国家改善疾病治疗补贴政策对多发性硬化症患者长期残疾结局的影响

Suzi B Claflin, Julie A Campbell, Deborah F Mason, Tomas Kalincik, Steve Simpson-Yap, Richard Norman, Helmut Butzkueven, William M Carroll, Andrew J Palmer, C Leigh Blizzard, Ingrid van der Mei, Bruce V Taylor
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引用次数: 6

摘要

背景:疾病修饰疗法(dmt)用于治疗复发性多发性硬化症(ROMS)患者,但我们的知识在很大程度上仅限于其短期效果。目的:确定(1)国家级DMT补贴政策对MS (PwMS)患者DMT使用和健康结局的影响;(2)DMT对残疾和生活质量的长期影响;5级EQ-5D版本(EQ-5D- 5l)实用价值)。方法:这项观察性队列研究比较了澳大利亚和新西兰的人群,他们在roms诊断后10-20年有不同水平的DMT可用性。使用标准化差异评估国与国之间的差异。用多变量线性回归模型评估相关性。结果:我们招募了328名澳大利亚人和256名新西兰人。澳大利亚队列的DMT治疗时间较长,治疗病程的比例较大,诊断和开始DMT之间的时间较短。澳大利亚队列的扩展残疾状态量表(EDSS)中位数较低(3.5 vs 4.0),多发性硬化严重程度评分(MSSS)中位数较低(3.05 vs 3.71),生活质量较高(0.71 vs 0.65)。在多变量模型中,残疾和生活质量的国家间差异主要归因于DMT使用的差异。结论:本研究为国家级DMT政策对PwMS残障结局的影响提供了证据。在更容易获得dmt的地方,PwMS的残疾进展较少,诊断后10-20年的生活质量得到改善。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
The effect of national disease-modifying therapy subsidy policy on long-term disability outcomes in people with multiple sclerosis.

Background: Disease-modifying therapies (DMTs) are used to treat people with relapsing-onset multiple sclerosis (ROMS), but our knowledge is largely limited to their short-term effects.

Objective: To determine (1) the impact of national-level DMT subsidy policy on DMT use and health outcomes in people with MS (PwMS) and (2) the long-term effects of DMT on disability and quality of life (QoL; 5-level EQ-5D version (EQ-5D-5L) utility value).

Methods: This observational cohort study compared Australian and New Zealand populations with different levels of DMT availability 10-20 years post-ROMS diagnosis. Between-country differences were assessed using standardised differences. Associations were assessed with multivariable linear regression models.

Results: We recruited 328 Australians and 256 New Zealanders. The Australian cohort had longer DMT treatment duration, greater proportion of disease course treated and shorter duration between diagnosis and starting DMT. The Australian cohort had lower median Expanded Disability Status Scale (EDSS) (3.5 vs 4.0) and Multiple Sclerosis Severity Score (MSSS) (3.05 vs 3.71) and higher QoL (0.71 vs 0.65). In multivariable models, between-country differences in disability and QoL were largely attributed to differential use of DMT.

Conclusions: This study provides evidence for the impact of national-level DMT policy on disability outcomes in PwMS. Where DMTs are more accessible, PwMS experienced less disability progression and improved QoL 10-20 years post-diagnosis.

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