Authors :
First Author: Stéphane Auvin, MD, PhD – Service de Neurologie Pédiatrique, Hôpital Robert Debré, Paris, France
Presenting Author: Amélie Lothe, PhD – Zogenix International Limited
Ingrid Scheffer, MBBS, PhD, FRACP, FRS – University of Melbourne, Austin Hospital and Royal Children’s Hospital; Antonio Gil-Nagel, MD, PhD – Hospital Ruber Internacional; Amélie Lothe, PhD – UCB Pharma, S.A.; Shikha Polega, PharmD – UCB Pharma; Lieven Lagae, MD – Member of the European Reference Network EpiCARE; Department of Paediatric Neurology, University of Leuven; Kelly Knupp, MD, MSCS, FAES – University of Colorado, Children’s Hospital Colorado
Rationale: Fenfluramine (FFA) is approved for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS) in patients ≥ 2 years of age in the US and Europe. In a placebo-controlled, phase 3, randomized clinical trial (RCT), LGS patients treated with the recommended dose of 0.7 mg/kg/day FFA had a significant reduction in seizures associated with a fall, sustained in the open-label extension (OLE). Additional analysis of seizure-free days may provide insights regarding seizure control and quality of life. A post-hoc analysis evaluated the percentage of seizure-free days in patients with LGS treated with FFA in the 14-week RCT and its one year OLE.
Methods:
After a four week baseline (BL) period in the RCT, LGS patients aged 2-35 years were randomized to FFA 0.7mg/kg/day, FFA 0.2mg/kg/day, or placebo (max: 26mg/day). After two weeks’ titration, assigned doses were maintained for 12 weeks (titration and maintenance [T+M], 14 weeks). In the OLE, patients initiated FFA at 0.2mg/kg/day for one month. Thereafter, doses were titrated based on effectiveness and tolerability. In the OLE, FFA dose groups were defined as < 0.3 mg/kg/day, 0.3-0.5 mg/kg/day, and >