Abstract Details

Title Real-World Use of Fenfluramine for Dravet Syndrome: a Retrospective Cohort Study Using a National Pharmacy Database

Topic Epilepsy/Clinical Neurophysiology (EEG)

Presentation(s) P12 - Poster Session 12 (11:45 AM-12:45 PM)

Poster/Presentation
Number 9-002

Objective This study describes mortality rates and real-world use of fenfluramine (FFA) in United States (US) patients with Dravet syndrome (DS) who were new FFA users, obtained from a single specialty pharmacy.

Background Patients with DS are at risk of premature mortality, with all-cause mortality rates previously reported as 8.6-15.8/1000 person-years (Cooper Epilepsy Res 2016; Donnan Neurology 2023). In the US, FFA is approved for managing seizures in patients ≥2 years old with DS. A prior post-hoc analysis of FFA studies demonstrated lower all-cause mortality rates in FFA-treated patients compared to expected rates (Cross Seizure 2021).

Design/Methods This retrospective study evaluated patients with DS who were new FFA users and received their first shipment ≥6 months prior to the data cut. Outcomes include demographics, FFA doses, persistence rates by index year, and mortality rates. Standardized mortality ratios (SMRs) with 95% confidence intervals (CIs) for 2021-2023 were reported. Descriptive statistics were used.

Results From 07/01/2020 to 3/15/2024, 1043 patients with DS were new FFA users; 49.2% were 6-17 years old, 56.6% were prescribed FFA by pediatric epileptologists, and 56.0% had previously taken cannabidiol. Initial median FFA dose was 0.3 mg/kg/day (range: 0.1-1.1 mg/kg/day) and median maintenance dose was 0.5 mg/kg/day (range: 0.02-1.4 mg/kg/day). Persistence rates at 6 and 12 months, respectively, were: 90.9% and 78.5% in 2020, 85.8% and 76.9% in 2021, 83.5% and 74.8% in 2022. Median treatment duration was 21.3 months; 729 patients continued on FFA at data cut. There were 16 deaths over 1865 patient-years, resulting in an estimated mortality rate of 8.6/1000 person-years (95% CI: 5.6-11.6); SMR from 2021-2023 was 2.0 (95% CI: 1.1-3.3).

Conclusions This real-world analysis revealed that mortality rates in patients with DS treated with FFA were similar to previous studies and demonstrated an encouraging SMR. Further studies will evaluate causes of death, including sudden unexpected death in epilepsy.

