University Hospitals Leuven, Leuven, Belgium.
Clinical Data Science, Basel, Switzerland.
J Neuromuscul Dis. 2018;5(4):419-430. doi: 10.3233/JND-180338.
Loss of pulmonary function is a main cause of early morbidity and mortality in patients with Duchenne muscular dystrophy (DMD). Standard of care guidelines recommend regular assessment of pulmonary function by hospital-based spirometry to detect onset and monitor progression of pulmonary function decline.
To assess the feasibility of home-based monitoring of pulmonary function by a hand-held device (HHD) in adolescent and adult patients with DMD over a period of 12 months.
In the phase III randomized placebo-controlled DELOS trial in 10-18 year old DMD patients, peak expiratory flow (PEF) measurements were collected weekly at home by the patient (assisted by parent/caregiver) using a peak flow meter HHD. Adherence to the use of the HHD was assessed and 12-month changes in PEF as percent of predicted (PEF% p) for the idebenone (N = 31) and the placebo treatment groups (N = 33) from HHD-derived data were compared to results from hospital-based spirometry.
A total of 2689 individual HHD assessments were analysed. Overall adherence to the use of the HHD over the course of the 12-month study duration was good (75.9%, SD 21.5%) and PEF% p data obtained at the same day by HHD and standard spirometry correlated well (Spearman's rho 0.80; p < 0.001). Several analysis methods of HHD-derived data for PEF% p consistently demonstrate that idebenone treatment slowed the decline in PEF% p compared to placebo, which supports the statistically significant difference in favour of idebenone for PEF% p measured by standard spirometry.
This study demonstrates that home-based monitoring of pulmonary function in adolescent patients with DMD using a HHD is feasible, provides reliable data compared to hospital-based spirometry and is therefore suitable for use in clinical practice and for clinical trials.
肺功能丧失是杜氏肌营养不良症（DMD）患者早期发病率和死亡率的主要原因。基于标准护理的指南建议通过基于医院的肺活量测定法定期评估肺功能，以检测肺功能下降的开始和监测其进展。
评估手持式设备（HHD）在 12 个月期间对青少年和成年 DMD 患者进行家庭肺功能监测的可行性。
在 10-18 岁 DMD 患者的 III 期随机安慰剂对照 DELOS 试验中，患者（在父母/照顾者的协助下）每周在家中使用峰值流量计 HHD 进行峰值呼气流量（PEF）测量。评估对 HHD 使用的依从性，并比较从 HHD 衍生数据得出的依地苯醌（N = 31）和安慰剂治疗组（N = 33）的 12 个月 PEF 与预测值的百分比（PEF%p）的变化，以及基于医院的肺活量测定法的结果。
分析了总共 2689 项 HHD 评估。在 12 个月的研究过程中，总体上对 HHD 使用的依从性良好（75.9%，SD 21.5%），HHD 和标准肺活量计在同一天获得的 PEF%p 数据相关性良好（Spearman's rho 0.80；p < 0.001）。HHD 衍生数据的几种分析方法均表明，与安慰剂相比，依地苯醌治疗减缓了 PEF%p 的下降，这支持了通过标准肺活量计测量的 PEF%p 对依地苯醌有利的统计学差异。
这项研究表明，使用 HHD 对青少年 DMD 患者进行家庭肺功能监测是可行的，与基于医院的肺活量测定法相比提供了可靠的数据，因此适合在临床实践和临床试验中使用。
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