Neuropediatric Department, BHZ Vogtareuth, Vogtareuth, Germany.
Epilepsy Behav. 2010 Apr;17(4):546-8. doi: 10.1016/j.yebeh.2010.01.005. Epub 2010 Feb 24.
Recently, we published the first postmarketing European experience with rufinamide (RUF) in a retrospective 12-week observational study. This follow-up report summarizes the long-term effectiveness and tolerability of RUF after 18 months for the same patient sample.
In total, 52 of 60 initially included patients from eight centers in Germany and Austria (45 children and 15 adults aged 1-50 years) with various severe and inadequately controlled epilepsy syndromes continued treatment with RUF after the initial 3-month observation period (mean final dose: 38.2+/-17.3mg/kg/day). Efficacy was assessed by seizure frequency evaluated by comparison with baseline frequency. Tolerability was evaluated by analysis of parental report of adverse events and laboratory tests. Responders were defined as patients who achieved a 50% or greater decrease in countable seizures within 18 months of initiating RUF therapy.
Mean overall duration of RUF treatment was 14.5 months (range: 3-18 months). Retention rate, defined as the percentage of patients still taking RUF after 18 months, was 41.7% (n=25/60). The overall response rate after 18 months was 26.7% (16/60 patients). The highest response rates were found in the subgroup of patients with Lennox-Gastaut syndrome (LGS, 35.5%) and in patients with other generalized epilepsy syndromes. Complete seizure control was maintained in one patient (1.6%). A total of 73 adverse events were reported in 37 of 60 patients. The most frequently occurring adverse events were fatigue (18.3%), vomiting (15.0%), and loss of appetite (10.0%). Only 4 new adverse events were reported after week 12. No serious adverse events were observed.
The present data suggest that RUF is efficacious and well tolerated in the long-term treatment of children and adults with various epilepsy syndromes and difficult-to-control seizures.
最近，我们发表了一项回顾性的 12 周观察研究，报告了首项在欧洲开展的鲁非酰胺（RUF）上市后经验。本随访报告总结了相同患者样本在 18 个月后 RUF 的长期疗效和耐受性。
德国和奥地利的 8 个中心共纳入 60 例患者（45 例儿童，15 例成人，年龄 1-50 岁），他们患有各种严重且控制不佳的癫痫综合征。这些患者在最初的 3 个月观察期后继续接受 RUF 治疗（平均最终剂量：38.2+/-17.3mg/kg/天）。通过与基线频率比较评估发作频率来评估疗效。通过分析父母报告的不良事件和实验室检查评估耐受性。将在开始 RUF 治疗的 18 个月内发作次数减少 50%或更多的患者定义为应答者。
RUF 治疗的平均总持续时间为 14.5 个月（范围：3-18 个月）。18 个月后仍服用 RUF 的患者比例（定义为保留率）为 41.7%（n=25/60）。18 个月后总体应答率为 26.7%（60 例患者中的 16 例）。Lennox-Gastaut 综合征（LGS）患者亚组和其他全身性癫痫综合征患者的应答率最高（分别为 35.5%和 35.5%）。一名患者（1.6%）保持完全无发作。60 例患者中有 37 例报告了 73 种不良事件。最常发生的不良事件是疲劳（18.3%）、呕吐（15.0%）和食欲不振（10.0%）。仅在第 12 周后报告了 4 例新的不良事件。未观察到严重不良事件。
目前的数据表明，在治疗各种癫痫综合征和难以控制的发作的儿童和成人患者中，RUF 长期治疗是有效且耐受良好的。
