Centre for Patient Reported Outcome Research (CPROR), Institute of Applied Health Research, University of Birmingham, Edgbaston, Birmingham, B15 2TT, UK.
NIHR Surgical Reconstruction and Microbiology Research Centre, University of Birmingham, Birmingham, UK.
J Patient Rep Outcomes. 2023 Oct 9;7(1):98. doi: 10.1186/s41687-023-00634-3.
BACKGROUND: Electronic patient-reported outcome (ePRO) systems are increasingly used in clinical trials to provide evidence of efficacy and tolerability of treatment from the patient perspective. The aim of this study is twofold: (1) to describe how we developed an electronic platform for patients to report their symptoms, and (2) to develop and undertake usability testing of an ePRO solution for use in a study of cell therapy seeking to provide early evidence of efficacy and tolerability of treatment and test the feasibility of the system for use in later phase studies. METHODS: An ePRO system was designed to be used in a single arm, multi-centre, phase II basket trial investigating the safety and activity of the use of ORBCEL-C™ in the treatment of patients with inflammatory conditions. ORBCEL-C™ is an enriched Mesenchymal Stromal Cells product isolated from human umbilical cord tissue using CD362+ cell selection. Usability testing sessions were conducted using cognitive interviews and the 'Think Aloud' method with patient advisory group members and Research Nurses to assess the usability of the system. RESULTS: Nine patient partners and seven research nurses took part in one usability testing session. Measures of fatigue and health-related quality of life, the PRO-CTCAE™ and FACT-GP5 global tolerability question were included in the ePRO system. Alert notifications to the clinical team were triggered by PRO-CTCAE™ and FACT-GP5 scores. Patient participants liked the simplicity and responsiveness of the patient-facing app. Two patients were unable to complete the testing session, due to technical issues. Research Nurses suggested minor modifications to improve functionality and the layout of the clinician dashboard and the training materials. CONCLUSION: By testing the effectiveness, efficiency, and satisfaction of our novel ePRO system (PROmics), we learnt that most people with an inflammatory condition found it easy to report their symptoms using an app on their own device. Their experiences using the PROmics ePRO system within a trial environment will be further explored in our upcoming feasibility testing. Research nurses were also positive and found the clinical dashboard easy-to-use. Using ePROs in early phase trials is important in order to provide evidence of therapeutic responses and tolerability, increase the evidence based, and inform methodology development. TRIAL REGISTRATION: ISRCTN, ISRCTN80103507. Registered 01 April 2022, https://www.isrctn.com/ISRCTN80103507.
背景:电子患者报告结局(ePRO)系统在临床试验中越来越多地被用于从患者角度提供治疗疗效和耐受性的证据。本研究的目的有两个:(1)描述我们如何开发一个电子平台,让患者报告自己的症状;(2)开发并进行电子患者报告结局(ePRO)解决方案的可用性测试,用于一项细胞治疗研究,旨在提供治疗疗效和耐受性的早期证据,并测试该系统在后期研究中的可行性。
方法:ePRO 系统旨在用于一项单臂、多中心、二期篮子试验,该试验调查了使用 ORBCEL-C™治疗炎症性疾病患者的安全性和活性。ORBCEL-C™是一种从人脐带组织中分离出来的富含间充质基质细胞的产品,使用 CD362+细胞选择。使用认知访谈和“大声思考”方法,让患者咨询小组成员和研究护士进行可用性测试会议,以评估系统的可用性。
结果:9 名患者伙伴和 7 名研究护士参加了一次可用性测试会议。疲劳和健康相关生活质量、PRO-CTCAE™和 FACT-GP5 整体耐受性问题被纳入 ePRO 系统。PRO-CTCAE™和 FACT-GP5 评分触发了对临床团队的警报通知。患者参与者喜欢患者端应用程序的简单性和响应性。由于技术问题,有两名患者无法完成测试。研究护士建议进行一些小的修改,以提高临床医生仪表板和培训材料的功能和布局。
结论:通过测试我们新型 ePRO 系统(PROmics)的有效性、效率和满意度,我们了解到大多数患有炎症性疾病的人发现使用自己设备上的应用程序报告自己的症状很容易。他们在试验环境中使用 PROmics ePRO 系统的经验将在我们即将进行的可行性测试中进一步探讨。研究护士也持积极态度,认为临床医生仪表板易于使用。在早期阶段试验中使用 ePRO 对于提供治疗反应和耐受性的证据、增加基于证据的证据以及为方法学发展提供信息非常重要。
试验注册:ISRCTN,ISRCTN80103507。于 2022 年 4 月 1 日注册,https://www.isrctn.com/ISRCTN80103507。
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