Efficacy of Stiripentol Beyond Dravet Syndrome: A Retrospective Medical Record Review of Patients with Drug-Resistant Epilepsies
Abstract Introduction Stiripentol is approved as an add-on therapy with clobazam and/or valproate for seizures associated with Dravet syndrome (DS). Evidence of stiripentol efficacy in other drug-resistant epilepsies is limited. Methods This was a single-centre, retrospective, observational study of...
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Adis, Springer Healthcare
2025-05-01
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| Series: | Neurology and Therapy |
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| Online Access: | https://doi.org/10.1007/s40120-025-00755-5 |
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| author | Víctor Soto-Insuga Elena González-Alguacil María Ballarà-Petitbò Nuria Lamagrande-Casanova Anna Duat-Rodríguez Cristina Benítez-Provedo Elena Cardenal-Muñoz Juan José García-Peñas |
| author_facet | Víctor Soto-Insuga Elena González-Alguacil María Ballarà-Petitbò Nuria Lamagrande-Casanova Anna Duat-Rodríguez Cristina Benítez-Provedo Elena Cardenal-Muñoz Juan José García-Peñas |
| author_sort | Víctor Soto-Insuga |
| collection | DOAJ |
| description | Abstract Introduction Stiripentol is approved as an add-on therapy with clobazam and/or valproate for seizures associated with Dravet syndrome (DS). Evidence of stiripentol efficacy in other drug-resistant epilepsies is limited. Methods This was a single-centre, retrospective, observational study of patients aged ≤ 15 years with non-Dravet epilepsy or DS who initiated stiripentol treatment in Spain. Results The study included 18 patients with DS and 17 with non-Dravet epilepsy; 76.5% of the latter had a developmental and epileptic encephalopathy. Median (range) age at stiripentol initiation was 52 (4–180) months. Three months of add-on stiripentol provided overall improvement in seizures (number, duration and/or intensity) for 76.5% of the non-Dravet and 61.1% of the DS patients (p = 0.30), all of whom were drug-resistant to prior antiseizure medications (ASMs). Stiripentol reduced seizure frequency by ≥ 50% in 58.8% of the non-Dravet patients and 44.4% of the DS cohort (p = 0.40); 20% of all patients became seizure-free. Stiripentol reduced all seizure types in both cohorts. Kaplan–Meier survival analysis found a higher probability of sustained stiripentol efficacy in the DS cohort (120 months) than the non-Dravet cohort (16 months; p = 0.012). Stiripentol improved cognition and Clinical Global Impression scale scores in approximately 60% of all patients; sleep improved for 19.2%. Acute stiripentol treatment (maximum dose 6.7–100 mg/kg/day) initiated in five patients (four with non-Dravet epilepsy and one with DS) during refractory status epilepticus (SE) successfully resolved SE over a median 0.5 days. Adverse events, mainly mild-to-moderate, occurred in 47.1% and 41.2% of patients in the non-Dravet and DS cohorts, respectively. Six patients (35.3%) with non-Dravet epilepsy discontinued ≥ 1 other ASMs after stiripentol initiation. Conclusion Add-on stiripentol provides overall improvement in different seizure types and non-seizure manifestations for paediatric patients with drug-resistant epilepsy, including epileptic syndromes besides DS, and appeared effective in acute treatment of SE. Stiripentol was generally well tolerated. |
| format | Article |
| id | doaj-art-b20a46a5533643fc90391bef07228bbb |
| institution | DOAJ |
| issn | 2193-8253 2193-6536 |
| language | English |
| publishDate | 2025-05-01 |
| publisher | Adis, Springer Healthcare |
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| series | Neurology and Therapy |
