Standardised Outcomes in Nephrology-Polycystic Kidney Disease (SONG-PKD): study protocol for establishing a core outcome set in polycystic kidney disease

BACKGROUND: 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. METHODS: 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. DISCUSSION: 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

Artificial neural networks and robust Bayesian classifiers for risk stratification following uncomplicated myocardial infarction

OBJECTIVE: To compare artificial neural networks (ANN) and robust Bayesian classifiers (RBC) in predicting outcome following acute myocardial infarction (AMI). METHODS: Clinical, exercise ECG and stress echo variables by 496 patients with AMI were used to predict the cumulative end-point of cardiac death, nonfatal reinfarction and unstable angina. Revascularized patients were censored. Short (200 days)-, medium (400 days)- and long (1000 days)-term observation intervals, including 50%, 75% and 90% of the events, respectively, were considered. At each interval, any patient was binary assigned to the "event" or "no event" class. A multilayer feedforward ANN, trained by a back propagation algorithm, was used. RBC, using the leave-one-out technique, were derived. The accuracy of both techniques was compared to the default accuracy (DA) obtained by assigning all subjects to the largest class. RESULTS: 14 death, 27 reinfarction and 29 unstable angina were observed during a mean follow-up of 24 [95% confidence interval (CI) 19 to 22] months. The accuracy of ANN and RBC and DA were 70%, 81% and 74% at short, 67%, 73% and 56% at medium and 64%, 68% and 62% at long-term follow-up. CONCLUSIONS: (1) ANN do not improve the prognostic classification of patients with uncomplicated AMI as compared to RBC. (2) In particular, short-term prognostic accuracy seems insufficient