Use of fenfluramine in MECP2-related Rett syndrome: Findings from a retrospective multicenter pediatric case series - PubMed

3 hours ago

Fenfluramine (FFA) was studied in pediatric patients with MECP2-related Rett syndrome (RTT) and drug-resistant epilepsy.
The study included four patients (mean age 11 years) treated with FFA for an average of 10.5 months.
Three out of four patients showed significant seizure reduction (>50% in two cases), particularly in tonic-clonic seizures.
One patient discontinued FFA due to lack of improvement.
Adverse effects (apathy, psychomotor slowing) were reported in one patient but resolved after temporary discontinuation.
EEG improvements were noted in responders, with reduced interictal abnormalities.
No cardiac adverse events were observed.
Behavioral improvements (alertness, interaction, reduced irritability) were reported in two patients.
FFA was generally well-tolerated, with minimal side effects despite polytherapy.
Prospective studies with larger cohorts are needed to validate efficacy, safety, and cognitive-behavioral outcomes.

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