BACKGROUND In PARADIGMS, a double-blind phase III trial in 215 paediatric patients with multiple sclerosis (MS) (10 to <18 years), fingolimod administered for up to 2 years significantly reduced the annualised relapse rate (ARR) and rate of new/newly enlarged T2 (n/neT2) lesions compared with interferon (IFN) β-1a. OBJECTIVES To investigate (1) differences between treatment groups across subpopulations (treatment-naïve, younger/prepubertal patients); (2) disability progression. METHODS ARRs at 10, 11 and 12 years were estimated based on predefined modelling extrapolations. Changes in Expanded Disability Status Scale (EDSS), and in 3 month (3M) and 6 month (6M) confirmed disability progression (CDP) were evaluated post hoc. RESULTS In the treatment-naïve subpopulation, fingolimod reduced ARR and n/neT2 lesions by 85.8% and 53.4%, respectively versus INF β-1a (both p<0.001), compared with 81.9% and 52.6% in the overall population. Model-based ARR reductions in younger patients (≤12 years) were 91.9%-94.6%. Twice as many IFN β-1a-treated than fingolimod-treated patients had worse EDSS scores at study end (20.6% vs 10.5%, p=0.043). Risk reductions in 3M-CDP and 6M-CDP were 77.2% (p=0.007) and 80.2% (p=0.040), respectively. CONCLUSIONS Fingolimod in paediatric MS was associated with consistent control of disease activity versus IFN β-1a (including treatment-naïve and younger patients) and resulted in less disability progression for up to 2 years. TRIAL REGISTRATION NUMBER NCT01892722.

中文翻译：

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芬戈莫德和IFNβ-1a在儿童发作性多发性硬化症中对疾病活动的一致控制：来自PARADIGMS的进一步见解。

背景在PARADIGMS中，一项针对215名多发性硬化症（MS）（10至<18岁）的儿科患者的双盲III期临床试验，使用芬戈莫德长达2年，显着降低了年复发率（ARR）和新药/新药的发生率与干扰素（IFN）β-1a相比，新近扩大的T2（n / neT2）病变。目的研究（1）不同亚人群（未接受过治疗，年轻/青春期前患者）的治疗组之间的差异；（2）残疾进展。方法根据预定义的模型推断估算10年，11年和12年的ARR。事后评估扩大的残疾状态量表（EDSS）以及3个月（3M）和6个月（6M）确认的残疾进展（CDP）的变化。结果在未经治疗的亚人群中，芬戈莫德可将ARR和n / neT2病变减少85.8％和53.4％，分别与INFβ-1a相比（均为p <0.001），而总人口中分别为81.9％和52.6％。在年轻患者（≤12岁）中，基于模型的ARR降低为91.9％-94.6％。在研究结束时，接受干扰素β-1a治疗的患者比接受芬戈莫德治疗的患者多两倍，EDSS评分更差（20.6％比10.5％，p = 0.043）。3M-CDP和6M-CDP的风险降低分别为77.2％（p = 0.007）和80.2％（p = 0.040）。结论芬戈莫德在小儿MS中与疾病控制相对于IFNβ-1a（包括初治和较年轻的患者）的疾病控制一致，并导致长达2年的残疾进展减少。试用注册号NCT01892722。在研究结束时，接受干扰素β-1a治疗的患者比接受芬戈莫德治疗的患者多两倍，EDSS评分更差（20.6％比10.5％，p = 0.043）。3M-CDP和6M-CDP的风险降低分别为77.2％（p = 0.007）和80.2％（p = 0.040）。结论芬戈莫德在小儿MS中与疾病控制相对于IFNβ-1a（包括初治和较年轻的患者）的疾病控制一致，并导致长达2年的残疾进展减少。试用注册号NCT01892722。在研究结束时，接受干扰素β-1a治疗的患者比接受芬戈莫德治疗的患者多两倍，EDSS评分更差（20.6％比10.5％，p = 0.043）。3M-CDP和6M-CDP的风险降低分别为77.2％（p = 0.007）和80.2％（p = 0.040）。结论芬戈莫德在小儿MS中与疾病控制相对于IFNβ-1a（包括初治和较年轻的患者）的疾病控制一致，并导致长达2年的残疾进展减少。试用注册号NCT01892722。结论芬戈莫德在小儿MS中与疾病控制相对于IFNβ-1a（包括初治和较年轻的患者）的疾病控制一致，并导致长达2年的残疾进展减少。试用注册号NCT01892722。结论芬戈莫德在小儿MS中与疾病控制相对于IFNβ-1a（包括初治和较年轻的患者）的疾病控制一致，并导致长达2年的残疾进展减少。试用注册号NCT01892722。