Background

Poor monitoring of respiratory function may lead to late initiation of non-invasive ventilation (NIV) in patients with motor neuron diseases (MND). Monitoring could be improved by remotely assessing hypoventilation symptoms between clinic visits. We aimed to determine which patient-reported hypoventilation symptoms are best for screening reduced respiratory function in patients with MND, and compared them to the respiratory domain of the amyotrophic lateral sclerosis functional rating scale (ALSFRS-R).

Methods

This prospective multi-center study included 100 patients with MND, who were able to perform a supine vital capacity test. Reduced respiratory function was defined as a predicted supine vital capacity ≤ 80%. We developed a 14-item hypoventilation symptom questionnaire (HYSQ) based on guidelines, expert opinion and think-aloud interviews with patients. Symptoms of the HYSQ were related to dyspnea, sleep quality, sleepiness/fatigue and pneumonia. The diagnostic performances of these symptoms and the ALSFRS-R respiratory domain were determined from the receiver operating characteristic (ROC) curves, area under the curve (AUC), sensitivity, specificity, predictive values and accuracy.

Results

Dyspnea-related symptoms (dyspnea while eating/talking, while lying flat and during light activity) were combined into the MND Dyspnea Scale (MND-DS). ROC curves showed that the MND-DS had the best diagnostic performance, with the highest AUC = 0.72, sensitivity = 75% and accuracy = 71%. Sleep-quality symptoms, sleepiness/fatigue-related symptoms and the ALSFRS-R respiratory domain showed weak diagnostic performance.

Conclusion

The diagnostic performance of the MND-DS was better than the respiratory domain of the ALSFRS-R for screening reduced respiratory function in patients with MND, and is, therefore, the preferred method for (remotely) monitoring respiratory function.

中文翻译：

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使用患者报告的呼吸困难症状筛查运动神经元疾病患者呼吸功能下降。

背景

呼吸功能监测不良可能导致运动神经元疾病（MND）患者的无创通气（NIV）延迟启动。可以通过远程评估两次就诊之间的换气不足症状来改善监测。我们旨在确定哪种患者报告的通气不足症状最适合筛查MND患者呼吸功能降低，并将其与肌萎缩性侧索硬化功能等级量表（ALSFRS-R）的呼吸域进行比较。

方法

这项前瞻性多中心研究纳入了100名能够进行仰卧位肺活量测试的MND患者。呼吸功能下降被定义为预计仰卧肺活量≤80％。我们根据指南，专家意见和对患者的智商访谈，开发了一项14项通气不足症状问卷（HYSQ）。HYSQ的症状与呼吸困难，睡眠质量，嗜睡/疲劳和肺炎有关。这些症状和ALSFRS-R呼吸域的诊断性能由接收器工作特征（ROC）曲线，曲线下面积（AUC），敏感性，特异性，预测值和准确性确定。

结果

与呼吸困难相关的症状（进食/说话，平躺和轻度活动时呼吸困难）被合并到MND呼吸困难量表（MND-DS）中。ROC曲线显示MND-DS具有最佳的诊断性能，最高AUC = 0.72，灵敏度= 75％，准确度= 71％。睡眠质量症状，与困倦/疲劳相关的症状以及ALSFRS-R呼吸域显示出较弱的诊断性能。

结论

MND-DS的诊断性能优于ALSFRS-R的呼吸域，用于筛查MND患者呼吸功能降低，因此是（远程）监测呼吸功能的首选方法。