Associations of SLCO gene polymorphisms with perampanel response and plasma concentrations in patients: a longitudinal retrospective real-world study in refractory epilepsy.

OBJECTIVE: This study analyzed the correlation between SLCO gene polymorphisms and the efficacy and plasma concentrations of perampanel (PER) in pediatric patients with refractory epilepsy (RE). METHODS: SLCO genotypes were detected using first-generation Sanger sequencing, and the plasma concentration of PER was measured using ultra-performance liquid chromatography-tandem mass spectrometry. RESULTS: The overall efficacy of PER in treating patients with RE was observed in 64.3% of patients (74 of 115). SLCO1A2 rs4149000, SLCO1B1 rs2306283, and rs12422149 gene polymorphisms were significantly associated with the efficacy of PER in treating RE. Age, epilepsy duration, concomitant medications, and SLCO1A2 rs4149000 polymorphisms were important predictors of effective seizure control related to PER treatment (p < 0.05). PER plasma concentrations were significantly higher in the effective group of pediatric patients receiving the once-daily regimen than in the ineffective group (598.5 ± 390.2 vs 452.9 ± 296.5 ng/mL; p = 0.027). CONCLUSION: SLCO gene polymorphisms were shown to be important predictors of effective seizure control related to PER treatment and were identified as factors affecting plasma concentrations of PER. The findings on SLCO genotypes and PER plasma concentration monitoring provide a promising treatment strategy for Chinese patients with RE.