Mustafa Reem A, Kawtharany Hassan, Kalot Mohamad A, Lumpkins Crystal Y, Kimminau Kim S, Creed Cathy, Fowler Kevin, Perrone Ronald D, Jaure Allison, Cho Yeoungjee, Baron David, Yu Alan S L
Division of Nephrology and Hypertension,Department of Internal Medicine, University of Kansas Medical Centre, Kansas City, Kansas.
Evidence-Based Practice and Impact Center, Department of Internal Medicine, University of Kansas Medical Center, Kansas City, Kansas.
Kidney360. 2025 Apr 1;6(4):573-582. doi: 10.34067/KID.0000000695. Epub 2025 Jan 14.
Participants are less concerned about mortality than they are about kidney health. Psychological effect emerged as the most important theme regarding the effects of polycystic kidney disease on patients.
Patient involvement in research can help to ensure that the evidence generated aligns with their needs and priorities. In the Establishing Meaningful Patient-Centered Outcomes with Relevance for Patients with Polycystic Kidney Disease (PKD) project, we aimed to identify patient-important outcomes and discuss the effect of PKD on patients.
Nine focus groups were held with adult patients with PKD, caregivers, and clinical or research experts in PKD. We used a nominal, multivote technique to rank patient-important outcomes to be prioritized by future PKD research. We conducted a thematic analysis of verbatim transcriptions to identify themes regarding the effect of PKD on their daily lives. Other focus group topics included insurability and patient engagement.
Ninety patients and/or caregivers and eight clinicians and/or researchers participated in the focus groups. Nine focus groups yielded 35 outcomes important to patients that were grouped into six categories, ranked in order of importance: kidney health, comorbidities, lifestyle, psychological effect, family and awareness, and mortality. Regarding the effect of PKD on the patient's daily lives, we identified five themes, listed in order of importance: psychological effect, effect on daily living, issues affecting decision making, health care-related issues, and PKD-specific testing dilemmas.
This study of stakeholder engagement in patients with PKD revealed important priorities and values that should be considered for future research and when caring for patients with PKD. Future research should focus on kidney health and managing comorbidities in patients with PKD. This will help to bridge the knowledge gap and develop meaningful comparative effectiveness research in PKD.
参与者对死亡率的关注低于对肾脏健康的关注。心理影响成为多囊肾病对患者影响方面最重要的主题。
患者参与研究有助于确保所产生的证据符合他们的需求和优先事项。在“为多囊肾病（PKD）患者建立有意义的以患者为中心的结局”项目中，我们旨在确定对患者重要的结局，并讨论PKD对患者的影响。
与成年PKD患者、护理人员以及PKD方面的临床或研究专家进行了9次焦点小组讨论。我们使用名义多投票技术对未来PKD研究应优先考虑的对患者重要的结局进行排序。我们对逐字记录进行了主题分析，以确定PKD对他们日常生活影响的主题。其他焦点小组主题包括可保险性和患者参与度。
90名患者和/或护理人员以及8名临床医生和/或研究人员参加了焦点小组讨论。9次焦点小组讨论得出了35项对患者重要的结局，这些结局被分为六类，按重要性排序：肾脏健康、合并症、生活方式、心理影响、家庭与认知以及死亡率。关于PKD对患者日常生活的影响，我们确定了五个主题，按重要性排序：心理影响、对日常生活的影响、影响决策的问题、医疗保健相关问题以及PKD特异性检测困境。
这项关于PKD患者利益相关者参与的研究揭示了未来研究以及照顾PKD患者时应考虑的重要优先事项和价值观。未来的研究应关注PKD患者的肾脏健康和合并症管理。这将有助于弥合知识差距，并开展有意义的PKD比较疗效研究。
