Abstract
OBJECTIVE: This study aimed to compare the effectiveness and safety of oxcarbazepine (OXC) vs. levetiracetam (LEV) for treating infantile focal epilepsy in a longitudinal cohort study. METHODS: We enrolled 187 consecutive patients aged 2-24 months who received OXC or LEV as initial monotherapy; 161 patients completed the study. The longitudinal analysis involved anti-seizure medication (ASM) responsiveness, safety, the establishment of epilepsy syndrome, and etiology over a median follow-up of 2 years (interquartile range [IQR] 1.6-2.4). The relative efficacy and retention rates of OXC vs. LEV were evaluated using generalized linear regression models and the Cox proportional hazards model. RESULTS: The 161 patients who completed the study had comparable baseline demographics and clinical variables between the OXC group (n = 83) and LEV group (n = 78). Overall, the mean age at onset was 6 months (IQR 4.3-9). The most common epilepsy syndrome was self-limited familial/non-familial infantile epilepsy (54.7%). Epilepsy was related to genetic and unknown causes in 34.2 and 52.2% of the patients, respectively. OXC achieved significantly higher responses than LEV for seizure freedom (risk ratio [RR] = 1.71, 95% confidence interval [CI] = 1.28-2.73, P < 0.001) and 12-month retention rate after onset (hazard ratio [HR] = 1.84, 95% CI = 1.15-2.95, P = 0.007). Moreover, OXC showed more obvious effects for patients aged < 1 year diagnosed with self-limited familial/non-familial infantile epilepsy and non-syndromic epilepsy with genetic or unknown causes. The adverse events related to both OXC and LEV were well-tolerated. SIGNIFICANCE: OXC could be an alternative to LEV for treating infantile focal epilepsy. OXC monotherapy can be considered first-line treatment for patients aged <12 months and those with epilepsy without developmental and epileptic encephalopathy.