Home monitoring improves endpoint efficiency in idiopathic pulmonary fibrosis

被引:70
作者
Johannson, Kerri A. [1 ,2 ]
Vittinghoff, Eric [3 ]
Morisset, Julie [4 ]
Lee, Joyce S. [5 ]
Balmes, John R. [6 ,7 ]
Collard, Harold R. [7 ]
机构
[1] Univ Calgary, Dept Med, Calgary, AB, Canada
[2] Univ Calgary, Dept Community Hlth Sci, Calgary, AB, Canada
[3] Univ Calif San Francisco, Dept Biostat & Epidemiol, San Francisco, CA 94143 USA
[4] CHU Montreal, Dept Med, Montreal, PQ, Canada
[5] Univ Colorado Denver, Dept Med, Aurora, CO USA
[6] Univ Calif Berkeley, Sch Publ Hlth, Div Environm Hlth Sci, Berkeley, CA 94720 USA
[7] Univ Calif San Francisco, Dept Med, San Francisco, CA USA
关键词
CLINICAL-COURSE; PIRFENIDONE; NINTEDANIB; SPIROMETRY; CAPACITY; DYSPNEA; TRIALS;
D O I
10.1183/13993003.02406-2016
中图分类号
R56 [呼吸系及胸部疾病];
学科分类号
摘要
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.
引用
收藏
页数:8
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