Skuteczność i bezpieczeństwo clobazamu jako leczenia uzupełniającego u dzieci z opornymi na leczenie napadami padaczki w Xinjiang, Chiny: wieloośrodkowe badanie obserwacyjne

OBJECTIVE: This real-world retrospective observational study evaluated the efficacy and safety of clobazam as adjunctive therapy in pediatric patients with refractory epilepsy (RE) in Xinjiang to provide evidence for its rational clinical application. METHODS: Treatment efficacy was assessed according to the four-level efficacy criteria established by the First National Epilepsy Academic Conference of the Chinese Medical Association using seizure frequency recorded at the final follow-up. Changes in seizure frequency from baseline were evaluated at 3, 6, and 12 months after treatment initiation. All adverse drug reactions occurring during follow-up were recorded. RESULTS: The response rates to clobazam therapy were 50.7%, 50.7%, 53.8%, and 56.7% at 1, 3, 6, and 12 months, respectively. The effective group received a significantly higher daily clobazam dose than the ineffective group ( < 0.05). The proportion of pediatric patients with no reduction in seizure frequency decreased significantly from 14.7% at 1 month to 1.7% at ≥12 months ( < 0.05), while the proportion of pediatric patients with a <50% reduction in seizure frequency gradually increased over time. The highest efficacy was observed in patients with a disease duration of 3-6 years (62.5%, 10/16), while those with a duration of <1 year showed relatively lower efficacy (30.8%, 4/13). Sex and comorbidities were significant predictors of clobazam controlling seizures ( = 0.039 and = 0.030, respectively). During the clobazam treatment period, 31 pediatric patients (41.33%) experienced at least one adverse drug reaction. CONCLUSION: Clobazam demonstrated favorable efficacy, safety, and tolerability as adjunctive therapy in pediatric patients with RE. These real-world findings provide clinical evidence supporting the rational use of clobazam in pediatric RE.