Use of Stiripentol in Patients with Dravet Syndrome: Common Practice Among Experts in Spain
Abstract Background Despite considerable evidence for the efficacy and safety of stiripentol in Dravet syndrome (DS), some aspects of stiripentol use remain challenging in clinical practice, such as dose titration and the adjustment of concomitant antiseizure medications (ASMs) to prevent potential...
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Adis, Springer Healthcare
2024-11-01
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| Series: | Neurology and Therapy |
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| Online Access: | https://doi.org/10.1007/s40120-024-00677-8 |
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| author | Juan José García-Peñas Rocío Calvo-Medina Adrián García-Ron Antonio Gil-Nagel Vicente Villanueva Rocío Sánchez-Carpintero |
| author_facet | Juan José García-Peñas Rocío Calvo-Medina Adrián García-Ron Antonio Gil-Nagel Vicente Villanueva Rocío Sánchez-Carpintero |
| author_sort | Juan José García-Peñas |
| collection | DOAJ |
| description | Abstract Background Despite considerable evidence for the efficacy and safety of stiripentol in Dravet syndrome (DS), some aspects of stiripentol use remain challenging in clinical practice, such as dose titration and the adjustment of concomitant antiseizure medications (ASMs) to prevent potential adverse effects. Aim To (1) provide practical recommendations on the initiation of stiripentol treatment in patients with DS, (2) evaluate its effectiveness in the patient, and (3) guide the management of drug interactions and other aspects of treatment monitoring. Methods Six Spanish neurologists (the authors) with expertise in the management of pediatric and adult patients with DS held a meeting in early 2024 to develop expert recommendations regarding the use of stiripentol in DS, based on a review of the literature and their common clinical experience. Results According to these recommendations, stiripentol can be administered to patients with DS of any age, although its initiation and titration vary according to age group. Individualized adjustment of concomitant ASMs, such as valproic acid and clobazam or drugs specifically for DS (i.e., fenfluramine), at initiation and during stiripentol treatment, can mitigate drug interactions, thereby increasing the long-term tolerability of stiripentol treatment. In specific cases, stiripentol doses of > 50 mg/kg/day may be contemplated, and acute stiripentol administration may be considered to control refractory status epilepticus. Blood tests should be performed before starting stiripentol, at 3, 6, and 12 months after starting treatment, and then annually, except in the event of adverse effects, when additional testing may be necessary. Most adverse effects can be adequately managed by adjusting concomitant medications. Conclusion These practical recommendations may be easily adapted for use in different countries, and should increase physicians’ confidence in the initiation and monitoring of stiripentol treatment, thus facilitating effective management of patients with DS and improving clinical outcomes. |
| format | Article |
| id | doaj-art-cf6c7cb5005f41f49352787a2685dd6f |
| institution | Kabale University |
| issn | 2193-8253 2193-6536 |
| language | English |
| publishDate | 2024-11-01 |
| publisher | Adis, Springer Healthcare |
| record_format | Article |
| series | Neurology and Therapy |
| spelling | doaj-art-cf6c7cb5005f41f49352787a2685dd6f2025-01-26T12:58:42ZengAdis, Springer HealthcareNeurology and Therapy2193-82532193-65362024-11-01141274310.1007/s40120-024-00677-8Use of Stiripentol in Patients with Dravet Syndrome: Common Practice Among Experts in SpainJuan José García-Peñas0Rocío Calvo-Medina1Adrián García-Ron2Antonio Gil-Nagel3Vicente Villanueva4Rocío Sánchez-Carpintero5Unidad de Epilepsia, Sección de Neurología, Hospital Infantil Universitario Niño JesúsUnidad de Neuropediatría, Hospital Regional Universitario de MálagaUnidad del Niño y del Adolescente, Hospital Clínico San CarlosPrograma de Epilepsia, Hospital Ruber InternacionalUnidad de Epilepsia Refractaria, Hospital Universitario y Politécnico La Fe. Member of ERN EpicareUnidad de Neurología Pediátrica, Clínica Universidad de Navarra. Member of the Instituto de Investigación Sanitaria de Navarra (IdisNa)Abstract Background Despite considerable evidence for the efficacy and safety of stiripentol in Dravet syndrome (DS), some aspects of stiripentol use remain challenging in clinical practice, such as dose titration and the adjustment of concomitant antiseizure medications (ASMs) to prevent potential adverse effects. Aim To (1) provide practical recommendations on the initiation of stiripentol treatment in patients with DS, (2) evaluate its effectiveness in the patient, and (3) guide the management of drug interactions and other aspects of treatment monitoring. Methods Six Spanish neurologists (the authors) with expertise in the management of pediatric and adult patients with DS held a meeting in early 2024 to develop expert recommendations regarding the use of stiripentol in DS, based on a review of the literature and their common clinical experience. Results According to these recommendations, stiripentol can be administered to patients with DS of any age, although its initiation and titration vary according to age group. Individualized adjustment of concomitant ASMs, such as valproic acid and clobazam or drugs specifically for DS (i.e., fenfluramine), at initiation and during stiripentol treatment, can mitigate drug interactions, thereby increasing the long-term tolerability of stiripentol treatment. In specific cases, stiripentol doses of > 50 mg/kg/day may be contemplated, and acute stiripentol administration may be considered to control refractory status epilepticus. Blood tests should be performed before starting stiripentol, at 3, 6, and 12 months after starting treatment, and then annually, except in the event of adverse effects, when additional testing may be necessary. Most adverse effects can be adequately managed by adjusting concomitant medications. Conclusion These practical recommendations may be easily adapted for use in different countries, and should increase physicians’ confidence in the initiation and monitoring of stiripentol treatment, thus facilitating effective management of patients with DS and improving clinical outcomes.https://doi.org/10.1007/s40120-024-00677-8Dravet syndromeEpilepsyEvidence-based recommendationsQuality of lifeSeizure reductionSafety |
| spellingShingle | Juan José García-Peñas Rocío Calvo-Medina Adrián García-Ron Antonio Gil-Nagel Vicente Villanueva Rocío Sánchez-Carpintero Use of Stiripentol in Patients with Dravet Syndrome: Common Practice Among Experts in Spain Neurology and Therapy Dravet syndrome Epilepsy Evidence-based recommendations Quality of life Seizure reduction Safety |
| title | Use of Stiripentol in Patients with Dravet Syndrome: Common Practice Among Experts in Spain |
| title_full | Use of Stiripentol in Patients with Dravet Syndrome: Common Practice Among Experts in Spain |
| title_fullStr | Use of Stiripentol in Patients with Dravet Syndrome: Common Practice Among Experts in Spain |
| title_full_unstemmed | Use of Stiripentol in Patients with Dravet Syndrome: Common Practice Among Experts in Spain |
| title_short | Use of Stiripentol in Patients with Dravet Syndrome: Common Practice Among Experts in Spain |
| title_sort | use of stiripentol in patients with dravet syndrome common practice among experts in spain |
| topic | Dravet syndrome Epilepsy Evidence-based recommendations Quality of life Seizure reduction Safety |
| url | https://doi.org/10.1007/s40120-024-00677-8 |
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