Kessler Sudha Kilaru, McCarthy Ann, Cnaan Avital, Dlugos Dennis J
Departments of Neurology and Pediatrics, Perelman School of Medicine at the University of Pennsylvania and the Children's Hospital of Philadelphia, United States.
Temple University School of Medicine, United States.
Epilepsy Res. 2015 May;112:18-26. doi: 10.1016/j.eplepsyres.2015.02.003. Epub 2015 Feb 14.
To evaluate the effectiveness of rufinamide (RFM) in patients with Lennox-Gastaut Syndrome (LGS) compared to those with other epilepsy syndromes using time to treatment failure (retention rate) as the outcome measure.
In this retrospective cohort study, characteristics and outcomes of all patients receiving RFM in 2009 and 2010 were recorded. The primary outcome measure was RFM failure, defined as discontinuation of RFM or initiation of an additional antiepileptic therapy. The secondary outcome measure was discontinuation of RFM. Kaplan-Meier method survival curves were generated for time to RFM failure, for all patients and by the presence or absence of Lennox Gastaut Syndrome (LGS). The impact of age, seizure type, fast or slow drug titration, and concomitant therapy with valproate on retention rate were evaluated using Cox regression models.
One hundred thirty-three patients were included, 39 (30%) of whom had LGS. For all patients, the probability of remaining on RFM without additional therapy was 45% at 12 months and 30% at 24 months. LGS diagnosis was an independent predictor of time to RFM failure (HR 0.51, 95% CI 0.31-0.83), with a median time to failure of 18 months in LGS compared to 6 months in all others (p=0.006).
In a broad population of children with refractory epilepsy, around half will continue taking the medication for at least a year without additional therapy. Patients with LGS are two times more likely to continue RFM without additional therapy compared to those without LGS.
以治疗失败时间（保留率）作为结局指标，评估鲁非酰胺（RFM）在Lennox-Gastaut综合征（LGS）患者中与其他癫痫综合征患者相比的有效性。
在这项回顾性队列研究中，记录了2009年和2010年所有接受RFM治疗的患者的特征和结局。主要结局指标为RFM治疗失败，定义为停用RFM或开始加用另一种抗癫痫治疗。次要结局指标为停用RFM。采用Kaplan-Meier法生成所有患者以及根据是否存在Lennox Gastaut综合征（LGS）分组的RFM治疗失败时间的生存曲线。使用Cox回归模型评估年龄、癫痫发作类型、药物滴定快或慢以及丙戊酸盐联合治疗对保留率的影响。
共纳入133例患者，其中39例（30%）患有LGS。对于所有患者，在无额外治疗的情况下继续使用RFM的概率在12个月时为45%，在24个月时为30%。LGS诊断是RFM治疗失败时间的独立预测因素（风险比0.51，95%置信区间0.31 - 0.83），LGS患者的中位失败时间为18个月，而其他所有患者为6个月（p = 0.006）。
在一大群难治性癫痫儿童中，约一半患者在无额外治疗的情况下将继续服用该药物至少一年。与无LGS的患者相比，LGS患者在无额外治疗的情况下继续使用RFM的可能性高出两倍。
