Cho Yeoungjee, Sautenet Benedicte, Rangan Gopala, Craig Jonathan C, Ong Albert C M, Chapman Arlene, Ahn Curie, Chen Dongping, Coolican Helen, Kao Juliana Tze-Wah, Gansevoort Ron, Perrone Ronald, Harris Tess, Torres Vicente, Pei York, Kerr Peter G, Ryan Jessica, Gutman Talia, Howell Martin, Ju Angela, Manera Karine E, Teixeira-Pinto Armando, Hamiwka Lorraine A, Tong Allison
Department of Nephrology, Princess Alexandra Hospital, 199 Ipswich Road, Woolloongabba, Brisbane, QLD, 4102, Australia.
Australasian Kidney Trials Network, University of Queensland, Brisbane, Australia.
Trials. 2017 Nov 23;18(1):560. doi: 10.1186/s13063-017-2298-4.
Autosomal dominant polycystic kidney disease (ADPKD) is the most common potentially life threatening inherited kidney disease and is responsible for 5-10% of cases of end-stage kidney disease (ESKD). Cystic kidneys may enlarge up to 20 times the weight of a normal kidney due to the growth of renal cysts, and patients with ADPKD have an increased risk of morbidity, premature mortality, and other life-time complications including renal and hepatic cyst and urinary tract infection, intracranial aneurysm, diverticulosis, and kidney pain which impair quality of life. Despite some therapeutic advances and the growing number of clinical trials in ADPKD, the outcomes that are relevant to patients and clinicians, such as symptoms and quality of life, are infrequently and inconsistently reported. This potentially limits the contribution of trials to inform evidence-based decision-making. The Standardised Outcomes in Nephrology-Polycystic Kidney Disease (SONG-PKD) project aims to establish a consensus-based set of core outcomes for trials in PKD (with an initial focus on ADPKD but inclusive of all stages) that patients and health professionals identify as critically important.
The five phases of SONG-PKD are: a systematic review to identify outcomes that have been reported in existing PKD trials; focus groups with nominal group technique with patients and caregivers to identify, rank, and describe reasons for their choices; qualitative stakeholder interviews with health professionals to elicit individual values and perspectives on outcomes for trials involving patients with PKD; an international three-round Delphi survey with all stakeholder groups (including patients, caregivers, healthcare providers, policy makers, researchers, and industry) to gain consensus on critically important core outcome domains; and a consensus workshop to review and establish a set of core outcome domains and measures for trials in PKD.
The SONG-PKD core outcome set is aimed at improving the consistency and completeness of outcome reporting across ADPKD trials, leading to improvements in the reliability and relevance of trial-based evidence to inform decisions about treatment and ultimately improve the care and outcomes for people with ADPKD.
常染色体显性多囊肾病（ADPKD）是最常见的潜在威胁生命的遗传性肾病，占终末期肾病（ESKD）病例的5% - 10%。由于肾囊肿的生长，多囊肾的体积可能增大至正常肾脏重量的20倍，ADPKD患者出现发病、过早死亡以及其他终身并发症的风险增加，这些并发症包括肾囊肿和肝囊肿、尿路感染、颅内动脉瘤、憩室病以及肾痛，会损害生活质量。尽管在ADPKD治疗方面取得了一些进展，且临床试验数量不断增加，但与患者和临床医生相关的结果，如症状和生活质量，却很少被报告且报告不一致。这可能会限制试验对基于证据的决策的贡献。肾脏病 - 多囊肾病标准化结局（SONG - PKD）项目旨在为PKD试验（最初侧重于ADPKD，但涵盖所有阶段）建立一套基于共识的核心结局，患者和卫生专业人员认为这些结局至关重要。
SONG - PKD的五个阶段包括：系统回顾以确定现有PKD试验中报告的结局；与患者及护理人员进行名义群体技术焦点小组讨论，以确定、排序并描述其选择的原因；对卫生专业人员进行定性利益相关者访谈，以获取他们对涉及PKD患者试验结局的个人价值观和观点；对所有利益相关者群体（包括患者、护理人员、医疗保健提供者、政策制定者、研究人员和行业）进行三轮国际德尔菲调查，以就至关重要的核心结局领域达成共识；以及召开共识研讨会，以审查并建立一套PKD试验的核心结局领域和测量指标。
SONG - PKD核心结局集旨在提高ADPKD试验结局报告的一致性和完整性，从而提高基于试验的证据在治疗决策方面的可靠性和相关性，并最终改善ADPKD患者的护理和结局。
