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多发性硬化症进展病程:一项临床队列长期残疾进展研究。

Multiple Sclerosis Progressive Courses: A Clinical Cohort Long-Term Disability Progression Study.

作者信息

Cortesi Paolo A, Fornari Carla, Capra Ruggero, Cozzolino Paolo, Patti Francesco, Mantovani Lorenzo G

机构信息

Research Centre on Public Health (CESP), University of Milano-Bicocca, Monza, Italy.

Research Centre on Public Health (CESP), University of Milano-Bicocca, Monza, Italy.

出版信息

Value Health. 2022 Sep;25(9):1489-1498. doi: 10.1016/j.jval.2022.03.010. Epub 2022 Apr 26.

DOI:10.1016/j.jval.2022.03.010
PMID:35484029
Abstract

OBJECTIVES

Improving the understanding of multiple sclerosis (MS) mechanism and disability progression over time is essential to assess the value of healthcare interventions. Poor or no data on disability progression are available for progressive courses. This study aims to fill this gap.

METHODS

An observational cohort study of patients with primary MS (PPMS) and secondary progressive MS (SPMS) was conducted on 2 Italian MS centers disease registries over an observational time of 34 years. Annual transition probabilities among Expanded Disability Status Scale (EDSS) states were estimated using continuous Markov models. A sensitivity analysis was performed in relation to clinical characteristic associated to disability progression.

RESULTS

The study cohort included 758 patients (274 PPMS and 434 SPMS) with a median follow-up of 8.2 years. Annual transition probability matrices of SPMS and PPMS reported different annual probabilities to move within EDSS levels. Excluding EDSS associated to relapse events or patient with relapses, the annual probability of staying stable in an EDSS level increased in both disease courses even not significantly.

CONCLUSIONS

This study provides estimates of annual disability progression as EDSS changes for PPMS and SPMS. These estimates could be a useful tool for healthcare decision makers and clinicians to properly assess impact of clinical interventions.

摘要

目的

增进对多发性硬化症(MS)机制以及残疾随时间进展情况的了解,对于评估医疗干预措施的价值至关重要。对于进展型病程,关于残疾进展的数据匮乏或根本没有。本研究旨在填补这一空白。

方法

在意大利的2个MS中心疾病登记处,对原发性MS(PPMS)和继发性进展型MS(SPMS)患者进行了一项观察性队列研究,观察时间为34年。使用连续马尔可夫模型估计扩展残疾状态量表(EDSS)各状态之间的年度转换概率。针对与残疾进展相关的临床特征进行了敏感性分析。

结果

研究队列包括758例患者(274例PPMS和434例SPMS),中位随访时间为8.2年。SPMS和PPMS的年度转换概率矩阵显示,在EDSS各水平之间移动的年度概率有所不同。排除与复发事件相关的EDSS或有复发的患者后,在两种疾病病程中,在EDSS水平保持稳定的年度概率均有所增加,尽管增加幅度不显著。

结论

本研究提供了PPMS和SPMS患者EDSS变化时的年度残疾进展估计值。这些估计值可能是医疗保健决策者和临床医生正确评估临床干预措施影响的有用工具。

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