远程呼吸阻力运动训练改善了VCP多系统蛋白质病变患者的呼吸功能
Madeline Halseth1, Ryan Mahoney1, Joyce Hsiou2
1Division of Genetics and Genomic Medicine, Department of Pediatrics, UC Irvine School of Medicine, Irvine, CA, United States.
这项研究表明,远程启发性肌肉训练计划在含瓦洛辛蛋白 (VCP) 疾病患者中改善了肺功能. 该计划对于患有这种罕见遗传疾病的个体来说是安全和可行的.
科学领域:
- 神经学 神经学
- 肺部病理学 肺部病理学
- 遗传学 遗传学 是一个
背景情况:
- 含瓦洛蛋白 (VCP) 疾病是一种遗传性疾病,导致多系统蛋白质病变.
- 呼吸道肌肉衰弱是VCP疾病的常见和危及生命的并发症.
- 在VCP疾病中治疗呼吸系统功能障碍的有效干预措施有限.
研究的目的:
- 评估在患有VCP疾病的个体中远程管理的启发性肌肉训练计划的可行性,安全性和有效性.
- 评估灵感肌肉训练对呼吸肌肉强度和功能结果的影响.
主要方法:
- 一项为期40周的远程研究,涉及9名患有VCP突变阳性家族肌病的成年人.
- 一个为期8周的基线期,随后是32周的启发性肌肉训练.
- 主要结局:最大吸气压 (MIP);次要结局:螺旋计,握力,功能评分尺度 (IBMFRS,ALSFRS) 和移动性测试 (TUG,6MWT).
主要成果:
- 在训练阶段,最大吸入压力 (MIP) 每周显著增加0.392厘米H2O (p=0.023).
- 预计握力和ALSFRS得分的显著下降,反映了疾病的进展.
- 培训计划是可行的,安全的,并被参与者容忍.
结论:
- 远程管理的启发性肌肉训练可以显著改善VCP疾病患者的启发性肌肉力量.
- 这种干预提供了一个有希望的,可访问的方法来管理VCP疾病的呼吸道并发症.
- 需要进一步的研究来探索长期影响和更广泛的临床益处.
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