Department of Pediatrics, Children's Hospital of Eastern Ontario, Ottawa, Ontario, Canada
CHEO Research Institute, Ottawa, Ontario, Canada.
Thorax. 2022 Aug;77(8):805-811. doi: 10.1136/thoraxjnl-2021-218196. Epub 2022 Mar 2.
Impaired cough results in airway secretion retention, atelectasis and pneumonia in individuals with Duchenne muscular dystrophy (DMD). Lung volume recruitment (LVR) stacks breaths to inflate the lungs to greater volumes than spontaneous effort. LVR is recommended in DMD clinical care guidelines but is not well studied. We aimed to determine whether twice-daily LVR, compared with standard of care alone, attenuates the decline in FVC at 2 years in boys with DMD.
In this multicentre, assessor-blinded, randomised controlled trial, boys with DMD, aged 6-16 years with FVC >30% predicted, were randomised to receive conventional treatment or conventional treatment plus manual LVR twice daily for 2 years. The primary outcome was FVC % predicted at 2 years, adjusted for baseline FVC % predicted, age and ambulatory status. Secondary outcomes included change in chest wall distensibility (maximal insufflation capacity minus FVC) and peak cough flow.
Sixty-six boys (36 in LVR group, 30 in control) were evaluated (median age (IQR): 11.5 years (9.5-13.5), median baseline FVC (IQR): 85% predicted (73-96)). Adjusted mean difference in FVC between groups at 2 years was 1.9% predicted (95% CI -6.9% to 10.7%; p=0.68) in the direction of treatment benefit. We found no differences in secondary outcomes.
There was no difference in decline in FVC % predicted with use of twice-daily LVR for boys with DMD and relatively normal lung function. The burden associated with routine LVR may outweigh the benefit. Benefits of LVR to maintain lung health in boys with worse baseline lung function still need to be clarified.
NCT01999075.
杜兴氏肌肉营养不良症（DMD）患者由于咳嗽能力受损，导致气道分泌物潴留、肺不张和肺炎。肺容积复张（LVR）通过叠加呼吸将肺部充气至比自主努力更大的容积。LVR 已被推荐用于 DMD 临床护理指南，但研究并不充分。我们旨在确定与单独标准治疗相比，每日两次 LVR 是否可以减缓 DMD 男孩在 2 年内 FVC 的下降。
在这项多中心、评估者盲法、随机对照试验中，年龄在 6-16 岁、FVC>30%预测值的 DMD 男孩被随机分为接受常规治疗或常规治疗加每日两次手动 LVR，为期 2 年。主要结局是 2 年后 FVC%预测值，根据基线 FVC%预测值、年龄和步行状态进行调整。次要结局包括胸壁顺应性（最大充气量减去 FVC）和最大咳嗽流量的变化。
共有 66 名男孩（LVR 组 36 名，对照组 30 名）接受了评估（中位年龄（IQR）：11.5 岁（9.5-13.5），中位基线 FVC（IQR）：85%预测值（73-96））。2 年后两组间 FVC 的调整平均差异为 1.9%预测值（95%CI：-6.9%至 10.7%；p=0.68），治疗方向有利。我们没有发现次要结局的差异。
对于具有相对正常肺功能的 DMD 男孩，每日两次 LVR 并没有减少 FVC%预测值的下降。常规 LVR 相关的负担可能超过了益处。对于基线肺功能更差的男孩，LVR 维持肺健康的益处仍需进一步阐明。
NCT01999075。
