van Rijn Marieke H C, van de Luijtgaarden Moniek, van Zuilen Arjan D, Blankestijn Peter J, Wetzels Jack F M, Debray Thomas P A, van den Brand Jan A J G
Department of Nephrology, Radboud Institute of Health Sciences, Radboud University Medical Center, Nijmegen, The Netherlands.
Department of Nephrology, University Medical Center Utrecht, Utrecht, The Netherlands.
Nephrol Dial Transplant. 2021 Sep 27;36(10):1837-1850. doi: 10.1093/ndt/gfaa155.
Accurate risk prediction is needed in order to provide personalized healthcare for chronic kidney disease (CKD) patients. An overload of prognosis studies is being published, ranging from individual biomarker studies to full prediction studies. We aim to systematically appraise published prognosis studies investigating multiple biomarkers and their role in risk predictions. Our primary objective was to investigate if the prognostic models that are reported in the literature were of sufficient quality and to externally validate them.
We undertook a systematic review and appraised the quality of studies reporting multivariable prognosis models for end-stage renal disease (ESRD), cardiovascular (CV) events and mortality in CKD patients. We subsequently externally validated these models in a randomized trial that included patients from a broad CKD population.
We identified 91 papers describing 36 multivariable models for prognosis of ESRD, 50 for CV events, 46 for mortality and 17 for a composite outcome. Most studies were deemed of moderate quality. Moreover, they often adopted different definitions for the primary outcome and rarely reported full model equations (21% of the included studies). External validation was performed in the Multifactorial Approach and Superior Treatment Efficacy in Renal Patients with the Aid of Nurse Practitioners trial (n = 788, with 160 events for ESRD, 79 for CV and 102 for mortality). The 24 models that reported full model equations showed a great variability in their performance, although calibration remained fairly adequate for most models, except when predicting mortality (calibration slope >1.5).
This review shows that there is an abundance of multivariable prognosis models for the CKD population. Most studies were considered of moderate quality, and they were reported and analysed in such a manner that their results cannot directly be used in follow-up research or in clinical practice.
为了给慢性肾脏病（CKD）患者提供个性化医疗服务，需要准确的风险预测。目前，从单个生物标志物研究到全面预测研究，大量的预后研究不断发表。我们旨在系统评估已发表的关于多种生物标志物及其在风险预测中作用的预后研究。我们的主要目标是调查文献中报道的预后模型是否具有足够的质量，并对其进行外部验证。
我们进行了一项系统综述，并评估了报告慢性肾脏病患者终末期肾病（ESRD）、心血管（CV）事件和死亡率多变量预后模型的研究质量。随后，我们在一项随机试验中对这些模型进行了外部验证，该试验纳入了广泛的慢性肾脏病患者群体。
我们确定了91篇论文，其中描述了36个用于ESRD预后的多变量模型、50个用于CV事件的模型、46个用于死亡率的模型以及17个用于复合结局的模型。大多数研究被认为质量中等。此外，它们对主要结局往往采用不同的定义，并且很少报告完整的模型方程（纳入研究的21%）。在借助执业护士的多因素方法和肾脏患者卓越治疗疗效试验（n = 788，其中ESRD事件160例，CV事件79例，死亡事件102例）中进行了外部验证。报告完整模型方程的24个模型在性能上表现出很大差异，尽管大多数模型的校准仍然相当充分，但预测死亡率时除外（校准斜率>1.5）。
本综述表明，慢性肾脏病群体有大量的多变量预后模型。大多数研究被认为质量中等，并且它们的报告和分析方式使得其结果不能直接用于后续研究或临床实践。
