Therapeutic Maintenance of Stiripentol in Dravet Syndrome: A Comprehensive Literature Analysis
Abstract number :
1.414
Submission category :
7. Anti-seizure Medications / 7C. Cohort Studies
Year :
2024
Submission ID :
1362
Source :
www.aesnet.org
Presentation date :
12/7/2024 12:00:00 AM
Published date :
Authors :
Presenting Author: Rima Nabbout, MD, PhD – Member of European Reference Network EpiCARE, Reference Centre for Rare Epilepsies, Necker Enfants Malades Hospital, APHP, U 1163 Institut Imagine, Université Paris Cité
Stéphane Auvin, MD, PhD, FAES – Paris-Cité University & Robert-Debré University Hospital, Paris, France
Andreas Brunklaus, MD – Royal Hospital for Children
J. Helen Cross, MBChB, PhD – UCL-Great Ormond Street Institute of Child Health; Great Ormond Street Hospital
Antonio Gil-Nagel, MD, PhD – Hospital Ruber Internacional
Renzo Guerrini, MD, FRCP, FAES – Meyer Children’s Hospital IRCCS, and University of Florence
Nicola Specchio, MD, PhD – Bambino Gesu’ Children’s Hospital, IRCCS, Full Member of European Reference Network on Rare and Complex Epilepsies EpiCARE
Adam Strzelczyk, MD, MHBA, FEAN – Goethe-University Frankfurt
Vincente Villanueva, MD, PhD – Hospital Universitario y Politécnico La Fe
Ingrid Scheffer, MBBS, PhD, FRACP, FRS – University of Melbourne, Austin Hospital and Royal Children's Hospital, Florey and Murdoch Children’s Research Institutes
Rationale: Dravet syndrome (DS), a severe developmental and epileptic encephalopathy beginning in infancy, is characterized by drug-resistant seizures despite the use of standard antiseizure medications (Epilepsia. 2022;63(7):1761–77.). Stiripentol (STP) is a first-line add-on therapy to the broad-spectrum anti-seizure medications valproate (VPA) and clobazam (CLB) for DS (Epilepsia. 2022;63(7):1761–77.; CNS Drugs. 2022;36(3):217–37; Adv Ther. 2024;41(4):1351–71.). STP has been widely used in clinical practice since its approval for the treatment of DS in 2007. This analysis aims to provide comprehensive data on long-term use of STP in patients with DS as well as reasons for discontinuation in real-world therapeutic settings. Understanding these aspects can guide clinicians in optimizing management strategies for patients with DS.
Methods: A search of real-life evidence studies from the last decade was conducted on Pubmed, excluding randomized controlled trials (RCTs) due to their limited treatment duration. Data from nine studies were analyzed (764 patients). The analysis included treatment duration, retention rates, responder rates (defined as 50% or higher reduction in generalized tonic-clonic seizures (GTCs)), age of treatment initiation and reasons for discontinuation which were categorized into short-term (< 6 months), medium-term (6-12 months), and long-term ( >12 months).
Results: Overall, the median or mean age of STP initiation ranged from 1.1 to 26 years across the studies and the median or mean duration of treatment ranged from 1.1 to 18.2 years. The mean retention rate across all studies was 79% (range 54-100%) with a mean responder rate of 48% (range 31-66%). The primary reason for discontinuation of STP in the short, medium, and long-term was lack of treatment efficacy, followed by adverse events. A retrospective study showed that the patients on STP from infancy/childhood (aged < 15 years) tended to have better seizure outcome in mid-adulthood than the patients treated from adolescence ( >15 years).
Conclusions: Despite the disparity in published data, STP demonstrated high retention rates at long-term follow-up, especially when initiated in childhood. The high level of long-term use is likely to be due to STP efficacy and tolerability in patients with DS.
Funding: This study received institutional support from the Biocodex Laboratory.
Anti-seizure Medications