| spelling | doaj-art-b20a46a5533643fc90391bef07228bbb2025-08-20T03:08:35ZengAdis, Springer HealthcareNeurology and Therapy2193-82532193-65362025-05-011431129115010.1007/s40120-025-00755-5Efficacy of Stiripentol Beyond Dravet Syndrome: A Retrospective Medical Record Review of Patients with Drug-Resistant EpilepsiesVíctor Soto-Insuga0Elena González-Alguacil1María Ballarà-Petitbò2Nuria Lamagrande-Casanova3Anna Duat-Rodríguez4Cristina Benítez-Provedo5Elena Cardenal-Muñoz6Juan José García-Peñas7Neurology Service, Niño Jesús University Children’s HospitalNeurology Service, Niño Jesús University Children’s HospitalNeurology Service, Niño Jesús University Children’s HospitalNeurology Service, Niño Jesús University Children’s HospitalNeurology Service, Niño Jesús University Children’s HospitalNeurology Service, Niño Jesús University Children’s HospitalMedical Affairs, BiocodexNeurology Service, Niño Jesús University Children’s HospitalAbstract Introduction Stiripentol is approved as an add-on therapy with clobazam and/or valproate for seizures associated with Dravet syndrome (DS). Evidence of stiripentol efficacy in other drug-resistant epilepsies is limited. Methods This was a single-centre, retrospective, observational study of patients aged ≤ 15 years with non-Dravet epilepsy or DS who initiated stiripentol treatment in Spain. Results The study included 18 patients with DS and 17 with non-Dravet epilepsy; 76.5% of the latter had a developmental and epileptic encephalopathy. Median (range) age at stiripentol initiation was 52 (4–180) months. Three months of add-on stiripentol provided overall improvement in seizures (number, duration and/or intensity) for 76.5% of the non-Dravet and 61.1% of the DS patients (p = 0.30), all of whom were drug-resistant to prior antiseizure medications (ASMs). Stiripentol reduced seizure frequency by ≥ 50% in 58.8% of the non-Dravet patients and 44.4% of the DS cohort (p = 0.40); 20% of all patients became seizure-free. Stiripentol reduced all seizure types in both cohorts. Kaplan–Meier survival analysis found a higher probability of sustained stiripentol efficacy in the DS cohort (120 months) than the non-Dravet cohort (16 months; p = 0.012). Stiripentol improved cognition and Clinical Global Impression scale scores in approximately 60% of all patients; sleep improved for 19.2%. Acute stiripentol treatment (maximum dose 6.7–100 mg/kg/day) initiated in five patients (four with non-Dravet epilepsy and one with DS) during refractory status epilepticus (SE) successfully resolved SE over a median 0.5 days. Adverse events, mainly mild-to-moderate, occurred in 47.1% and 41.2% of patients in the non-Dravet and DS cohorts, respectively. Six patients (35.3%) with non-Dravet epilepsy discontinued ≥ 1 other ASMs after stiripentol initiation. Conclusion Add-on stiripentol provides overall improvement in different seizure types and non-seizure manifestations for paediatric patients with drug-resistant epilepsy, including epileptic syndromes besides DS, and appeared effective in acute treatment of SE. Stiripentol was generally well tolerated.https://doi.org/10.1007/s40120-025-00755-5ChildrenDravet syndromeDrug-resistant epilepsyStatus epilepticusStiripentol |
| spellingShingle | Víctor Soto-Insuga Elena González-Alguacil María Ballarà-Petitbò Nuria Lamagrande-Casanova Anna Duat-Rodríguez Cristina Benítez-Provedo Elena Cardenal-Muñoz Juan José García-Peñas Efficacy of Stiripentol Beyond Dravet Syndrome: A Retrospective Medical Record Review of Patients with Drug-Resistant Epilepsies Neurology and Therapy Children Dravet syndrome Drug-resistant epilepsy Status epilepticus Stiripentol |
| title | Efficacy of Stiripentol Beyond Dravet Syndrome: A Retrospective Medical Record Review of Patients with Drug-Resistant Epilepsies |
| title_full | Efficacy of Stiripentol Beyond Dravet Syndrome: A Retrospective Medical Record Review of Patients with Drug-Resistant Epilepsies |
| title_fullStr | Efficacy of Stiripentol Beyond Dravet Syndrome: A Retrospective Medical Record Review of Patients with Drug-Resistant Epilepsies |
| title_full_unstemmed | Efficacy of Stiripentol Beyond Dravet Syndrome: A Retrospective Medical Record Review of Patients with Drug-Resistant Epilepsies |
| title_short | Efficacy of Stiripentol Beyond Dravet Syndrome: A Retrospective Medical Record Review of Patients with Drug-Resistant Epilepsies |
| title_sort | efficacy of stiripentol beyond dravet syndrome a retrospective medical record review of patients with drug resistant epilepsies |
| topic | Children Dravet syndrome Drug-resistant epilepsy Status epilepticus Stiripentol |
| url | https://doi.org/10.1007/s40120-025-00755-5 |
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