Francis Guillemin, Romain Casey, Jonathan Epstein, Yohann Foucher, David Laplaud, Hamza Achit, Fabien Rollot, Emmanuelle Leray, Sandra Vukusic
CHRU, INSERM, Université de Lorraine, CIC Clinical Epidemiology, Nancy, Grand Est, France
Université de Lorraine, INSERM, INSPIIRE, Paris, Île-de-France, France.
BMJ Open. 2025 Apr 7;15(4):e094688. doi: 10.1136/bmjopen-2024-094688.
To determine prognostic factors of disability in multiple sclerosis (MS), that is, (1) identify determinants of the dynamics of disability progression; (2) study the effectiveness of disease-modifying treatments (DMTs); (3) merge determinants and DMTs for creating patient-centred prognostic tools and (4) conduct an economic analysis.
Individuals registered in the French Observatoire Français de la Sclérose en Plaques (OFSEP) database were included in this OFSEP-high definition cohort if they had a diagnosis of MS, were ≥15 years old and had an Expanded Disability Status Scale (EDSS) score <7. The outcomes will be assessed annually: (1) time to reach irreversible EDSS scores of 4, 6 and 7; (2) relapses and disease progression; (3) MRI-based progression, patient-reported outcomes, social consequences; and (4) combined outcomes on activity and progression. Clinical and quality-of-life data, MRI results and biological (blood, serum) samples will be collected at each follow-up.
A cohort of 2842 individuals, 73.4% women, mean (SD) age of 42.7 (11.6) years, median disease duration of 8.8 years, has been recruited from July 2018 to September 2020. The course of MS was relapsing remitting in 67.7%, secondary progressive in 11.9%. The mean annual relapse rate was 0.98. The disease-modifying treatment received was highly effective therapy in 50.3% and moderately effective therapy in 30.7%.
The participants will be followed until December 2026. Disease course up to four landmarks will be examined as predictors of disease progression: (1) diagnosis of MS; (2) relapse activity worsening and independent progression; (3) any recent disease activity and (4) any visit with absence of disease activity in the past 5 years. The marginal effectiveness and tolerability of treatments will be assessed. Stratified algorithms will be proposed for medical decision-making. Economic evaluation of disease cost and cost-effectiveness of new DMTs will be conducted from a public payer perspective.
NCT03603457.
确定多发性硬化症（MS）患者残疾的预后因素，即：（1）确定残疾进展动态的决定因素；（2）研究疾病修正治疗（DMT）的有效性；（3）整合决定因素和DMT以创建以患者为中心的预后工具；（4）进行经济分析。
如果个体被诊断为MS，年龄≥15岁且扩展残疾状态量表（EDSS）评分<7，则将其纳入法国多发性硬化症观察站（OFSEP）数据库中登记的个体，组成该OFSEP高清队列。每年将评估以下结果：（1）达到不可逆EDSS评分4、6和7的时间；（2）复发和疾病进展；（3）基于MRI的进展、患者报告的结果、社会后果；（4）活动和进展的综合结果。每次随访时将收集临床和生活质量数据、MRI结果以及生物（血液、血清）样本。
2018年7月至2020年9月招募了2842名个体组成队列，其中73.4%为女性，平均（标准差）年龄为42.7（11.6）岁，疾病中位持续时间为8.8年。MS病程为复发缓解型的占67.7%，继发进展型的占11.9%。年平均复发率为0.98。接受的疾病修正治疗中，高效治疗占50.3%，中度有效治疗占30.7%。
对参与者进行随访直至2026年12月。将检查直至四个时间节点的疾病病程作为疾病进展的预测因素：（1）MS诊断；（2）复发活动恶化和独立进展；（3）任何近期疾病活动；（4）过去5年中任何无疾病活动的就诊情况。将评估治疗的边际有效性和耐受性。将提出分层算法用于医疗决策。将从公共支付者的角度对疾病成本和新DMT的成本效益进行经济评估。
NCT03603457。
