학술논문
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
Document Type
article
Author
Source
Epilepsia Open, Vol 9, Iss 1, Pp 164-175 (2024)
Subject
Language
English
ISSN
2470-9239
Abstract
Abstract Objective To assess efficacy and tolerability of stiripentol (STP) as adjunctive treatment in Dravet syndrome and non‐Dravet refractory developmental and epileptic encephalopathies (DREEs). Methods 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. Results 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