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      Development and Validation of a Nomogram for Renal Survival Prediction in Patients with Autosomal Dominant Polycystic Kidney Disease

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          Abstract

          Introduction

          Due to the wide variation in the prognosis of autosomal dominant polycystic kidney disease (ADPKD), prediction of risk of renal survival in ADPKD patients is a tough challenge. We aimed to establish a nomogram for the prediction of renal survival in ADPKD patients.

          Methods

          We conducted a retrospective observational cohort study in 263 patients with ADPKD. The patients were randomly assigned to a training set ( N = 198) and a validation set ( N = 65), and demographic and statistical data at baseline were collected. The total kidney volume was measured using stereology. A clinical prediction nomogram was developed based on multivariate Cox regression results. The performance and clinical utility of the nomogram were assessed by calibration curves, the concordance index (C-index), and decision curve analysis (DCA). The nomogram was compared with the height-adjusted total kidney volume (htTKV) model by receiver operating characteristic curve analysis and DCA.

          Results

          The five independent factors used to construct the nomogram for prognosis prediction were age, htTKV, estimated glomerular filtration rate, hypertension, and hemoglobin. The calibration curve of predicted probabilities against observed renal survival indicated excellent concordance. The model showed very good discrimination with a C-index of 0.91 (0.83–0.99) and an area under the curve of 0.94, which were significantly higher than those of the htTKV model. Similarly, DCA demonstrated that the nomogram had a better net benefit than the htTKV model.

          Conclusion

          The risk prediction nomogram, incorporating easily assessable clinical parameters, was effective for the prediction of renal survival in ADPKD patients. It can be a useful clinical adjunct for clinicians to evaluate the prognosis of ADPKD patients and provide individualized decision-making.

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          Most cited references37

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          Decision curve analysis: a novel method for evaluating prediction models.

          Diagnostic and prognostic models are typically evaluated with measures of accuracy that do not address clinical consequences. Decision-analytic techniques allow assessment of clinical outcomes but often require collection of additional information and may be cumbersome to apply to models that yield a continuous result. The authors sought a method for evaluating and comparing prediction models that incorporates clinical consequences,requires only the data set on which the models are tested,and can be applied to models that have either continuous or dichotomous results. The authors describe decision curve analysis, a simple, novel method of evaluating predictive models. They start by assuming that the threshold probability of a disease or event at which a patient would opt for treatment is informative of how the patient weighs the relative harms of a false-positive and a false-negative prediction. This theoretical relationship is then used to derive the net benefit of the model across different threshold probabilities. Plotting net benefit against threshold probability yields the "decision curve." The authors apply the method to models for the prediction of seminal vesicle invasion in prostate cancer patients. Decision curve analysis identified the range of threshold probabilities in which a model was of value, the magnitude of benefit, and which of several models was optimal. Decision curve analysis is a suitable method for evaluating alternative diagnostic and prognostic strategies that has advantages over other commonly used measures and techniques.
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            Multivariable prognostic models: issues in developing models, evaluating assumptions and adequacy, and measuring and reducing errors.

            Multivariable regression models are powerful tools that are used frequently in studies of clinical outcomes. These models can use a mixture of categorical and continuous variables and can handle partially observed (censored) responses. However, uncritical application of modelling techniques can result in models that poorly fit the dataset at hand, or, even more likely, inaccurately predict outcomes on new subjects. One must know how to measure qualities of a model's fit in order to avoid poorly fitted or overfitted models. Measurement of predictive accuracy can be difficult for survival time data in the presence of censoring. We discuss an easily interpretable index of predictive discrimination as well as methods for assessing calibration of predicted survival probabilities. Both types of predictive accuracy should be unbiasedly validated using bootstrapping or cross-validation, before using predictions in a new data series. We discuss some of the hazards of poorly fitted and overfitted regression models and present one modelling strategy that avoids many of the problems discussed. The methods described are applicable to all regression models, but are particularly needed for binary, ordinal, and time-to-event outcomes. Methods are illustrated with a survival analysis in prostate cancer using Cox regression.
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              Nomograms in oncology: more than meets the eye.

              Nomograms are widely used as prognostic devices in oncology and medicine. With the ability to generate an individual probability of a clinical event by integrating diverse prognostic and determinant variables, nomograms meet our desire for biologically and clinically integrated models and fulfill our drive towards personalised medicine. Rapid computation through user-friendly digital interfaces, together with increased accuracy, and more easily understood prognoses compared with conventional staging, allow for seamless incorporation of nomogram-derived prognosis to aid clinical decision making. This has led to the appearance of many nomograms on the internet and in medical journals, and an increase in nomogram use by patients and physicians alike. However, the statistical foundations of nomogram construction, their precise interpretation, and evidence supporting their use are generally misunderstood. This issue is leading to an under-appreciation of the inherent uncertainties regarding nomogram use. We provide a systematic, practical approach to evaluating and comprehending nomogram-derived prognoses, with particular emphasis on clarifying common misconceptions and highlighting limitations.

                Author and article information

                Journal
                Kidney Dis (Basel)
                Kidney Dis (Basel)
                KDD
                KDD
                Kidney Diseases
                S. Karger AG (Basel, Switzerland )
                2296-9381
                2296-9357
                6 June 2023
                October 2023
                : 9
                : 5
                : 398-407
                Affiliations
                [a ]Department of Nephrology, The First Affiliated Hospital of Wenzhou Medical University, Wenzhou, China
                [b ]Department of Nephrology, Affiliated Hangzhou First People’s Hospital, Zhejiang University School of Medicine, Hangzhou, China
                [c ]Department of Critical Care Medicine, Sir Run Run Shaw Hospital, School of Medicine, Zhejiang University, Hangzhou, China
                [d ]Research Center for Intelligent Supercomputing, Zhejiang Laboratory, Hangzhou, China
                [e ]Department of Radiology, The First Affiliated Hospital of Wenzhou Medical University, Wenzhou, China
                Author notes
                Correspondence to: Zhen Su, cnsuzhen@ 123456wmu.edu.cn

                Xiaomei Wang, Rui Zheng, and Zhende Liu have contributed equally to this work.

                Article
                531329
                10.1159/000531329
                10601962
                5549e06d-085b-4db5-9046-9fe24524c18d
                © 2023 The Author(s). Published by S. Karger AG, Basel

                This article is licensed under the Creative Commons Attribution-NonCommercial 4.0 International License (CC BY-NC) ( http://www.karger.com/Services/OpenAccessLicense). Usage and distribution for commercial purposes requires written permission.

                History
                : 13 August 2022
                : 23 May 2023
                : 2023
                Page count
                Figures: 4, Tables: 3, References: 38, Pages: 10
                Funding
                This work was supported by grants from the National Natural Science Foundation of China (81671403, 30871179) for Zhen Su, Zhejiang Provincial Health Science and Technology Foundation of China (2021KY203) for Zhen Su, and Special Support Plan for High-level Talents in Wenzhou – Famous Doctors in Wenzhou for Zhen Su.
                Categories
                Research Article

                nomogram,autosomal dominant polycystic kidney disease,renal survival,prediction model,end-stage renal disease

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