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.
Keywords: Dravet syndrome; Epilepsy; Evidence-based recommendations; Quality of life; Safety; Seizure reduction; Stiripentol; Tolerability.
In this article, six Spanish neurologists with expertise in the treatment of individuals with Dravet syndrome (DS) developed recommendations based on a review of the academic literature and their common clinical experience on the use of stiripentol in the treatment of this syndrome. DS is a rare and severe form of lifelong genetic epilepsy that first develops in infancy, impairs neurologic and psychomotor development, and increases the risk of premature death. Stiripentol is an antiseizure medication for which there is considerable evidence of its efficacy and safety as a treatment for DS, but dose titration and its use in combination with other antiseizure medications to minimize potential adverse effects remain challenging in clinical practice. The expert recommendations presented here provide neurologists with practical guidance on the effective initiation of stiripentol treatment, assessment of its effectiveness in patients with DS, and guidance on the management and monitoring of its potential interaction with other medications. It is hoped that these recommendations will facilitate the effective management of patients with DS and improve their clinical outcomes.
© 2024. The Author(s).