Effectiveness and safety of Lacosamide in pediatric patients with epilepsy under four years: Results from a prospective cohort study in China

Background Lacosamide (LCM) has shown promising efficacy and safety outcomes in clinical trials. However, the evidence is limited among pediatric patients especially under four years in real-world. The study investigated the treatment outcomes and safety of LCM in patients under four years based on the data of the epilepsy registry of Children in China. Methods A prospective cohort study was conducted among patients under 4 years who newly received LCM as monotherapy or adjunctive therapy. The treatment outcomes were measured by retention rate of LCM, 50% response rates and seizure-free rates during follow-up. The retention rate of LCM was assessed using the Kaplan-Meier survival model. Adverse events were reported as a percentage of all participants. Results Of 109 participants (mean follow-up: 18.6 months), 59 received LCM as monotherapy and 50 as adjunctive therapy. Sixty patients had focal epilepsy, 44 had generalized epilepsy and 5 had combined generalized and focal epilepsy. 70% of patients in the monotherapy group and 41% in the adjunctive therapy group remained on LCM treatment without additional treatments for at least one year. In patients with monotherapy, 50% response rate and seizure-free rate were 75% and 56% at 12 months, respectively. In adjunctive therapy group, these rates were 51% and 36%, respectively. Lower baseline seizure frequency in both treatment groups (monotherapy: p<0.001; adjunctive therapy: p=0.02) and younger age groups within the monotherapy group (P=0.04) correlated with a higher LCM retention rate. Adverse events were reported by 15 patients (13.8%), with somnolence being the most common (7 of 15 patients). Conclusion With a comprehensive information and high-quality of data, the study demonstrates the effective treatment outcome and safety of LCM. The study adds reliable evidence to exiting real-word evidence of LCM in the specific age group of patients with epilepsy to fill the evidence gap.