Krupp Lauren, Banwell Brenda, Chitnis Tanuja, Deiva Kumaran, Gaertner Jutta, Ghezzi Angelo, Huppke Peter, Waubant Emmanuelle, DeLasHeras Virginia, Azmon Amin, Karan Rajesh
Pediatric MS Center, NYU Langone Health, New York, New York, USA.
The Children's Hospital of Philadelphia, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
BMJ Neurol Open. 2022 Feb 24;4(1):e000215. doi: 10.1136/bmjno-2021-000215. eCollection 2022.
In the PARADIG Study, fingolimod demonstrated superior efficacy versus interferon (IFN) β-1a and comparable overall incidence of adverse events but slightly higher rate of serious adverse events in patients with paediatric-onset multiple sclerosis (PoMS). Here, we report the health-related quality of life (HRQoL) outcomes from PARADIG.
Patients with PoMS (N=215; aged 10-<18 years) were randomised to once-daily oral fingolimod (N=107) or once-weekly intramuscular IFN β-1a (N=108). HRQoL outcomes were assessed using the 23-item Pediatric Quality of Life (PedsQL) scale that comprises Physical and Psychosocial Health Summary Scores (including Emotional, Social and School Functioning). A post hoc inferential analysis evaluated changes in self-reported or parent-reported PedsQL scores from baseline up to 2 years between treatment groups using an analysis of covariance model.
Treatment with fingolimod showed improvements versus IFN β-1a on the PedsQL scale in both the self-reported and parent-reported Total Scale Scores (4.66 vs -1.16, p≤0.001 and 2.71 vs -1.02, p≤0.05, respectively). The proportion of patients achieving a clinically meaningful improvement in the PedsQL Total Scale Score was two times higher with fingolimod versus IFN β-1a per the self-reported scores (47.5% vs 24.2%, p=0.001), and fingolimod was favoured versus IFN β-1a per the parent-reported scores (37.8% vs 24.7%, p=non-significant). Group differences in self-reported Total Scale Scores in favour of fingolimod were most pronounced among patients who had ≥2 relapses in the year prior to study entry or who showed improving or stable Expanded Disability Status Scale scores during the study.
Fingolimod improved HRQoL compared with IFN β-1a in patients with PoMS as evidenced by the self-reported and parent-reported PedsQL scores.
在PARADIG研究中，对于儿童多发性硬化症（PoMS）患者，芬戈莫德显示出优于干扰素（IFN）β-1a的疗效，且不良事件的总体发生率相当，但严重不良事件的发生率略高。在此，我们报告PARADIG研究中与健康相关的生活质量（HRQoL）结果。
PoMS患者（N = 215；年龄10 - <18岁）被随机分为每日一次口服芬戈莫德组（N = 107）或每周一次肌肉注射IFNβ-1a组（N = 108）。使用包含身体和心理社会健康总结评分（包括情绪、社交和学校功能）的23项儿童生活质量（PedsQL）量表评估HRQoL结果。事后推断分析使用协方差分析模型评估治疗组之间从基线到2年自我报告或家长报告的PedsQL评分的变化。
在自我报告和家长报告的总评分量表上，芬戈莫德治疗组在PedsQL量表上的改善均优于IFNβ-1a组（自我报告的总分：4.66对 -1.16，p≤0.001；家长报告的总分：2.71对 -1.02，p≤0.05）。根据自我报告的评分，在PedsQL总评分量表上实现临床意义上改善的患者比例，芬戈莫德组是IFNβ-1a组的两倍（47.5%对24.2%，p = 0.001），根据家长报告的评分，芬戈莫德组优于IFNβ-1a组（37.8%对24.7%，p无统计学意义）。在研究入组前一年有≥2次复发或在研究期间扩展残疾状态量表评分改善或稳定的患者中，自我报告的总评分量表上支持芬戈莫德的组间差异最为明显。
自我报告和家长报告的PedsQL评分表明，与IFNβ-1a相比，芬戈莫德改善了PoMS患者的HRQoL。
