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随时间追踪重症肌无力的严重程度:来自法国医疗保险理赔数据库的见解。

Tracking myasthenia gravis severity over time: Insights from the French health insurance claims database.

作者信息

Attarian Shahram, Camdessanché Jean-Philippe, Echaniz-Laguna Andoni, Ciumas Mariana, Blein Cécile, Grenier Benjamin, Solé Guilhem

机构信息

Reference Centre for Neuromuscular Disease and ALS, Timone University Hospital, Aix-Marseille University, CHU Timone, Filnemus, Euro-NMD, Marseille, France.

Department of Neurology, Neuromuscular Disease Reference Centre, Hôpital Nord, University Hospital of Saint-Étienne, Saint-Étienne, France.

出版信息

Eur J Neurol. 2025 Jan;32(1):e16518. doi: 10.1111/ene.16518. Epub 2024 Nov 4.

Abstract

BACKGROUND AND PURPOSE

Few data are available on the course of myasthenia gravis (MG) regarding disease severity and stability over time in real-world settings. This study used the French National Health Insurance Database (SNDS) to assess markers of disease severity in patients with MG longitudinally.

METHODS

All patients with MG-related claims in the SNDS between 2013 and 2020 were identified. Patients were followed for up to 8 years after the first claim. Intensive care unit (ICU) stays, treatment with intravenous immunoglobulin (IVIg) or plasma exchange (PE), and death were documented throughout the follow-up period. Standardized mortality rates were estimated, and mortality-related variables were identified using a Cox model.

RESULTS

In all, 14,459 individuals constituted the full study population, including 6354 incident patients. In the incident population, 2199 (34.6%) were admitted to ICUs at least once, principally during the first year after the index date (N = 1477; 23.3%). This proportion decreased progressively to reach 3.0% in the seventh year. A total of 2817 patients received IVIg and 432 PE, again principally in the first year. In the full study population, the standardized mortality rate was 1.08 (95% confidence interval [CI] 1.03-1.13), being lower in men (0.95, 95% CI 0.89-1.02) than in women (1.15, 95% CI 1.07-1.23) and in patients aged >65 years (1.06, 95% CI 1.01-1.11) than in younger patients (1.50, 95% CI 1.24-1.76). Male gender, older age and higher comorbidity were independently associated with mortality.

CONCLUSIONS

A subgroup of patients with MG require ICU admission and rescue therapy with IVIg or PE, indicative of poor disease control. New therapies are needed to improve disease control and reduce disease burden.

摘要

背景与目的

在现实环境中,关于重症肌无力(MG)病程中疾病严重程度和随时间稳定性的数据较少。本研究利用法国国家健康保险数据库(SNDS)纵向评估MG患者的疾病严重程度指标。

方法

确定2013年至2020年间SNDS中所有与MG相关索赔的患者。在首次索赔后对患者进行长达8年的随访。记录整个随访期间的重症监护病房(ICU)住院情况、静脉注射免疫球蛋白(IVIg)或血浆置换(PE)治疗情况以及死亡情况。估计标准化死亡率,并使用Cox模型确定与死亡率相关的变量。

结果

共有14459人构成完整研究人群,其中包括6354例新发病例。在新发病例人群中,2199例(34.6%)至少有一次入住ICU,主要是在索引日期后的第一年(n = 1477;23.3%)。这一比例在第七年逐渐降至3.0%。共有2817例患者接受了IVIg治疗,432例接受了PE治疗,同样主要在第一年。在整个研究人群中,标准化死亡率为1.08(95%置信区间[CI] 1.03 - 1.13),男性(0.95,95% CI 0.89 - 1.02)低于女性(1.15,95% CI 1.07 - 1.23),年龄>65岁的患者(1.06,95% CI 1.01 - 1.11)低于年轻患者(1.50,95% CI 1.24 - 1.76)。男性、高龄和较高的合并症与死亡率独立相关。

结论

一部分MG患者需要入住ICU并接受IVIg或PE的抢救治疗,这表明疾病控制不佳。需要新的疗法来改善疾病控制并减轻疾病负担。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/583d/11622507/f149624dca0c/ENE-32-e16518-g006.jpg

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