Abstract：
OBJECTIVE: Patient-reported outcome measures (PROMs) are used to collect data on disease symptoms in support of clinical trial endpoints. Clinical studies can last a year or more, and the patients\' adherence and response time to daily at-home questionnaires may vary significantly over time. The aim of this study was to understand patterns and changes in patients\' completion of daily PROMs during longitudinal clinical studies.
METHODS: Data were collected from 1342 patients randomized into three respiratory clinical trials (NCT03401229, NCT03347279, and NCT03406078). PROMs were completed by patients using electronic handheld devices that collected the starting and completion times. A Bayesian generalized linear mixed-effects model was used to identify unbiased coefficients associated with PROM adherence and response time using patient, site, and calendar features as covariates.
RESULTS: Adherence decreased over time after randomization, and the rate of decrease was higher in younger patients. The 14-day pre-randomization adherence was correlated with adherence throughout the study. Patients were also more adherent during working days compared to non-working days. Oldest patients took twice as long to complete PROMs throughout the study; however, the response time for all patients decreased during the first month of the study regardless of age. Response time increased 7 days before and after the date of a scheduled clinic visit and when a patient-reported higher symptom burden.
CONCLUSIONS: Detailed analyses of adherence and response time for daily PROMs in clinical trials can provide significant insights about trends of patient behavior in longitudinal clinical studies with high baseline adherence.
METHODS: Data were collected from 1342 patients randomized into three respiratory clinical trials (NCT03401229, NCT03347279, and NCT03406078). PROMs were completed by patients using electronic handheld devices that collected the starting and completion times. A Bayesian generalized linear mixed-effects model was used to identify unbiased coefficients associated with PROM adherence and response time using patient, site, and calendar features as covariates.
RESULTS: Adherence decreased over time after randomization, and the rate of decrease was higher in younger patients. The 14-day pre-randomization adherence was correlated with adherence throughout the study. Patients were also more adherent during working days compared to non-working days. Oldest patients took twice as long to complete PROMs throughout the study; however, the response time for all patients decreased during the first month of the study regardless of age. Response time increased 7 days before and after the date of a scheduled clinic visit and when a patient-reported higher symptom burden.
CONCLUSIONS: Detailed analyses of adherence and response time for daily PROMs in clinical trials can provide significant insights about trends of patient behavior in longitudinal clinical studies with high baseline adherence.
摘要：
目的：患者报告的结果指标（PROMs）用于收集疾病症状数据，以支持临床试验终点。临床研究可以持续一年或更长时间，患者对每日家庭问卷的依从性和响应时间可能会随着时间的推移而显著变化。这项研究的目的是了解患者在纵向临床研究中完成每日PROM的模式和变化。
方法：收集了1342例患者的数据，这些患者随机分为3项呼吸临床试验（NCT03401229、NCT03347279和NCT03406078）。PROM由患者使用收集开始和完成时间的电子手持设备完成。使用贝叶斯广义线性混合效应模型来识别与患者的PROM依从性和响应时间相关的无偏系数，site,和日历功能作为协变量。
结果：随机化后，依从性随着时间的推移而下降，年轻患者的下降率较高。在整个研究中，14天的随机化前依从性与依从性相关。与非工作日相比，工作日患者的依从性也更高。在整个研究中，年龄最大的患者花费了两倍的时间来完成PROM；然而，在研究的第1个月,所有患者的应答时间均减少,而与年龄无关.在预定的诊所就诊日期之前和之后以及患者报告的症状负担较高时，响应时间增加了7天。
结论：对临床试验中每日PROMs的依从性和响应时间进行详细分析，可以为具有高基线依从性的纵向临床研究中患者行为趋势提供重要见解。
方法：收集了1342例患者的数据，这些患者随机分为3项呼吸临床试验（NCT03401229、NCT03347279和NCT03406078）。PROM由患者使用收集开始和完成时间的电子手持设备完成。使用贝叶斯广义线性混合效应模型来识别与患者的PROM依从性和响应时间相关的无偏系数，site,和日历功能作为协变量。
结果：随机化后，依从性随着时间的推移而下降，年轻患者的下降率较高。在整个研究中，14天的随机化前依从性与依从性相关。与非工作日相比，工作日患者的依从性也更高。在整个研究中，年龄最大的患者花费了两倍的时间来完成PROM；然而，在研究的第1个月,所有患者的应答时间均减少,而与年龄无关.在预定的诊所就诊日期之前和之后以及患者报告的症状负担较高时，响应时间增加了7天。
结论：对临床试验中每日PROMs的依从性和响应时间进行详细分析，可以为具有高基线依从性的纵向临床研究中患者行为趋势提供重要见解。
