R. Nabbout, Arun Mistry, S. Zuberi
Dec 2, 2019
Citations
10
Influential Citations
135
Citations
Quality indicators
Journal
JAMA Neurology
Abstract
Key Points Question Is fenfluramine safe and effective for treating patients with Dravet syndrome who have frequent seizures despite taking a stiripentol-inclusive antiepileptic drug regimen? Findings Oral fenfluramine (0.4 mg/kg/d; maximum 17 mg/d) provided a 54.0% greater reduction in mean monthly convulsive seizure frequency than placebo in patients with Dravet syndrome who were taking stiripentol-containing antiepileptic drug regimens; a significantly greater proportion of patients who were taking fenfluramine (vs placebo) experienced a clinically meaningful (≥50%) or profound (≥75%) reduction in monthly convulsive seizure frequency. The most common adverse events included decreased appetite, pyrexia, fatigue, and diarrhea; no patient developed valvular heart disease or pulmonary hypertension. Meaning Adjunctive fenfluramine may be a safe, effective new treatment option for patients with Dravet syndrome with seizures that are not controlled by a regimen including stiripentol.