Authors/Disclosures
Wesley Kerr, MD, PhD (University of Pittsburgh) PRESENTER	Dr. Kerr has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for SK Lifesciences. Dr. Kerr has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biohaven Pharmaceuticals. Dr. Kerr has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UCB. Dr. Kerr has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Neurelis. Dr. Kerr has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for QurAlis. Dr. Kerr has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biohaven Pharmaceuticals. Dr. Kerr has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Epilepsia. The institution of Dr. Kerr has received research support from NINDS. The institution of Dr. Kerr has received research support from American Epilepsy Society. The institution of Dr. Kerr has received research support from ��ɫ��. The institution of Dr. Kerr has received research support from SK Life Science. The institution of Dr. Kerr has received research support from Biohaven Pharmaceuticals. Dr. Kerr has received publishing royalties from a publication relating to health care.
Samden Lhatoo, MD (University Hospitals Case Medical Center)	Dr. Lhatoo has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for LivaNova. The institution of Dr. Lhatoo has received research support from NIH. Dr. Lhatoo has received intellectual property interests from a discovery or technology relating to health care. Dr. Lhatoo has received publishing royalties from a publication relating to health care.
Elaine C. Wirrell, MD, FAAN (Mayo Clinic/Dept of Child Neurology)	Dr. Wirrell has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Biocodex. Dr. Wirrell has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Encoded. Dr. Wirrell has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Neurocrine. Dr. Wirrell has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for GRIN. Dr. Wirrell has received publishing royalties from a publication relating to health care.
Elizabeth Donner, MD, FRCPC (The Hospital for Sick Children)	Dr. Donner has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Eisai. Dr. Donner has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UCB. Dr. Donner has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Pendopharm. Dr. Donner has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Seizure. The institution of Dr. Donner has received research support from SickKids Centre for Brain and Mental Health. The institution of Dr. Donner has received research support from Ontario Brain Institute. The institution of Dr. Donner has received research support from CIHR.
Joseph E. Sullivan, MD	Dr. Sullivan has received personal compensation in the range of $500-$4,999 for serving as a Consultant for BrightMinds. Dr. Sullivan has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for UCB. Dr. Sullivan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for NeuroPace. Dr. Sullivan has stock in Zynerba. The institution of Dr. Sullivan has received research support from Stoke. The institution of Dr. Sullivan has received research support from Takeda. The institution of Dr. Sullivan has received research support from Neurocrine. The institution of Dr. Sullivan has received research support from Encoded. The institution of Dr. Sullivan has received research support from Zogenix.
Renzo Guerrini (Hotelplan)	Renzo Guerrini has nothing to disclose.
Ingrid E. Scheffer, MBBS, PhD, FRACP, AO (Melbourne Brain Centre)	Dr. Scheffer has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biohaven Pharmaceuticals, Inc. Dr. Scheffer has received personal compensation in the range of $0-$499 for serving as a Consultant for Longboard Pharmaceuticals . Dr. Scheffer has received personal compensation in the range of $0-$499 for serving as a Consultant for UCB. Dr. Scheffer has received personal compensation in the range of $0-$499 for serving as a Consultant for Biocodex. Dr. Scheffer has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Bellberry Ltd. The institution of Dr. Scheffer has received research support from National Health and Medical Research Council, Australia. The institution of Dr. Scheffer has received research support from National Health and Medical Research Council, Australia. The institution of Dr. Scheffer has received research support from National Health and Medical Research Council, Australia. The institution of Dr. Scheffer has received research support from Medical Research Future Fund, Australia. The institution of Dr. Scheffer has received research support from Medical Research Future Fund, Australia. The institution of Dr. Scheffer has received research support from Medical Research Future Fund, Australia. The institution of Dr. Scheffer has received research support from Medical Research Future Fund, Australia. The institution of Dr. Scheffer has received research support from Medical Research Future Fund, Australia. The institution of Dr. Scheffer has received research support from Einstein Visiting Fellowship. The institution of Dr. Scheffer has received research support from National Health and Medical Research Council, Australia. Dr. Scheffer has received intellectual property interests from a discovery or technology relating to health care. Dr. Scheffer has received intellectual property interests from a discovery or technology relating to health care. Dr. Scheffer has received intellectual property interests from a discovery or technology relating to health care. Dr. Scheffer has received personal compensation in the range of $0-$499 for serving as a Speaker honoraria with Akumentis. Dr. Scheffer has received personal compensation in the range of $0-$499 for serving as a Speaker honoraria with Biocodex. Dr. Scheffer has received personal compensation in the range of $0-$499 for serving as a Speaker honoraria with Chiesi . Dr. Scheffer has received personal compensation in the range of $0-$499 for serving as a Travel/Conference/Speaker honoraria with Stoke Therapeutics. Dr. Scheffer has received personal compensation in the range of $0-$499 for serving as a Speaker honoraria with Zuellig Pharma Inc.. Dr. Scheffer has a non-compensated relationship as a Trial Investigator with Anavex Life Sciences that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Trial investigator with Cerebral Therapeutics that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Trial investigator with Cerecin that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Trial investigator with Cereval Therapeutics that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Trial investigator with Biohaven Ltd. that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Scientific Advisory Board, Trial investigator with Encoded Therapeutics that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Trial investigator with EpiMinder Inc that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Trial investigator with ES-Therapeutics that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Trial investigator with Bright Minds Biosciences that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Trial investigator with Marinus Pharmaceuticals that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Trial investigator with Neurocrine BioSciences that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Trial investigator with Neuren Pharmaceuticals that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Trial investigator with Praxis Precision Medicines that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Trial investigator, Scientific Advisory Board with Takeda Pharmaceuticals