背景:临床试验中越来越多地使用电子患者报告结果(ePRO)系统,以从患者角度提供治疗疗效和耐受性的证据。这项研究的目的是双重的:(1)描述我们如何开发一个电子平台,患者报告他们的症状,和(2)开发和进行用于细胞疗法研究的ePRO解决方案的可用性测试,以寻求提供治疗的有效性和耐受性的早期证据,并测试该系统用于后期研究的可行性。
方法:一种ePRO系统设计用于单臂,多中心,研究使用ORBCEL-C™治疗炎症患者的安全性和活性的II期篮式试验.ORBCEL-C™是使用CD362+细胞选择从人脐带组织分离的富集的间充质基质细胞产品。使用认知访谈和“大声思考”方法与患者咨询小组成员和研究护士进行可用性测试,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。衡量疲劳和与健康相关的生活质量,PRO-CTCAE™和FACT-GP5全球耐受性问题纳入ePRO系统.通过PRO-CTCAE™和FACT-GP5评分触发临床团队的警报通知。患者参与者喜欢面向患者的应用程序的简单性和响应性。两名患者无法完成测试,由于技术问题。研究护士建议稍作修改,以改善临床医生仪表板和培训材料的功能和布局。
结论:通过测试有效性,效率,以及我们新颖的EPRO系统(PROmicsR)的满意度,我们了解到,大多数患有炎症的人发现很容易在自己的设备上使用应用程序报告他们的症状。他们在试验环境中使用PROmicsRePRO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。研究护士也很积极,发现临床仪表板易于使用。为了提供治疗反应和耐受性的证据,在早期试验中使用epros很重要。增加证据基础,并为方法论开发提供信息。
背景:ISRCTN,ISRCTN80103507。2022年4月1日注册,https://www。isrctn.com/ISRCTN80103507.
越来越多的患者通过电子填写问卷来告诉临床医生他们的感受。因此,重要的是要评估患者这样做的容易程度。在这项研究中,我们描述了我们如何为患者开发一个电子平台来报告他们的症状,以及我们如何与患者合作伙伴和研究护士测试该平台的可用性.一旦电子平台被开发出来,生活质量和症状问卷被编程到它。如果在症状问卷上获得了具体分数,则会向临床团队发送警报。尽管由于技术问题,两名患者合作伙伴无法完成测试,完成会议的人喜欢它的简单性和响应性。研究护士也喜欢该系统,只建议进行微小的修改。在这个测试之后,我们对电子平台进行了改进,以便在一项更大的研究中对其进行进一步测试,该研究调查了药物的安全性和使用情况.我们希望感谢这个电子平台,我们将获得有关治疗安全性和有效性的有用信息。
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.
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.
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.
By testing the effectiveness, efficiency, and satisfaction of our novel ePRO system (PROmicsR), 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 PROmicsR 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.
ISRCTN, ISRCTN80103507. Registered 01 April 2022, https://www.isrctn.com/ISRCTN80103507.
More and more patients tell clinicians how they feel by completing questionnaires electronically. Therefore, it is important to assess how easy it is for patients to do this. In this study, we describe how we developed an electronic platform for patients to report their symptoms and how we tested the usability of this platform with patient partners and research nurses. Once the electronic platform was developed, quality of life and symptoms questionnaires were programmed onto it. Alerts were sent to the clinical team if specific scores were obtained on the symptoms questionnaires. Although two patient partners were not able to finish the testing session because of technical issues, the ones who completed the session liked its simplicity and responsiveness. The research nurses also liked the system and only suggested minor modifications. Following this testing, we refined the electronic platform to test it further in a larger
study which investigates the safety and use of a drug. We hope that thanks to this electronic platform, we will obtain useful information on the safety and efficacy of treatment.