Use of daily electronic patient-reported outcome (PRO) diaries in randomized controlled trials for rheumatoid arthritis: rationale and implementation
Use of daily electronic patient-reported outcome (PRO) diaries in randomized controlled trials for rheumatoid arthritis: rationale and implementation
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DOI：
10.1186/s13063-019-3272-0
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发表时间：
2019-03-22
期刊：
影响因子：
2.5
通讯作者：
Taylor, Peter C.
中科院分区：
文献类型：
--
作者：
Bingham, Clifton O., III;Gaich, Carol L.;Taylor, Peter C.
背景风湿性关节炎（RA）与显著降低的健康相关生活质量相关。患者报告的结局（PRO）在RA中被认为是重要的;然而，美国风湿病学会RA试验的“核心集”指标未捕获患者认为重要的一些症状，如晨起关节僵硬（MJS）和疲劳。美国食品和药物管理局已批准临床试验数据的电子采集，包括PRO，与纸质方法相比，电子PRO（ePRO）系统可能会导致更准确和完整的数据采集，提高依从性和患者接受度。我们的目的是评估在baricitinib RA-BEAM和RA-BUILD 3期随机临床试验（RCT）中实施ePRO测量MJS的持续时间和严重程度、最严重疲劳的严重程度和最严重关节疼痛的严重程度。用于采集RCT中的PRO数据。纳入了三个“报告窗口”选项，以适应患者日常日程安排的差异，并为每个报告窗口编程报警。MJS的持续时间以“小时和分钟”记录，MJS的严重程度、最严重的疲劳和最严重的关节疼痛以0 - 10的评分量表记录，评分越高表示症状越严重。结果中重度活动性RA患者在RA-BEAM研究（N = 1305）和RA-BUILD研究（N = 684）中使用电子日志。平均依从性（计算为患者完成日志的总天数与预期完成日志的总天数相比）在第12周较高（RA-BEAM 94%的患者; RA-BUILD 93%的患者），可能归因于适当的培训、说明的清晰度、简单的用户界面和电子器械设计。已确定的流程挑战包括设备发放不及时、电池电量低、数据收集前对患者的培训不足、日记设置不当以及基线访视后1天首次响应录入。结论高依从性支持使用大型随机对照试验中的每日电子PRO日记。尽管存在预期的问题，但预计每日电子日志将减少回忆偏倚并提高PRO数据收集的质量。
Background Rheumatoid arthritis (RA) is associated with significantly diminished health-related quality of life. Patient-reported outcomes (PROs) are considered important in RA; however, some symptoms such as morning joint stiffness (MJS) and fatigue that are considered important by patients are not captured by the American College of Rheumatology "core set" measures for RA trials. The US Food and Drug Administration has endorsed electronic capture of clinical trial data including PROs, and electronic PRO (ePRO) systems may lead to more accurate and complete data capture, improved compliance, and patient acceptance compared with paper-based methods. Our objective was to assess the implementation of ePRO measures of Duration and Severity of MJS, Severity of Worst Tiredness, and Severity of Worst Joint Pain in baricitinib RA-BEAM and RA-BUILD phase 3 randomized clinical trials (RCTs).Methods A daily electronic diary (handheld device; Invivodata (R), Inc.) was utilized to capture PRO data in the RCTs. Three "reporting window" options were incorporated to accommodate differences in patients' routine daily schedules, and alarms were programmed for each reporting window. Duration of MJS was recorded in "hours and minutes," and Severity of MJS, Worst Tiredness, and Worst Joint Pain were captured on a 0 to 10 rating scale, with a higher score indicating more severe symptoms. The patients and site staff were trained to use the daily electronic diary.Results Patients with moderately to severely active RA used the daily electronic diary in the RA-BEAM study (N = 1305) and RA-BUILD study (N = 684). The average compliance, calculated as total days completed by patients compared with total days expected to complete the diary, through Week 12 was high (RA-BEAM 94% patients; RA-BUILD 93% patients), potentially attributable to appropriate training, clarity of instructions, simple user interface, and electronic device design. Identified process challenges included non-timely issuance of the device, low battery, inadequate training of patients before data collection, inappropriate diary set-up, and first response entry 1 day after the baseline visit.Conclusions High compliance rates support the use of the daily electronic PRO diary in large RCTs. Despite the anticipated issues, the daily electronic diary is expected to reduce recall bias and improve the quality of PRO data collection.