Long-term Outcomes of Patients on Stiripentol, Fenfluramine, and Cannabidiol – Real-World Evidence
Long-term real-world data on the efficacy and safety of stiripentol, fenfluramine, and cannabidiol provide valuable insights beyond controlled clinical trials for Dravet Syndrome.
Observational studies indicate sustained seizure reductions with these agents, often with improved cognitive and behavioral outcomes over multiple years. Stiripentol, used in combination with clobazam and valproate, has demonstrated durable seizure control and an acceptable safety profile.
Fenfluramine’s long-term use is associated with persistent seizure reduction and manageable side effects, though cardiac monitoring remains important given historical concerns about valvulopathy. Cannabidiol continues to show efficacy with good tolerability, though liver enzyme monitoring is recommended especially when combined with clobazam.
Patient registries and post-marketing surveillance further confirm these findings, highlighting the importance of individualized therapy and ongoing monitoring to optimize outcomes.
