PDF(Pubmed)
Abstract：
UNASSIGNED: 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.
UNASSIGNED: The ADPKD Registry is hosted online with multiple disease-specific patient-reported outcomes modules to characterize the patient experience in the United States.
UNASSIGNED: 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.
UNASSIGNED: Patient-reported latest estimated glomerular filtration rate or creatinine used to calculate stage of chronic kidney disease.
UNASSIGNED: Health-related quality of life, measured using validated ADPKD-specific tools; access to polycystic kidney disease-specific health care.
UNASSIGNED: 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.
UNASSIGNED: 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.
UNASSIGNED: Currently, all the information collected is patient reported without health record validation of clinical variables.
UNASSIGNED: 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.
The Autosomal Dominant Polycystic Kidney Disease Registry is a longitudinal, patient-powered research tool created with the goal to better understand the impacts of ADPKD on affected individuals in the United States. Here, we analyze pain and other health-related quality of life outcomes in 1,086 individuals using validated tools and comment on the utility of these tools for future use in clinical trials and observational studies. We found that sharp pain, dull pain, fullness discomfort, and other related impacts affected individuals across the disease spectrum, although some participants reported more dull pain in later stages (CKD stages 4 and 5). Future analysis of these trends over time will be valuable in understanding how to assess and address the burden of pain in autosomal dominant polycystic kidney disease.
UNASSIGNED: The ADPKD Registry is hosted online with multiple disease-specific patient-reported outcomes modules to characterize the patient experience in the United States.
UNASSIGNED: 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.
UNASSIGNED: Patient-reported latest estimated glomerular filtration rate or creatinine used to calculate stage of chronic kidney disease.
UNASSIGNED: Health-related quality of life, measured using validated ADPKD-specific tools; access to polycystic kidney disease-specific health care.
UNASSIGNED: 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.
UNASSIGNED: 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.
UNASSIGNED: Currently, all the information collected is patient reported without health record validation of clinical variables.
UNASSIGNED: 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.
The Autosomal Dominant Polycystic Kidney Disease Registry is a longitudinal, patient-powered research tool created with the goal to better understand the impacts of ADPKD on affected individuals in the United States. Here, we analyze pain and other health-related quality of life outcomes in 1,086 individuals using validated tools and comment on the utility of these tools for future use in clinical trials and observational studies. We found that sharp pain, dull pain, fullness discomfort, and other related impacts affected individuals across the disease spectrum, although some participants reported more dull pain in later stages (CKD stages 4 and 5). Future analysis of these trends over time will be valuable in understanding how to assess and address the burden of pain in autosomal dominant polycystic kidney disease.
摘要：
■常染色体显性多囊肾病（ADPKD）影响健康相关生活质量（HRQoL），包括疼痛，不适,疲劳,情绪困扰,和行动不便。利益相关者将肾囊肿相关性疼痛作为临床试验中重要的核心结果领域，导致特定疾病评估工具的开发。
ADPKDRegistry在线托管，其中包含多种疾病特异性患者报告结果模块，以描述美国患者的经历。
■ADPKD注册表允许同意的参与者访问包括人口统计在内的核心问卷，合并症条件，目前的症状，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-疼痛和不适量表(ADPKD-PDS)，ADPKD影响量表(ADPKD-IS)和医疗保健访问和利用模块。
■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病的分期。
■与健康相关的生活质量，使用经过验证的ADPKD特异性工具进行测量；获得多囊肾疾病特异性医疗保健。
■对于2种HRQoL工具，分数是针对身体计算的，情感,和疲劳领域；疼痛严重程度；和疼痛干扰（基于许可的用户手册）。还评估了获得医疗保健的关联。
■到2022年7月，1,086名ADPKD患者完成了至少1个HRQoL模块，319完成了4年。参与者的平均年龄为53岁。总的来说,71%是女性,91%是白人,以所有慢性肾病（CKD）阶段为代表。总的来说,2.5%报告接受透析治疗，23%的人接受了肾脏移植。CKD4/5期参与者报告的肾痛最沉闷，而剧烈的肾痛在CKD早期阶段分布均匀。无论CKD阶段如何，肾痛对睡眠都有影响。ADPKD-PDS与ADPKD-IS之间呈显著正相关。HRQoL中性或阳性的患者不太可能被拒绝接受成像或其他护理。
■目前，收集的所有信息均为患者报告,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中整个CKD频谱的疼痛负担的细粒度信息。ADPKD注册允许评估ADPKD对社区的影响，该社区数十年来健康和肾功能下降。
常染色体显性多囊肾病登记是纵向的，目的是更好地了解ADPKD对美国受影响个体的影响。这里,我们使用经过验证的工具分析了1,086例患者的疼痛和其他与健康相关的生活质量结局,并评论了这些工具在未来临床试验和观察性研究中的实用性.我们发现剧痛，隐隐作痛,饱腹不适，和其他相关影响影响整个疾病谱的个体，尽管一些参与者在后期（CKD第4和第5阶段）报告了更多的隐痛。随着时间的推移，对这些趋势的未来分析对于理解如何评估和解决常染色体显性多囊肾疾病的疼痛负担将是有价值的。
ADPKDRegistry在线托管，其中包含多种疾病特异性患者报告结果模块，以描述美国患者的经历。
■ADPKD注册表允许同意的参与者访问包括人口统计在内的核心问卷，合并症条件，目前的症状，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-疼痛和不适量表(ADPKD-PDS)，ADPKD影响量表(ADPKD-IS)和医疗保健访问和利用模块。
■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病的分期。
■与健康相关的生活质量，使用经过验证的ADPKD特异性工具进行测量；获得多囊肾疾病特异性医疗保健。
■对于2种HRQoL工具，分数是针对身体计算的，情感,和疲劳领域；疼痛严重程度；和疼痛干扰（基于许可的用户手册）。还评估了获得医疗保健的关联。
■到2022年7月，1,086名ADPKD患者完成了至少1个HRQoL模块，319完成了4年。参与者的平均年龄为53岁。总的来说,71%是女性,91%是白人,以所有慢性肾病（CKD）阶段为代表。总的来说,2.5%报告接受透析治疗，23%的人接受了肾脏移植。CKD4/5期参与者报告的肾痛最沉闷，而剧烈的肾痛在CKD早期阶段分布均匀。无论CKD阶段如何，肾痛对睡眠都有影响。ADPKD-PDS与ADPKD-IS之间呈显著正相关。HRQoL中性或阳性的患者不太可能被拒绝接受成像或其他护理。
■目前，收集的所有信息均为患者报告,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中整个CKD频谱的疼痛负担的细粒度信息。ADPKD注册允许评估ADPKD对社区的影响，该社区数十年来健康和肾功能下降。
常染色体显性多囊肾病登记是纵向的，目的是更好地了解ADPKD对美国受影响个体的影响。这里,我们使用经过验证的工具分析了1,086例患者的疼痛和其他与健康相关的生活质量结局,并评论了这些工具在未来临床试验和观察性研究中的实用性.我们发现剧痛，隐隐作痛,饱腹不适，和其他相关影响影响整个疾病谱的个体，尽管一些参与者在后期（CKD第4和第5阶段）报告了更多的隐痛。随着时间的推移，对这些趋势的未来分析对于理解如何评估和解决常染色体显性多囊肾疾病的疼痛负担将是有价值的。
