No single treatment modality exhibits clear superiority for Infantile Spasms Syndrome: insights from a retrospective cohort study.

Abstract Background: Infantile spasms syndrome is a severe form of infantile epilepsy. It is commonly treated with hormonal therapies or vigabatrin, either alone or in combination. This study aimed to assess the efficacy of these treatment modalities and explore associations with aetiology, and pre-existing developmental delay. Methods: Patients diagnosed with and treated for infantile spasms syndrome at the University Hospital of Leuven between 1 January 2018 and 23 November 2022, were identified. Retrospective clinical data were analysed descriptively. Short-term and long-term outcomes determined the efficacy of the current treatments, with seizure freedom and sustained normal development at 6 and 12 months after treatment initiation as primary treatment goals. Results: The study included 26 patients. No single treatment modality demonstrated clear superiority in terms of both short-term and long-term outcomes. Patients with unknown aetiology showed better developmental outcomes after 12 months than those with proven aetiology (30.77% with normal development versus 7.69%.) Another clear difference in treatment efficacy emerged between patients with and without pre-existing developmental issues, with only 6.67% achieving normal development after 12 months compared to 36.36%, respectively. Conclusion: No single treatment modality demonstrated clear superiority. Associations were found between treatment efficacy and aetiology as well as pre-existing developmental delay, suggesting these as potential prognostic indicators.