预测首次脱髓鞘事件后长期预后的巴塞罗那基线风险评分：一项前瞻性观察性研究。

The Barcelona baseline risk score to predict long-term prognosis after a first demyelinating event: a prospective observational study.

作者信息

Tur Carmen, Carbonell-Mirabent Pere, Otero-Romero Susana, Cobo-Calvo Álvaro, Arévalo María Jesús, Ariño Helena, Arrambide Georgina, Auger Cristina, Carvajal René, Castilló Joaquín, Comabella Manuel, Galán Ingrid, Midaglia Luciana, Nos Carlos, Pappolla Agustín, Pareto Deborah, Río Jordi, Rodríguez-Acevedo Breogán, Saez de Gordoa Estibaliz, Vidal-Jordana Ángela, Zabalza Ana, Sastre-Garriga Jaume, Rovira Àlex, Montalban Xavier, Tintoré Mar

机构信息

Multiple Sclerosis Centre of Catalonia (Cemcat), Department of Neurology, Hospital Universitari Vall d'Hebron, Universitat Autònoma de Barcelona, Barcelona, Spain.

Section of Neuroradiology, Department of Radiology, Vall d'Hebron University Hospital, Spain.

出版信息

Lancet Reg Health Eur. 2025 Apr 19;53:101302. doi: 10.1016/j.lanepe.2025.101302. eCollection 2025 Jun.

DOI:10.1016/j.lanepe.2025.101302

PMID:40291402

原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC12033939/

Abstract

BACKGROUND

In multiple sclerosis (MS), predicting at symptom onset who will develop early and severe disability is an unmet need with significant therapeutic implications. Here we propose the Barcelona-Baseline Risk Score (BRS) model to predict long-term disease outcomes in a flexible and generalisable manner.

METHODS

Using prospectively acquired data from the Barcelona first-attack cohort, we created the Barcelona-BRS model as a set of six Weibull survival models of time to an Expanded Disability Status Scale score of 3.0, built with flexible combinations of predictors, including sex, age at first attack, and number and topography of T2 lesions, among others, adaptable to data availability. Data-driven risk groups were identified and compared in terms of long-term clinical and MRI outcomes, including relapse-associated worsening (RAW), progression independent of relapse activity (PIRA), conversion to secondary progressive MS (SPMS), lesional and brain volumetric data, and patient-reported/administered clinical scores, through Kaplan-Meier and mixed-effects models. Finally, we externally validated our model in a completely unseen cohort.

FINDINGS

We included 1074 patients (737 [69%] female, mean age: 31.7 years) with a first demyelinating attack. Over a median follow-up of 11.9 years, 375 (35%), 298 (28%), and 94 (8.8%) developed RAW, PIRA, and SPMS, respectively. Weibull models included age at first attack, number of brain T2 lesions, and disability at first visit as main predictors. Four data-driven groups of increasing risk of unfavourable outcomes were created: Light-Green-BRS (N = 258), Dark-Green-BRS (N = 319), Orange-BRS (N = 321), and Red-BRS (N = 176), which, over time, behaved significantly differently across disability, quality of life, and MRI measures, being the Red-BRS the group with worst outcomes (p < 0.01). The results in the external validation cohort (N = 139, 100 female [72%], 34 years) mirrored those of the original one.

INTERPRETATION

The robustness, flexibility, and generalisability of the Barcelona-BRS model support its consideration as a ready-to-use tool for clinical practice.

FUNDING

None.

摘要

背景

在多发性硬化症（MS）中，在症状出现时预测谁会早期出现严重残疾且尚未得到满足，这具有重大的治疗意义。在此，我们提出巴塞罗那基线风险评分（BRS）模型，以灵活且可推广的方式预测长期疾病转归。

方法

利用从巴塞罗那首发队列前瞻性获取的数据，我们创建了巴塞罗那 - BRS模型，作为一组六个韦布尔生存模型，用于预测达到扩展残疾状态量表评分3.0的时间，该模型由预测因素的灵活组合构建而成，包括性别、首次发作时的年龄以及T2病变的数量和部位等，可根据数据可用性进行调整。通过Kaplan - Meier和混合效应模型，确定数据驱动的风险组，并比较其长期临床和MRI结果，包括复发相关恶化（RAW）、与复发活动无关的进展（PIRA）、转化为继发进展型MS（SPMS）、病变和脑容量数据以及患者报告/管理的临床评分。最后，我们在一个完全独立的队列中对我们的模型进行了外部验证。

研究结果

我们纳入了1074例首次脱髓鞘发作的患者（737例[69%]为女性，平均年龄：31.7岁）。在中位随访11.9年期间，分别有375例（35%）、298例（28%）和94例（8.8%）发生了RAW、PIRA和SPMS。韦布尔模型将首次发作时的年龄、脑T2病变数量和首次就诊时的残疾程度作为主要预测因素。创建了四个数据驱动的不良结局风险递增组：浅绿 - BRS组（N = 258）、深绿 - BRS组（N = 319）、橙色 - BRS组（N = 321）和红色 - BRS组（N = 176），随着时间推移，这些组在残疾、生活质量和MRI测量方面表现出显著差异，红色 - BRS组结局最差（p < 0.01）。外部验证队列（N = 139，100例女性[72%]，34岁）的结果与原始队列相似。