Authors :
Presenting Author: Antonio Gil-Nagel, MD, PhD – Department of Neurology, Hospital Ruber Internacional, Madrid, Spain
Adrián Valls-Carbó, MD, PhD – Fundación INCE (Iniciativa para las Neurociencias); Madrid; Spain; Irene García-Morales, MD – Department of Neurology, Hospital Ruber Internacional, Madrid, Spain; Álvaro Beltrán-Corbellini, MD – Department of Neurology, Hospital Ruber Internacional, Madrid, Spain; Rafael Toledano-Delgado, MD – Department of Neurology, Hospital Ruber Internacional, Madrid, Spain
Rationale:
Stiripentol (STP) is FDA approved for Dravet patients six months of age and older but some small case series have also suggested benefit for other refractory developmental and epileptic encephalopathies (RDEEs). Given the limited treatment options for early onset RDEEs this study was designed to evaluate the outcomes of STP used in real world clinical practice in patients with Dravet and non-Dravet RDEEs
Methods:
In this observational study, data for all children and adults with DREEs who received STP as add-on therapy at Hospital Ruber International (Madrid) from January 2000 to February 2023 were retrospectively collected. Retention rate, seizure outcomes and safety information were reported at three, six, and 12 months, and at final visit. Outcomes are reported overall, and for Dravet and other RDEEs. Non-Dravet REE patients were sub-classified based on their epilepsy etiology, according to the International League Against Epilepsy (ILAE) operational classification.
Results:
Eighty-two patients (55 Dravet syndrome and 27 other RDEEs) were included. Among the non-Dravet RDEE patients, eight had Lennox-Gastaut syndrome, three had Doose syndrome, and two had malignant migrating partial epilepsy of infancy. Median age was five 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< 0.001). Median duration of treatment with STP was 24.8 months (2 years; range 0.3-164 months), and was longer in the Dravet group (35.9 months; range 0.8-164) than non-Dravet (17 months range 0.3-62.3, p< 0.001). At 12 months, retention rate, responder rate and seizure free rate were 68.3% (56/82), 55% [36-68%] and 18% [5.7-29%], respectively. There were no statistically significant differences between groups on these seizure outcomes. A seizure-free status was achieved in zero out of eight Lennox patients, in two out of three Doose syndrome patients, and in one out of two patients with malignant migrating partial epilepsy of infancy. Adverse events were reported in 46.3% of patients (38/82), without differences between groups.