PT - JOURNAL ARTICLE AU - Jamil, Alisha AU - Levinson, Noah AU - Gelfand, Michael AU - Hill, Chloe E. AU - Khankhanian, Pouya AU - Davis, Kathryn A. TI - Efficacy and tolerability of clobazam in adults with drug-refractory epilepsy AID - 10.1212/CPJ.0000000000000992 DP - 2020 Oct 19 TA - Neurology: Clinical Practice PG - 10.1212/CPJ.0000000000000992 4099 - http://cp.neurology.org/content/early/2020/10/19/CPJ.0000000000000992.short 4100 - http://cp.neurology.org/content/early/2020/10/19/CPJ.0000000000000992.full AB - Objectives To evaluate the effectiveness and tolerability of clobazam as an adjunctive treatment for adults with drug-resistant epilepsy.Methods We performed a single-center, retrospective chart review of patients ≥18 years of age with drug-resistant epilepsy who started clobazam between 2010 and 2018. Included patients had outpatient visits both before and ≥1 month after clobazam initiation. Epilepsy classification, seizure frequency before and after clobazam, duration of clobazam treatment, and adverse effects were analyzed.Results A total of 417 patients met inclusion criteria. Mean age was 37.5 years, and 54% of patients were female. Patients were on a mean of 2.4 antiepileptic drugs at time of initiation of clobazam. Epilepsy types were focal (56.8%), Lennox-Gastaut syndrome (LGS) (21.1%), generalized (15.1%), and unclassified (7.0%). At the first follow-up visit ≥1 month after clobazam initiation, 50.3% of patients had >50% reduction in seizure frequency, and 20.5% were seizure-free. Of the initial cohort, 17.1% were followed >1 year and were seizure-free at last follow-up. Response rates did not differ between different epilepsy classifications. Fifty-one percent of patients experienced ≥1 side effect, most commonly lethargy/fatigue (30.7%) or mood changes (10.8%). A total of 178 (42.6%) patients discontinued clobazam, most commonly due to adverse effects (55%).Conclusions Clobazam is effective and safe as a long-term adjunctive therapy for adults with drug-resistant epilepsy; efficacy in off-label use is similar to that in LGS.Classification of evidence This study provides Class IV evidence that clobazam is an effective treatment for adults with drug-resistant epilepsy, independent of epilepsy classification.