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美国正在接受治疗的重症肌无力患者的疾病负担和成本。

Burden of illness and costs in patients with myasthenia gravis currently receiving treatment in the United States.

机构信息

Alexion, AstraZeneca Rare Disease, Boston, Massachusetts, USA.

Genesis Research Group, Hoboken, New Jersey, USA.

出版信息

Muscle Nerve. 2024 Feb;69(2):157-165. doi: 10.1002/mus.27992. Epub 2023 Nov 29.

Abstract

INTRODUCTION/AIMS: If myasthenia gravis (MG) symptoms are inadequately controlled, patients may experience exacerbations or life-threatening myasthenic crises. Patients with inadequately controlled MG symptoms tend to be treated with chronic intravenous immunoglobulin (IVIg) therapy and/or multiple immunosuppressant therapies (ISTs). This study aimed to examine disease burden, healthcare resource utilization, and associated costs in these patients.

METHODS

This was a retrospective observational study using a claims database. Patients with MG were classified into three cohorts based on treatment over a 1-y follow-up period: (a) treated with four or more IVIg episodes (chronic IVIg cohort); (b) received two or more non-steroidal ISTs (NSISTs) sequentially (multiple NSIST cohort); (c) received neither chronic IVIg nor multiple NSISTs (reference cohort). Incidences of crises and exacerbations and annual healthcare costs in each cohort were estimated.

RESULTS

In total, 3516 patients with MG were included in the analysis. Compared with the reference cohort (n = 2992), the MG crisis rate was approximately twice as high in both the chronic IVIg (n = 324) and multiple NSIST (n = 291) cohorts (p < 0.001); and the MG exacerbation rate was approximately four-fold higher in the chronic IVIg cohort (p < 0.001) and three-fold higher in the multiple NSIST cohort (p < 0.001). Median annual MG-related inflation-adjusted total healthcare costs were higher in the chronic IVIg ($81,900) and multiple NSIST ($30,300) cohorts than in the reference cohort ($2540).

DISCUSSION

The burden of crises/exacerbations was substantially higher and healthcare costs were considerably greater in patients with MG treated with chronic IVIg or multiple NSISTs than in patients not receiving these treatments.

摘要

简介/目的:如果重症肌无力 (MG) 症状控制不佳,患者可能会出现恶化或危及生命的肌无力危象。症状控制不佳的 MG 患者往往需要接受慢性静脉注射免疫球蛋白 (IVIg) 治疗和/或多种免疫抑制治疗 (IST)。本研究旨在评估这些患者的疾病负担、医疗资源利用情况和相关成本。

方法

这是一项使用索赔数据库的回顾性观察性研究。根据 1 年随访期间的治疗情况,将 MG 患者分为三组:(a)接受 4 次或更多 IVIg 治疗的患者(慢性 IVIg 组);(b)接受两种或更多非甾体 IST 序贯治疗的患者(多种非甾体 IST 组);(c)既未接受慢性 IVIg 治疗也未接受多种非甾体 IST 治疗的患者(参照组)。估计每个队列的危象和恶化发生率以及年度医疗费用。

结果

共纳入 3516 例 MG 患者进行分析。与参照组(n=2992)相比,慢性 IVIg 组(n=324)和多种非甾体 IST 组(n=291)的 MG 危象发生率均约为两倍(p<0.001);慢性 IVIg 组的 MG 恶化发生率约为四倍(p<0.001),多种非甾体 IST 组的 MG 恶化发生率约为三倍(p<0.001)。慢性 IVIg 组(81900 美元)和多种非甾体 IST 组(30300 美元)的年化 MG 相关通胀调整后总医疗费用中位数均显著高于参照组(2540 美元)。

讨论

接受慢性 IVIg 或多种非甾体 IST 治疗的 MG 患者的危象/恶化负担显著更高,医疗费用也显著更高。

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