| Title: |
Fenfluramine for Treatment-Resistant Seizures in Patients With Dravet Syndrome Receiving Stiripentol-Inclusive Regimens A Randomized Clinical Trial |
| Authors: |
Nabbout, R. (Rima); Mistry, A. (Arun); Zuberi, S. (Sameer); Villeneuve, N. (Nathalie); Perry, M.S. (M. Scott); Sanchez-Carpintero, R. (Rocío); Stephani, U. (Ulrich); Laux, L. (Linda); Wirrell, E. (Elaine); Knupp, K. (Kelly); Chiron, C. (Catherine); Farfel, G. (Gail); Galer, B.S. (Bradley S.); Morrison, G. (Glenn); Lock, M. (Michael); Agarwal, A. (Anupam); Auvin, S. (Stéphane) |
| Publisher Information: |
USA |
| Publication Year: |
2020 |
| Collection: |
dadun - Depósito Académico Digital Universidad de Navarra |
| Description: |
IMPORTANCE Fenfluramine treatment may reduce monthly convulsive seizure frequency in patients with Dravet syndrome who have poor seizure control with their current stiripentol-containing antiepileptic drug regimens. OBJECTIVE To determine whether fenfluramine reduced monthly convulsive seizure frequency relative to placebo in patients with Dravet syndrome who were taking stiripentol-inclusive regimens. DESIGN, SETTING, AND PARTICIPANTS This double-blind, placebo-controlled, parallel-group randomized clinical trial was conducted in multiple centers. Eligible patients were children aged 2 to 18 years with a confirmed clinical diagnosis of Dravet syndrome who were receiving stable, stiripentol-inclusive antiepileptic drug regimens. INTERVENTIONS Patients with 6 or more convulsive seizures during the 6-week baseline period were randomly assigned to receive fenfluramine, 0.4 mg/kg/d (maximum, 17 mg/d), or a placebo. After titration (3 weeks), patients’ assigned dosages were maintained for 12 additional weeks. Caregivers recorded seizures via a daily electronic diary. MAIN OUTCOMES AND MEASURES The primary efficacy end point was the change in mean monthly convulsive seizure frequency between fenfluramine and placebo during the combined titration and maintenance periods relative to baseline. RESULTS A total of 115 eligible patients were identified; of these, 87 patients (mean [SD], age 9.1 [4.8] years; 50 male patients [57%]; mean baseline frequency of seizures, approximately 25 convulsive seizures per month) were enrolled and randomized to fenfluramine, 0.4 mg/kg/d (n = 43) or placebo (n = 44). Patients treated with fenfluramine achieved a 54.0% (95% CI, 35.6%-67.2%; P < .001) greater reduction in mean monthly convulsive seizure frequency than those receiving the placebo. With fenfluramine, 54% of patients demonstrated a clinically meaningful (50%) reduction in monthly convulsive seizure frequency vs 5% with placebo (P < .001). The median (range) longest seizure-free interval was 22 (3.0-105.0) days with ... |
| Document Type: |
article in journal/newspaper |
| File Description: |
application/pdf |
| Language: |
English |
| Relation: |
https://hdl.handle.net/10171/66275 |
| Availability: |
https://hdl.handle.net/10171/66275 |
| Rights: |
info:eu-repo/semantics/openAccess |
| Accession Number: |
edsbas.4C86C115 |
| Database: |
BASE |