大规模的血清蛋白质组学识别了与杜申尼肌肉衰竭的性能下降和临床里程碑相关的蛋白质
N A Ikelaar1,2, A M Barnard3, S W M Eng4
1Department of Neurology, Leiden University Medical Center, Leiden, Zuid-Holland, the Netherlands.
medRxiv : the preprint server for health sciences
|August 16, 2024
概括
新的血清生物标志物显示出追踪杜申氏肌肉发育不良 (DMD) 进展的前景. 这些经过验证的生物标志物与临床里程碑和皮质类固醇使用相关,有助于患者管理.
科学领域:
- 生物标志物和诊断仪器
- 神经肌肉疾病 神经肌肉疾病
- 临床试验结果衡量临床试验的结果.
背景情况:
- 杜恩肌肉发育不良 (DMD) 缺乏可靠的生物标志物来确定临床进展.
- 对于DMD临床研究来说,最少的侵入性结果措施至关重要.
研究的目的:
- 确定与DMD临床表现相关的血清生物标志物.
- 确定生物标志物是否可以预测DMD患者的临床里程碑.
主要方法:
- 追溯多中心队列研究 (佛罗里达大学和莱登大学医学中心).
- 使用7K SomaScan测定方法分析了716个血清样本.
- 蛋白质水平与临床数据的相关性,包括NSAA,10mrv,6MWT和PUL2.0.0.
主要成果:
- 血清生物标志物预测了失去行走,头部伸展和手口功能时的年龄.
- 244 血清蛋白与皮质类固醇 (CS) 使用相关.
- 36种蛋白质与下肢和上肢的临床里程碑有关.
结论:
- 多个验证的血清生物标志物与DMD中CS使用和运动功能相关.
- 这些生物标志物显示出预测疾病进展和临床里程碑的潜力.
- 这些发现支持将这些生物标志物转化为更广泛的DMD患者群体.
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