Hoover Elise, Holliday Vanessa, Merullo Nicole, Oberdhan Dorothee, Perrone Ronald D, Rusconi Chris, Park Meyeon, Phadnis Milind A, Thewarapperuma Nadeesha, Dahl Neera K
PKD Foundation, Kansas City, MO.
Department of Medicine, Division of Nephrology, Tufts Medical Center, Boston, MA.
Kidney Med. 2024 Mar 24;6(5):100813. doi: 10.1016/j.xkme.2024.100813. eCollection 2024 May.
RATIONALE & OBJECTIVE: Autosomal dominant polycystic kidney disease (ADPKD) affects health-related quality of life (HRQoL) including pain, discomfort, fatigue, emotional distress, and impaired mobility. Stakeholders prioritized kidney cyst-related pain as an important core outcome domain in clinical trials, leading to the development of disease-specific assessment tools.
The ADPKD Registry is hosted online with multiple disease-specific patient-reported outcomes modules to characterize the patient experience in the United States.
SETTING & PARTICIPANTS: The ADPKD Registry allows consented participants access to a Core Questionnaire that includes demographics, comorbid conditions, current symptoms, and kidney function. Participants complete subsequent modules on a 3-month schedule, including 2 validated HRQoL tools, the ADPKD-Pain and Discomfort Scale (ADPKD-PDS), the ADPKD Impact Scale (ADPKD-IS) and a Healthcare Access and Utilization module.
Patient-reported latest estimated glomerular filtration rate or creatinine used to calculate stage of chronic kidney disease.
Health-related quality of life, measured using validated ADPKD-specific tools; access to polycystic kidney disease-specific health care.
For the 2 HRQoL tools, scores were calculated for physical, emotional, and fatigue domains; pain severity; and pain interference (based on the licensed user manuals). Associations to health care access were also assessed.
By July 2022, 1,086 individuals with ADPKD completed at least 1 of the HRQoL modules, and 319 completed 4 over a year. Participants were an average age of 53. In total, 71% were women, and 91% were White, with all chronic kidney disease (CKD) stages represented. In total, 2.5% reported being treated with dialysis, and 23% had a kidney transplant. CKD stage 4/5 participants reported the most dull kidney pain, whereas sharp kidney pain was evenly distributed across early CKD stages. Dull kidney pain had an impact on sleep regardless of CKD stage. There was a strong positive correlation between the ADPKD-PDS and ADPKD-IS. Patients with a neutral or positive HRQoL were less likely to have been denied access to imaging or other care.
Currently, all the information collected is patient reported without health record validation of clinical variables.
Use of the HRQoL tools in the ADPKD Registry provided a broad cross-sectional assessment in the United States and provided granular information on the burden of pain across the CKD spectrum in ADPKD. The ADPKD Registry allowed assessment of ADPKD impact in a community that experiences decline in health and kidney function over decades.
常染色体显性多囊肾病（ADPKD）会影响与健康相关的生活质量（HRQoL），包括疼痛、不适、疲劳、情绪困扰和行动能力受损。利益相关者将肾囊肿相关疼痛列为临床试验中一个重要的核心结局领域，这促使了针对该疾病的评估工具的开发。
ADPKD注册库在线托管，包含多个针对该疾病的患者报告结局模块，以描述美国患者的经历。
ADPKD注册库允许获得同意的参与者访问一份核心问卷，该问卷包括人口统计学信息、合并症、当前症状和肾功能。参与者按3个月的时间表完成后续模块，包括2个经过验证的HRQoL工具、ADPKD疼痛与不适量表（ADPKD-PDS）、ADPKD影响量表（ADPKD-IS）以及一个医疗保健获取与利用模块。
患者报告的最新估计肾小球滤过率或肌酐，用于计算慢性肾脏病阶段。
使用经过验证的特定于ADPKD的工具测量的与健康相关的生活质量；获得多囊肾病专科医疗保健的情况。
对于这2个HRQoL工具，计算身体、情绪和疲劳领域的得分；疼痛严重程度；以及疼痛干扰（根据许可用户手册）。还评估了与医疗保健获取情况的关联。
截至2022年7月，1086名ADPKD患者完成了至少1个HRQoL模块，319名患者在一年中完成了4个模块。参与者的平均年龄为53岁。总体而言，71%为女性，91%为白人，涵盖了所有慢性肾脏病（CKD）阶段。共有2.5%的患者报告正在接受透析治疗，23%的患者进行了肾移植。CKD 4/5期的参与者报告钝痛最为常见，而锐痛在早期CKD阶段分布较为均匀。无论CKD处于何阶段，钝痛都会影响睡眠。ADPKD-PDS与ADPKD-IS之间存在强正相关。HRQoL为中性或阳性的患者被拒绝进行影像学检查或其他治疗的可能性较小。
目前，所有收集到的信息均为患者报告，未对临床变量进行健康记录验证。
在ADPKD注册库中使用HRQoL工具在美国提供了广泛的横断面评估，并提供了关于ADPKD患者在整个CKD范围内疼痛负担的详细信息。ADPKD注册库能够评估ADPKD对一个健康和肾功能在数十年间逐渐下降的群体的影响。
