Department of Pediatrics, University of Chieti, Chieti, Italy.
Child Neurology and Psychiatry Unit, Children's Hospital "G. Salesi", Ospedali Riuniti Ancona, Ancona, Italy.
Epilepsia. 2023 Jun;64(6):e98-e104. doi: 10.1111/epi.17601. Epub 2023 Apr 10.
This retrospective study assessed long-term effectiveness of add-on perampanel (PER) in patients with Lennox-Gastaut syndrome (LGS). Outcomes included time to PER failure and time to seizure relapse in responders. PER failure was defined as either discontinuation of PER or initiation of another treatment. Seizure relapse in responders was defined as occurrence of a seizure in seizure-free patients and increase of at least 50% in average monthly seizure frequency for those who were responders. Eighty-seven patients were included. Treatment failure occurred in 52 (59.8%) subjects at a median time of 12 months. Treatment failure was due to lack of efficacy in 27 (52.0%) patients, lack of tolerability in 14 (27.0%), and both reasons in 11 (21.0%). A slower titration was associated with a lower risk of PER failure compared to faster titration schedules, and the occurrence of adverse events increased the risk of treatment failure. Thirty-six patients (41.4%) were responders during a median follow-up of 11 months. Seizure relapse occurred in 13 of 36 (36.1%) patients after a median time of 21 months. The overall rate of seizure responders was 23 of 87 (26.4%) at the end of follow-up. This study provides real-world evidence on the effectiveness of PER as adjunctive treatment in LGS patients.
这项回顾性研究评估了添加普瑞巴林(PER)治疗 Lennox-Gastaut 综合征(LGS)患者的长期疗效。结果包括 PER 治疗失败的时间和应答者癫痫发作复发的时间。PER 治疗失败定义为 PER 停药或开始其他治疗。应答者癫痫发作复发定义为无癫痫发作患者出现癫痫发作,以及平均每月癫痫发作频率增加至少 50%的患者。共纳入 87 例患者。中位时间为 12 个月时,52 例(59.8%)患者发生治疗失败。治疗失败的原因包括疗效不佳(27 例,52.0%)、不耐受(14 例,27.0%)和两者兼有的(11 例,21.0%)。与快速滴定相比,较慢的滴定与 PER 治疗失败的风险较低相关,且不良事件的发生增加了治疗失败的风险。36 例患者(41.4%)在中位随访 11 个月时为应答者。中位时间为 21 个月后,13 例(36.1%)应答者出现癫痫发作复发。随访结束时,87 例患者中总体癫痫发作应答率为 23 例(26.4%)。这项研究提供了 PER 作为 LGS 患者辅助治疗的有效性的真实世界证据。