Home monitoring improves endpoint efficiency in idiopathic pulmonary fibrosis.
Home monitoring improves endpoint efficiency in idiopathic pulmonary fibrosis.
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DOI：
10.1183/13993003.02406-2016
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发表时间：
2017-07
期刊：
影响因子：
--
通讯作者：
Collard HR
中科院分区：
文献类型：
--
作者：
Johannson KA;Vittinghoff E;Morisset J;Lee JS;Balmes JR;Collard HR
本研究的目的是探讨在家中测量用力肺活量(FVC)和呼吸困难作为特发性肺纤维化(IPF)临床终点的可靠性、可行性和分析影响。IPF患者每周在家中使用移动手持肺活量计和自我管理的呼吸困难问卷对FVC和呼吸困难进行评估。评估FVC和呼吸困难的周变异性，并使用传统的基于办公室的间隔测量或移动每周评估模拟假想的24周临床试验的样本量。总共有25名患者入选。每周评估的平均坚持时间超过24周，超过90%。与仅使用基线和24周测量的变化评估相比，每周评估FVC导致了更高的精确度和能力。例如，一项假设的以FVC为主要终点的24周临床试验将需要951名患者每周在家中进行肺活量测定，而只在第1周和24周使用办公室肺活量测定的患者为3840人。重复测量减少临床试验样本量的能力受数据的相关性结构的影响。家庭监测可以提高终点评估的精确度，从而提高IPF治疗的临床试验效率。
The objective of this study was to investigate the reliability, feasibility and analytical impact of home-based measurement of forced vital capacity (FVC) and dyspnoea as clinical endpoints in idiopathic pulmonary fibrosis (IPF). Patients with IPF performed weekly home-based assessment of FVC and dyspnoea using a mobile hand-held spirometer and self-administered dyspnoea questionnaires. Weekly variability in FVC and dyspnoea was estimated, and sample sizes were simulated for a hypothetical 24-week clinical trial using either traditional office-based interval measurement or mobile weekly assessment. In total, 25 patients were enrolled. Mean adherence to weekly assessments over 24 weeks was greater than 90%. Compared with change assessment using baseline and 24-week measurements only, weekly assessment of FVC resulted in enhanced precision and power. For example, a hypothetical 24-week clinical trial with FVC as the primary endpoint would require 951 patients using weekly home spirometry compared with 3840 patients using office spirometry measures at weeks 1 and 24 only. The ability of repeated measures to reduce clinical trial sample size was influenced by the correlation structure of the data. Home monitoring can improve the precision of endpoint assessments, allowing for greater efficiency in clinical trials of therapeutics for IPF.
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DOI：
10.7326/0003-4819-142-12_part_1-200506210-00005
发表时间：
2005-06-21
影响因子：
39.2
作者：
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通讯作者：
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影响因子：
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作者：
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通讯作者：
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DOI：
10.1164/rccm.2009-040gl
发表时间：
2011-03-15
影响因子：
24.7
作者：
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通讯作者：
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