Bridging evidence gaps in dravet syndrome: real-world safety insights from under-reported antiseizure therapies.
Dravet syndrome is an early-onset developmental and epileptic encephalopathy in which management must extend beyond seizure control to include the monitoring and treatment of neurodevelopmental and systemic comorbidities.
Well-established treatments, together with recently approved agents, are discussed alongside under-reported therapies. Safety profiles, clinically relevant pharmacokinetic interactions, and practical aspects of dose titration and monitoring are reviewed. Emerging targeted pharmacological and genetic strategies are also briefly considered as potential disease-modifying approaches.
In clinical practice, valproate-based regimens remain central to seizure management, with adjunctive therapies tailored to seizure type, comorbidities, tolerability, and drug interactions. While stiripentol, clobazam, fenfluramine, and cannabidiol are supported by the strongest evidence, less frequently reported therapies, including perampanel, topiramate, levetiracetam, cenobamate, and ketogenic dietary therapies, may benefit selected patients but require cautious use due to heterogeneous efficacy and safety data. The complexity of available options highlights the need for individualized, dynamic treatment strategies. Although emerging targeted and genetic therapies may represent a future paradigm shift beyond symptomatic seizure control, their clinical impact remains to be established, warranting careful implementation and long-term safety evaluation.
Well-established treatments, together with recently approved agents, are discussed alongside under-reported therapies. Safety profiles, clinically relevant pharmacokinetic interactions, and practical aspects of dose titration and monitoring are reviewed. Emerging targeted pharmacological and genetic strategies are also briefly considered as potential disease-modifying approaches.
In clinical practice, valproate-based regimens remain central to seizure management, with adjunctive therapies tailored to seizure type, comorbidities, tolerability, and drug interactions. While stiripentol, clobazam, fenfluramine, and cannabidiol are supported by the strongest evidence, less frequently reported therapies, including perampanel, topiramate, levetiracetam, cenobamate, and ketogenic dietary therapies, may benefit selected patients but require cautious use due to heterogeneous efficacy and safety data. The complexity of available options highlights the need for individualized, dynamic treatment strategies. Although emerging targeted and genetic therapies may represent a future paradigm shift beyond symptomatic seizure control, their clinical impact remains to be established, warranting careful implementation and long-term safety evaluation.
Authors
Ferretti Ferretti, Riva Riva, Perilli Perilli, Parisi Parisi, Striano Striano
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