Observational studyEpilepsia open2026
Fenfluramine in SCN1A-related GEFS+: A multicenter observational study on efficacy, EEG improvement, and tolerability.
Observational study in Epilepsia open, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
2 citing papers in PubMed.
- Fenfluramine in SCN1A-related GEFS+: A multicenter observational study on efficacy, EEG improvement, and tolerability.Epilepsia open · 2026Observational
- The genotypic spectrum of complex febrile seizures: insights from high-risk population genetic screening in a pediatric cohort.Frontiers in neuroscience · 2026Article
Corrections and comments
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Authors and funding
13 authors.
Funding
Abstract
The SCN1A gene is implicated in a broad spectrum of epilepsy phenotypes, ranging from self-limited genetic epilepsy with febrile seizures plus (GEFS+) to severe developmental and epileptic encephalopathies such as Dravet syndrome (DS). While fenfluramine (FFA) has demonstrated strong efficacy in DS, its role in SCN1A-related epilepsies beyond DS has not been thoroughly investigated. We conducted a multicenter observational study including 11 patients with SCN1A-related GEFS+ who received FFA as adjunctive therapy. All patients had previously failed to achieve adequate seizure control with valproate and, in most cases, additional antiseizure medications. FFA was introduced following the DS titration protocol, with a mean dose of 0.39 mg/kg/day. FFA addition led to a mean seizure frequency reduction of 91%, with more than half of the patients achieving complete seizure freedom. Reduced EEG abnormalities were documented in 5/11 patients of the cohort, including complete normalization in 3/11 patients. Furthermore, subjective caregiver reports indicated perceived improvements in patients' alertness and behavioral responses. FFA was well tolerated, with only mild and transient adverse events reported. These findings support the potential role of FFA as an effective and well-tolerated treatment option in patients with SCN1A-related GEFS+. PLAIN LANGUAGE SUMMARY: GEFS+ is a genetic epilepsy frequently caused by changes in the SCN1A gene. In a multicenter real-world study of 11 people with SCN1A-related GEFS+, adding fenfluramine to usual care substantially reduced seizures, with several becoming seizure-free. EEG recordings improved, and caregivers reported better alertness in some patients. Treatment was generally well tolerated, with only mild, temporary side effects.
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