The Effectiveness and Tolerability of Clobazam Therapy In The Pediatric Population: A Multicenter Large-Cohort Study

Objectives: To evaluate the effectiveness and tolerability of clobazam therapy in the pediatric population in terms of seizure semiology, epileptic syndromes, and etiological subgroups. Methods: A retrospective cohort study was conducted consisting of 1710 epileptic children from eight centers. The initial efficacy of clobazam therapy was evaluated after three months of treatment. The long-term effectiveness of the drug, overall seizure outcome, and tolerance were evaluated during 12 months of therapy. Results: Initial efficacy at the end of the first three months of clobazam therapy was as follows: 320 (18.7%) patients were seizure-free, 683 (39.9%) patients had >50% seizure reduction, and 297 (17.4%) patients had <50% seizure reduction. A positive response (seizure-free and >50% seizure reduction) was determined for focal-onset (62.3%) and generalized-onset (57.4%) seizures. Patients with infantile spasm syndrome (West syndrome) exhibited the highest positive response rate (64.6%) among the epileptic syndromes, followed by Lennox-Gastaut syndrome (49.6%). Magnetic resonance imaging (MRI) revealed a structural etiological diagnosis in 25.8% of the cohort. The seizure recurrence rate was higher in the patient group with genetic disorders, in individuals with more than one seizure type, in those using three or more antiseizure drugs, and in those with improvement rates lower than 50% on EEGs. Conclusion: This cohort study provides additional evidence that clobazam is an effective and well-tolerable drug with a high seizure-free rate (18.7%) and a significant seizure reduction rate (57.3%) in the pediatric population. Considerable reversible benefit (drug tolerance) was determined in 12.7% of the cohort.