that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Trial investigator with Xenon Pharmaceuticals that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Trial investigator with Zogenix that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Journal Editorial Board with Neurology that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Journal Editorial Board with Epileptic Disorders that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Journal Editorial Board with Progress in Epileptic Disorders series that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Journal Editorial Board with The Lancet Neurology that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Director with Australian Academy of Health and Medical Sciences that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Director with Royal Society (Australia). that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Trial investigator with Shanghai Zhimeng Biopharma that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Trial investigator with SK Life Science that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Trial investigator with Supernus Pharmaceuticals that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Trial investigator with UCB Biopharma SRL that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Trial investigator with Ultragenyx that is relevant to AAN interests or activities.
Sylvain Rheims	No disclosure on file
J. Helen Cross (UCL-Institute of Child Health)	Dr. Cross has nothing to disclose.
Lieven G. Lagae, PhD (University Hospital Gasthuisberg)	Dr. Lagae has nothing to disclose.
Philippe Ryvlin, MD, PhD (Unit 301)	Dr. Ryvlin has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Arvelle/Angelini. The institution of Dr. Ryvlin has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for UCB pharma. The institution of Dr. Ryvlin has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Angelini pharma. The institution of Dr. Ryvlin has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for UCB pharma. The institution of Dr. Ryvlin has received research support from Swiss National Fund. The institution of Dr. Ryvlin has received research support from European Commission (Horizon Europe).
Antonio Gil-Nagel, MD (Hospital Ruber Internacional)	Dr. Gil-Nagel has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biocodex. Dr. Gil-Nagel has received personal compensation in the range of $500-$4,999 for serving as a Consultant for EISAI . Dr. Gil-Nagel has received personal compensation in the range of $0-$499 for serving as a Consultant for GW Pharma. Dr. Gil-Nagel has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Angelini Pharma. Dr. Gil-Nagel has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for EISAI. Dr. Gil-Nagel has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for GW Pharma. Dr. Gil-Nagel has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for PTC Therapeutics . Dr. Gil-Nagel has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Stoke . Dr. Gil-Nagel has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Zogenix . Dr. Gil-Nagel has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Angelini Pharma. Dr. Gil-Nagel has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for BIAL . Dr. Gil-Nagel has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for EISAI . Dr. Gil-Nagel has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for GW Pharma. Dr. Gil-Nagel has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Jazz Pharma. Dr. Gil-Nagel has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for PTC Therapeutics . Dr. Gil-Nagel has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for UCB Pharma. Dr. Gil-Nagel has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Zogenix . The institution of Dr. Gil-Nagel has received research support from Jazz Pharmaceuticals. The institution of Dr. Gil-Nagel has received research support from PTC Therapeutics. The institution of Dr. Gil-Nagel has received research support from Zogenix. The institution of Dr. Gil-Nagel has received research support from Agencia Estatal Innovacion.
Jeffrey Noebels, MD, PhD (Baylor College of Medicine)	No disclosure on file
Jenna S. Roberts, PhD	Dr. Roberts has received personal compensation for serving as an employee of UCB Pharma. Dr. Roberts has stock in UCB Pharma.
Amelie Lothe, PhD	Dr. Lothe has received personal compensation for serving as an employee of UCB. Dr. Lothe has stock in UCB.
Milena Tryfon, MSc	Mrs. Tryfon has received personal compensation for serving as an employee of UCB Pharma.
Michael Ranopa, PhD	Dr. Ranopa has stock in UCB.
Orrin Devinsky, MD, FAAN (NYU Epilepsy Center)	Dr. Devinsky has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Eisai. Dr. Devinsky has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for California Cannabis Enterprises. Dr. Devinsky has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Engage. Dr. Devinsky has received personal compensation in the range of $50,000-$99,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Privateer Holdings. Dr. Devinsky has received personal compensation in the range of $1,000,000+ for serving on a Scientific Advisory or Data Safety Monitoring board for Receptor Holdings. Dr. Devinsky has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Rettco. Dr. Devinsky has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Silver Spike. Dr. Devinsky has received personal compensation in the range of $100,000-$499,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Tevard. Dr. Devinsky has received personal compensation in the range of $1,000,000+ for serving on a Scientific Advisory or Data Safety Monitoring board for Tilray. Dr. Devinsky has received personal compensation in the range of $100,000-$499,999 for serving as an officer or member of the Board of Directors for Engage. Dr. Devinsky has received personal compensation in the range of $50,000-$99,999 for serving as an officer or member of the Board of Directors for Tevard Biosciences. Dr. Devinsky has stock in Engage. Dr. Devinsky has stock in Empatica. Dr. Devinsky has stock in California Cannabis Enterprises. Dr. Devinsky has stock in Empatica. Dr. Devinsky has stock in Silver Spike. Dr. Devinsky has stock in Docklight Brands. Dr. Devinsky has stock in Egg Rock/Papa Barkley. Dr. Devinsky has stock in Leafly. Dr. Devinsky has stock in Left Coast Ventures. Dr. Devinsky has stock in Privateer Holdings. Dr. Devinsky has stock in Receptor Holdings. Dr. Devinsky has stock in Qstate. The institution of Dr. Devinsky has received research support from GW Pharma. The institution of Dr. Devinsky has received research support from Zogenix. The institution of Dr. Devinsky has received research support from PTC Pharma. The institution of Dr. Devinsky has received research support from NIH. The institution of Dr. Devinsky has received research support from DOD. The institution of Dr. Devinsky has received research support from NIH. The institution of Dr. Devinsky has received research support from Epilepsy Foundation. The institution of Dr. Devinsky has received research support from NIH. The institution of Dr. Devinsky has received research support from NIH. The institution of Dr. Devinsky has received research support from NSF. Dr. Devinsky has received intellectual property interests from a discovery or technology relating to health care. Dr. Devinsky has received intellectual property interests from a discovery or technology relating to health care. Dr. Devinsky has received publishing royalties from a publication relating to health care.

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