Department of Medicine, CORe, University of Melbourne, Melbourne, Victoria, Australia.
Department of Neurology, Neuroimmunology Centre, Royal Melbourne Hospital, Melbourne, Victoria, Australia.
Eur J Neurol. 2023 Apr;30(4):1014-1024. doi: 10.1111/ene.15706. Epub 2023 Feb 16.
This study assessed the effect of patient characteristics on the response to disease-modifying therapy (DMT) in multiple sclerosis (MS).
We extracted data from 61,810 patients from 135 centers across 35 countries from the MSBase registry. The selection criteria were: clinically isolated syndrome or definite MS, follow-up ≥ 1 year, and Expanded Disability Status Scale (EDSS) score ≥ 3, with ≥1 score recorded per year. Marginal structural models with interaction terms were used to compare the hazards of 12-month confirmed worsening and improvement of disability, and the incidence of relapses between treated and untreated patients stratified by their characteristics.
Among 24,344 patients with relapsing MS, those on DMTs experienced 48% reduction in relapse incidence (hazard ratio [HR] = 0.52, 95% confidence interval [CI] = 0.45-0.60), 46% lower risk of disability worsening (HR = 0.54, 95% CI = 0.41-0.71), and 32% greater chance of disability improvement (HR = 1.32, 95% CI = 1.09-1.59). The effect of DMTs on EDSS worsening and improvement and the risk of relapses was attenuated with more severe disability. The magnitude of the effect of DMT on suppressing relapses declined with higher prior relapse rate and prior cerebral magnetic resonance imaging activity. We did not find any evidence for the effect of age on the effectiveness of DMT. After inclusion of 1985 participants with progressive MS, the effect of DMT on disability mostly depended on MS phenotype, whereas its effect on relapses was driven mainly by prior relapse activity.
DMT is generally most effective among patients with lower disability and in relapsing MS phenotypes. There is no evidence of attenuation of the effect of DMT with age.
本研究评估了患者特征对多发性硬化症（MS）患者接受疾病修正治疗（DMT）反应的影响。
我们从 35 个国家的 135 个中心的 MSBase 注册中心提取了 61810 名患者的数据。入选标准为：临床孤立综合征或明确的 MS，随访时间≥1 年，扩展残疾状况量表（EDSS）评分≥3，每年至少记录 1 分。采用具有交互项的边缘结构模型比较了治疗和未治疗患者的残疾 12 个月确认恶化和改善的风险比（HR）和复发发生率，根据患者特征对其进行分层。
在 24344 例复发型 MS 患者中，DMT 治疗组的复发发生率降低了 48%（HR=0.52，95%可信区间[CI]为 0.45-0.60），残疾恶化风险降低了 46%（HR=0.54，95%CI 为 0.41-0.71），残疾改善的几率增加了 32%（HR=1.32，95%CI 为 1.09-1.59）。DMT 对 EDSS 恶化和改善以及复发风险的影响随着残疾程度的加重而减弱。DMT 抑制复发的效果与较高的既往复发率和既往脑磁共振成像活动有关。我们没有发现年龄对 DMT 有效性有影响的证据。纳入 1985 例进展型 MS 患者后，DMT 对残疾的影响主要取决于 MS 表型，而对复发的影响主要取决于既往复发活动。
DMT 在残疾程度较低和复发型 MS 表型的患者中通常最有效。没有证据表明年龄会减弱 DMT 的效果。
