论文部分内容阅读
背景特立氟胺为复发型多发性硬化症新口服病情改善疗法。方法本研究为随机对照试验,参试者入选条件:18～55岁、进展性残疾评估量表分数0～5.5分、至少前1年有1次复发或者前2年有2次复发,最终1 088例多发性硬化症患者纳入本研究。将患者随机分组(按1∶1∶1比例):安慰剂组;特立氟胺7 mg或14 mg 1次/d,共108周。主要终点为年复发率;次要终点至少12周做残疾进展评估。结果特立氟胺使多发性硬化症年复发率下降(安慰剂组0.54,7或14 mg特立氟胺组0.37),相关风险分别减少31.2%和31.5%(两组与安慰剂相比,P<0.001)。确定残疾进展患者比例,安慰机组27.3%;7 mg特立氟胺组21.7%(P=0.08);14 mg特立氟胺组20.2%(P=0.03)。MRI终点测量结果表明,2种剂量特立氟胺组均优于安慰剂组。与安慰剂相比,腹泻、恶心、头发稀疏特立氟胺组较常见。丙氨酸氨基转移酶水平升高(是正常值上限的≥1倍)发生率,7 mg和14 mg特立氟胺组(分别为54.0%和57.3%)比安慰剂组(35.9%)要高;水平是正常值上限3倍的发生率:7 mg和14 mg特立氟胺组与安慰机组相似(分别为6.3%、6.7%和6.7%)。3组所报告的严重感染分别占患者的1.6%、2.5%和2.2%。无死亡病例。结论与安慰剂相比,特立氟胺使复发率下降、残疾进展(高剂量)减慢,获得了疾病活动度减低的MRI证据。
Background Teriflunomide is a new oral condition-modifying therapy for recurrent multiple sclerosis. Methods This study was a randomized controlled trial. The participants were enrolled in the following age range from 18 to 55 years. The Disability Disability Rating Scale score ranged from 0 to 5.5. There were 1 recurrence in at least 1 year or 2 recurrences in the first 2 years. 088 cases of multiple sclerosis patients included in this study. Patients were randomized (1: 1: 1 ratio) to placebo; teriflunomide 7 mg or 14 mg once daily for 108 weeks. The primary endpoint was the annual recurrence rate; the secondary endpoint was assessed for disability progression for at least 12 weeks. Results Teriflunomine reduced the annualized relapse rate in MS (0.54, 7, or 14 mg in the placebo group, 0.37) with a corresponding reduction of 31.2% and 31.5%, respectively, in the risk of developing multiple sclerosis (compared with placebo, P <0.001). To determine the proportion of patients with disability progression, comfort units 27.3%; 7 mg teriflunil group 21.7% (P = 0.08); 14 mg teriflunil group 20.2% (P = 0.03). MRI end point measurements showed that both doses of teriflunomide were superior to placebo. Compared with placebo, diarrhea, nausea, hair thinning teriflunil group more common. The incidence of alanine aminotransferase levels (≥1 times the upper limit of normal) was significantly higher in the 7 and 14 mg teriflunomide groups (54.0% and 57.3%, respectively) than in the placebo group (35.9%) High; the level was three times the upper limit of normal incidence: 7 and 14 mg of teriflunomide were similar to placebo (6.3%, 6.7% and 6.7%, respectively). Serious infections reported in group 3 accounted for 1.6%, 2.5% and 2.2% of patients, respectively. No deaths. Conclusions Teriflunomide reduced relapse rate and disability progression (high dose) compared with placebo, with MRI evidence of reduced disease activity.