介绍
疾病行为可以指导间质性肺病 (ILD) 患者的诊断和治疗决策。STARLINER 旨在使用实时家庭评估来表征疑似 ILD 患者在诊断期间的疾病行为。
方法
STARLINER (NCT03261037) 是一项国际多中心研究。≥ 50 岁疑似 ILD 患者在整个诊断期间进行随访，包括诊断前阶段（从入组到诊断）和诊断后阶段（从诊断到治疗开始）。研究时间是可变的（≤ 18 个月）。主要终点是特发性肺纤维化 (IPF) 患者在诊断期间使用每日家庭肺活量测定法测量的经时间调整的半年用力肺活量 (FVC) 变化。次要结果包括非 IPF ILD 患者 FVC（家庭肺活量测定）的变化、FVC（部位肺活量测定）的变化、每日家庭加速度测量法和现场 6 分钟步行距离（6MWD）测量的身体机能变化，
结果
在参与研究的 178 名患者中，68 名患者被诊断为 IPF，62 名患者被诊断为非 IPF ILD，9 名患者被诊断为非 ILD，39 名患者未得到诊断。技术和分析问题导致在应用预先指定的线性回归模型来分析家庭 FVC 数据时出现问题。使用家庭和现场肺活量测定法测量的围诊断期内的时间调整中位数（四分位数 [Q]1、Q3）半年 FVC 变化分别为 – 147.7 (– 723.8, 376.2) ml 和 – 149.0 (– 314.6, IPF 为 163.9) ml，非 IPF ILD 为 19.1 (– 194.9, 519.0) ml 和 – 23.4 (– 117.9, 133.5) ml。观察到 IPF 与非 IPF ILD 相比，每天步数下降更大，而观察到 IPF 患者的 6MWD 增加与非 IPF ILD 患者的 6MWD 下降。
结论
尽管家庭肺活量测定法对大多数患者和中心都是可行的，但家庭评估中的技术和分析挑战阻止了关于疾病行为的确切结论。这凸显了在广泛实施之前需要进一步优化技术和分析方法。
试用注册
NCT03261037。
"点击查看英文标题和摘要"
Disease Behaviour During the Peri-Diagnostic Period in Patients with Suspected Interstitial Lung Disease: The STARLINER Study
Introduction
Disease behaviour may guide diagnosis and treatment decisions in patients with interstitial lung disease (ILD). STARLINER aimed to characterise disease behaviour in patients with suspected ILD during the peri-diagnostic period using real-time home-based assessments.
Methods
STARLINER (NCT03261037) was an international, multicentre study. Patients ≥ 50 years old with suspected ILD were followed throughout the peri-diagnostic period, consisting of a pre-diagnostic period (from enrolment to diagnosis) and a post-diagnostic period (from diagnosis to treatment initiation). Study length was variable (≤ 18 months). The primary endpoint was time-adjusted semi-annual forced vital capacity (FVC) change measured during the peri-diagnostic period using daily home spirometry in patients with idiopathic pulmonary fibrosis (IPF). Secondary outcomes included changes in FVC (home spirometry) in patients with non-IPF ILD, changes in FVC (site spirometry), changes in physical functional capacity measured by daily home accelerometry and site 6-min walk distance (6MWD), and changes in patient-reported outcomes (PROs) in IPF or non-IPF ILD.
Results
Of the 178 patients enrolled in the study, 68 patients were diagnosed with IPF, 62 patients were diagnosed with non-IPF ILD, 9 patients received a non-ILD diagnosis and 39 patients did not receive a diagnosis. Technical and analytical issues led to problems in applying the prespecified linear regression model to analyse the home FVC data. Time-adjusted median (quartile [Q]1, Q3) semi-annual FVC change during the peri-diagnostic period measured using home and site spirometry, respectively, was – 147.7 (– 723.8, 376.2) ml and – 149.0 (– 314.6, 163.9) ml for IPF and 19.1 (– 194.9, 519.0) ml and – 23.4 (– 117.9, 133.5) ml in non-IPF ILD. A greater decline in steps per day was observed for IPF versus non-IPF ILD, whereas an increase in 6MWD was observed for patients with IPF versus a decline in 6MWD for patients with non-IPF ILD. No clear patterns of disease behaviour were observed for IPF versus non-IPF ILD for PROs.
Conclusions
Despite home spirometry being feasible for most patients and centres, technical and analytical challenges in the home-based assessments prevented firm conclusions regarding disease behaviour. This highlights that further optimisation of the technology and analysis methods is required before widespread implementation.
Trial Registration
NCT03261037.
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