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Abstract：
Patient-reported outcomes (PROs) that assess individuals\' perceptions of life participation, medication adherence, disease symptoms, and therapy side effects are extremely relevant in the context of kidney transplantation. All PROs are potentially suitable as primary or secondary endpoints in interventional trials that aim to improve outcomes for transplant recipients. Using PRO measures (PROMs) in clinical trials facilitates assessment of the patient\'s perspective of their health, but few measures have been developed and evaluated in kidney transplant recipients; robust methodologies, which use validated instruments and established frameworks for reporting, are essential. Establishing a core PROM for life participation in kidney transplant recipients is a critically important need, which is being developed and validated by the Standardized Outcomes in Nephrology (SONG)-Tx Initiative. Measures involving electronic medication packaging and smart technologies are gaining traction for monitoring adherence, and could provide more robust information than questionnaires, interviews, and scales. This article summarizes information on PROs and PROMs that was included in a Broad Scientific Advice request on clinical trial design and endpoints in kidney transplantation. This request was submitted to the European Medicines Agency (EMA) by the European Society for Organ Transplantation in 2016. Following modifications, the EMA provided its recommendations in late 2020.
摘要：
患者报告的结果(PRO)评估个人对生活参与的看法，药物依从性,疾病症状,和治疗副作用与肾移植密切相关。所有PRO都可能适合作为旨在改善移植受者预后的介入试验的主要或次要终点。在临床试验中使用PRO措施（PROM）有助于评估患者对其健康的看法，但在肾移植受者中很少有措施被开发和评估；稳健的方法，使用经过验证的工具和既定的报告框架，是必不可少的。为肾移植受者的生命参与建立一个核心的PROM是一个至关重要的需求，这正在由肾病学标准化结果（SONG）-Tx计划开发和验证。涉及电子药物包装和智能技术的措施正在获得监控依从性的吸引力，并且可以提供比问卷更可靠的信息，采访,和尺度。本文总结了有关肾移植临床试验设计和终点的广泛科学建议要求中包含的PRO和PROM的信息。该请求于2016年由欧洲器官移植协会提交给欧洲药品管理局（EMA）。修改后，EMA在2020年底提出了建议。
