Background:The double-blind TERIKIDS study demonstrated the efficacy and safety of teriflunomide.Objective:To evaluate the efficacy, safety, and tolerability of continuous teriflunomide treatment in the TERIKIDS open-label extension.Methods:In the double-blind period, children with relapsing MS were randomized to placebo or teriflunomide (14 mg adult-equivalent dose) for ⩽ 96 weeks. Participants received teriflunomide for ⩽ 192 weeks post-randomization in the open-label extension.Results:The mean age at screening was 14.6 years. For teriflunomide/teriflunomide versus placebo/teriflunomide, estimated clinical relapse risk was reduced by 38% (hazard ratio (HR) 0.62; 95% confidence interval (CI) 0.39–0.98; p = 0.11) and numbers of gadolinium-enhancing T1 and new/enlarging T2 lesions were reduced by 43% (relative risk (RR) 0.570; 95% CI 0.33–0.98; p = 0.043) and 49% (RR 0.511; 95% CI 0.34–0.76; p = 0.001), respectively, in the combined double-blind and open-label periods. There was a trend toward reduced risk of 24-week sustained disability progression for teriflunomide/teriflunomide versus placebo/teriflunomide (HR 0.47; 95% CI 0.23–0.96). During the open-label extension, incidences of safety-related discontinuations were 4.0% (teriflunomide/teriflunomide) and 13.5% (placebo/teriflunomide), including two children who developed pancreatitis in the teriflunomide/teriflunomide group.Conclusion:Teriflunomide reduced the long-term risk of focal inflammatory activity, with generally manageable tolerability and no new safety signals. Further evidence would strengthen clinical efficacy findings. ClinicalTrials.gov: NCT02201108.

中文翻译：

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特立氟胺治疗复发性多发性硬化症儿科患者：TERIKIDS 的开放标签扩展

背景：双盲 TERIKIDS 研究证明了特立氟胺的有效性和安全性。目的：评价 TERIKIDS 开放标签扩展中持续特立氟胺治疗的有效性、安全性和耐受性。方法：在双盲期，对患有特立氟胺的儿童进行研究。复发性多发性硬化症患者随机接受安慰剂或特立氟胺（14 mg 成人等效剂量）治疗，疗程⩽ 96 周。在开放标签扩展中，参与者在随机分组后接受特立氟胺治疗 ⩽ 192 周。结果：筛选时的平均年龄为 14.6 岁。与安慰剂/特立氟胺相比，特立氟胺/特立氟胺的估计临床复发风险降低了 38%（风险比 (HR) 0.62；95% 置信区间 (CI) 0.39–0.98；p = 0.11），钆增强 T1 和新治疗的数量降低了 38%。扩大的 T2 病变分别减少了 43%（相对风险 (RR) 0.570；95% CI 0.33–0.98；p = 0.043）和 49%（RR 0.511；95% CI 0.34–0.76；p = 0.001）。双盲期和开放标签期的结合。与安慰剂/特立氟胺相比，特立氟胺/特立氟胺有降低 24 周持续残疾进展风险的趋势（HR 0.47；95% CI 0.23-0.96）。在开放标签扩展期间，与安全相关的停药发生率分别为 4.0%（特立氟胺/特立氟胺）和 13.5%（安慰剂/特立氟胺），其中包括特立氟胺/特立氟胺组中两名出现胰腺炎的儿童。局灶性炎症活动的长期风险，耐受性总体可控，并且没有新的安全信号。进一步的证据将加强临床疗效发现。 ClinicalTrials.gov：NCT02201108。