脊柱肌肉缩的全球经济负担:一个系统的文献综述
Rawda Elshahawy1, Baher Elezbawy2,3, Rasha Ashmawy4,5
1Health Economics, Syreon Middle East, Alexandria, EGY.
Cureus
|April 23, 2025
概括
脊椎肌肉缩 (SMA) 给人带来了巨大的经济负担,平均每年每名患者的费用为109,906美元. 1型SMA患者面临的费用最高,突出了对经济支持策略的需要.
科学领域:
- 神经学 神经学
- 卫生经济学 卫生经济学
- 罕见疾病 罕见疾病
背景情况:
- 脊椎肌肉缩 (SMA) 是一种罕见的,遗传的神经肌肉疾病.
- 肌肉衰弱和运动功能丧失的特征,SMA呈现四种主要亚型.
- 高死亡率,治疗费用和护理需求给患者,护理人员和医疗保健系统带来了巨大的负担.
研究的目的:
- 研究不同脊髓肌肉缩 (SMA) 亚型的经济影响.
- 分析直接的医疗费用,非医疗费用,医疗资源利用率和生产力损失.
- 为制定政策提供见解,以减轻SMA的财务压力.
主要方法:
- 进行了一次系统的文献审查,搜索诸如Medline,Embase和Google Scholar等数据库,查找自2010年以来发表的研究.
- 在SMA亚型中提取了有关成本,医疗资源和生产力损失的数据.
- 通过纽卡斯尔-太华量表和健康经济研究的质量工具来评估研究质量.
主要成果:
- 包括来自32个国家的55项研究,揭示了经济数据的显著差异.
- 每名SMA患者的加权平均年成本为109,906美元.
- 1型SMA患者的直接医疗费用最高 (187,88美元,不包括疾病修饰治疗),非医疗费用达到每名患者109,379美元,每年的护理需求为2,947小时.
结论:
- 与脊椎肌肉缩 (SMA) 相关的直接和间接成本在所有亚型中都是相当大的.
- 数据的异质性强调了需要标准化方法来评估SMA的经济影响.
- 调查结果可以为改善金融可持续性和患者在管理SMA方面的支持政策提供信息.
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