Efficacy and tolerability of add-on stiripentol in real-world clinical practice: An observational study in Dravet syndrome and non-Dravet developmental and epileptic encephalopathies

• Published in: Epilepsia open 2024-02, Vol.9 (1), p.164-175
• Main Authors: Gil-Nagel, Antonio, Aledo-Serrano, Angel, Beltrán-Corbellini, Álvaro, Martínez-Vicente, Laura, Jimenez-Huete, Adolfo, Toledano-Delgado, Rafael, Gacía-Morales, Irene, Valls-Carbó, Adrián
• Format: Article
• Language: English
• Subjects: Adolescent; Adult; Anticonvulsants - therapeutic use; antiseizure medication; Child; Child, Preschool; Clinical medicine; Convulsions & seizures; developmental and epileptic encephalopathy; Dioxolanes - therapeutic use; Dravet syndrome; Drug dosages; Epilepsies, Myoclonic - drug therapy; Epilepsy; Humans; Infant; Middle Aged; Observational studies; Original; Patients; Retention; Seizures - drug therapy; status epilepticus; stiripentol; Survival analysis; Young Adult
• ISSN: 2470-9239; 2470-9239
• DOI: 10.1002/epi4.12847

To assess efficacy and tolerability of stiripentol (STP) as adjunctive treatment in Dravet syndrome and non-Dravet refractory developmental and epileptic encephalopathies (DREEs). Retrospective observational study of all children and adults with DREE and prescribed adjunctive STP at Hospital Ruber Internacional from January 2000 to February 2023. Outcomes were retention rate, responder rate (proportion of patients with ≥50% reduction in total seizure frequency relative to baseline), seizure freedom rate, responder rate for status epilepticus, rate of adverse event and individual adverse events, reported at 3, 6, and 12 months and at final visit. Seizure outcomes are reported overall, and for Dravet and non-Dravet subgroups. A total of 82 patients (55 Dravet syndrome and 27 non-Dravet DREE) were included. Median age was 5 years (range 1-59 years), and median age of epilepsy onset was younger in the Dravet group (4.9 [3.6-6] months) than non-Dravet (17.9 [6-42.3], P