, with consistent benefits across RCTs and observational studies; no added advantage with combination therapy.