Evidence map›Paper›PMID 42389070›Full record

ReviewEpilepsy & behavior reports2026

Fenfluramine for seizures in Dravet syndrome and Lennox-Gastaut syndrome: Mechanisms, clinical evidence, safety, and practical integration.

József Janszky, József Janszky, Réka Horváth

Abstract readReview
In one paragraph

Review in Epilepsy & behavior reports, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

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.

2 · The registry

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.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

0 citing papers in PubMed.

No citing paper in PubMed yet.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

3 authors.

József JanszkyDepartment of Neurology, Medical School, Clinical Centre, University of Pécs, Rét u. 2, 7623 Pécs, Hungary.
József JanszkyDepartment of Psychiatry and Psychotherapy, Medical School, Clinical Centre, University of Pécs, Rét u. 2, 7623 Pécs, Hungary.
Réka HorváthDepartment of Neurology, Medical School, Clinical Centre, University of Pécs, Rét u. 2, 7623 Pécs, Hungary.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Developmental and epileptic encephalopathies such as Dravet syndrome and Lennox-Gastaut syndrome remain highly drug resistant and are associated with substantial neurodevelopmental, behavioral, and caregiver burdens. Fenfluramine, repurposed at low antiseizure doses, is approved as adjunctive therapy for both syndromes and has a distinct multimodal mechanism that combines serotonergic modulation with positive allosteric activity at the sigma-1 receptor. This narrative review summarizes key translational insights and the pivotal clinical trial evidence in Dravet syndrome and Lennox-Gastaut syndrome, including long-term open-label extension and emerging real-world data. Across randomized studies, fenfluramine reduces convulsive seizures in Dravet syndrome and drop seizures in Lennox-Gastaut syndrome, with clinically meaningful responder rates and durability over time. Particular attention is given to neurocognitive and behavioral outcomes, as caregiver-reported improvements in executive function have been observed in Dravet cohorts and may not be fully explained by seizure reduction alone. Given historical associations between high-dose fenfluramine and cardiopulmonary adverse effects, we also review cardiovascular safety findings at antiseizure doses and the rationale for baseline and periodic echocardiographic monitoring. Finally, practical dosing, drug-drug interaction considerations, and an integration algorithm are provided to support routine clinical implementation across pediatric and adult care pathways.

Indexed as

antiseizure medicationDravet syndromeechocardiographyfenfluramineLennox-Gastaut syndromeserotoninsigma-1 receptor

Identifiers

PMID42389070
PMCPMC13320519

What OpenQuestion holds

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Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.