Practical considerations for the use of fenfluramine to manage patients with Dravet syndrome or Lennox-Gastaut syndrome in clinical practice

被引：4

作者：

Wirrell, Elaine C. ^{[1
]}

Lagae, Lieven ^{[2
]}

Scheffer, Ingrid E. ^{[3
,4
]}

Cross, J. Helen ^{[5
,6
]}

Specchio, Nicola ^{[7
]}

Strzelczyk, Adam ^{[8
,9
]}

机构：

[1] Mayo Clin, Dept Neurol, Div Child & Adolescent Neurol & Epilepsy, 200 1st St SW, Rochester, MN 55905 USA

[2] Univ Leuven, Dept Pediat Neurol, European Reference Network EpiCARE, Leuven, Belgium

[3] Univ Melbourne, Austin Hosp, Florey & Murdoch Childrens Res Inst, Melbourne, Vic, Australia

[4] Univ Melbourne, Royal Childrens Hosp, Florey & Murdoch Childrens Res Inst, Melbourne, Vic, Australia

[5] UCL NIHR BRC Great Ormond St Inst Child Hlth, Dev Neurosci Res & Teaching Dept, London, England

[6] Great Ormond St Hosp Sick Children, Dept Neurol, London, England

[7] SIPSIC, Rome, Italy

[8] Univ Hosp Frankfurt, Goethe Univ Frankfurt, Epilepsy Ctr Frankfurt Rhine Main, Frankfurt, Germany

[9] Univ Hosp Frankfurt, Dept Neurol, Frankfurt, Germany

来源：

EPILEPSIA OPEN | 2024年 / 9卷 / 05期

关键词：

antiseizure medications; Dravet syndrome; fenfluramine; Lennox-Gastaut syndrome; polytherapy; SEVERE MYOCLONIC EPILEPSY; ANTI-SEIZURE MEDICATIONS; DRUG-INTERACTIONS; TERM EFFICACY; RISK-FACTORS; INFANCY; METAANALYSIS; POPULATION; DIAGNOSIS;

D O I：

10.1002/epi4.12998

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

Fenfluramine (FFA), an antiseizure medication (ASM) with serotonergic and sigma-1 receptor activity, is used to manage patients with developmental and epileptic encephalopathies (DEEs). It is approved in the US for treating seizures associated with Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS) in patients >= 2 years old and as add-on therapy for seizures associated with DS and LGS in the EU, UK, and Japan in similarly aged patients. Consensus guidelines for treatment of DS have recommended FFA to be an early-line ASM, and it has also shown efficacy in managing seizures associated with LGS. DS and LGS are DEEs associated with a range of seizure types, developmental impairments, and multiple comorbidities. Here we provide case vignettes describing 4 patients (3 DS and 1 LGS) aged 4-29 years old in whom up to 14 ASMs had previously failed, to illustrate real-world practice issues encountered by neurologists. This review provides guidance on the use of FFA in the context of ASM polytherapy and drug-drug interactions (DDIs), behavioral issues, dose titration, and adverse events. Along with data from the clinical trial program, these case vignettes emphasize the low risk of DDIs, a generally well-tolerated safety profile, and other seizure and nonseizure benefits (eg, improved cognition and sleep) associated with the use of FFA in DS or LGS.Plain Language SummaryFenfluramine is used to treat seizures in individuals with Dravet syndrome and Lennox-Gastaut syndrome, but there are a range of issues that clinicians may face when treating patients. This review highlights four patients from the authors' everyday clinical work and offers guidance and practical considerations by neurologists with expertise in managing these complex conditions related to drug interactions, dosing, and side effects associated with fenfluramine. This is a graphical representation of the abstract and article highlights. image

引用

页码：1643 / 1657

页数：15

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