Cardiovascular outcomes reported in hemodialysis trials

Patients on long-term hemodialysis are at very high risk for cardiovascular disease but are usually excluded from clinical trials conducted in the general population or in at-risk populations. There are no universally agreed cardiovascular outcomes for trials conducted specifically in the hemodialysis population. In this review, we highlight that trials reporting cardiovascular outcomes in hemodialysis patients are usually of short duration (median 3 to 6 months) and are small (59% of trials have \u3c100 participants). Overall, the cardiovascular outcomes are very heterogeneous and may not reflect outcomes that are meaningful to patients and clinicians in supporting decision making, as they are often surrogates of uncertain clinical importance. Composite outcomes used in different trials rarely share the same components. In a field in which a single trial is often insufficiently powered to fully assess the clinical and economic impact of interventions, differences in outcome reporting across trials make the task of meta-analysis and interpretation of all the available evidence challenging. Core outcome sets are now being established across many specialties in health care to prevent these problems. Through the global Standardized Outcomes in Nephrology-Hemodialysis initiative, cardiovascular disease was identified as a critically important core domain to be reported in all trials in hemodialysis. Informed by the current state of reporting of cardiovascular outcomes, a core outcome measure for cardiovascular disease is currently being established with involvement of patients, caregivers, and health professionals. Consistent reporting of cardiovascular outcomes that are critically important to hemodialysis patients and clinicians will strengthen the evidence base to inform care in this very high-risk population

A Universal Bleeding Risk Score in Native and Allograft Kidney Biopsies: A French Nationwide Cohort Study

BackgroundThe risk of bleeding after percutaneous biopsy in kidney transplant recipients is usually low but may vary. A pre-procedure bleeding risk score in this population is lacking.MethodsWe assessed the major bleeding rate (transfusion, angiographic intervention, nephrectomy, hemorrhage/hematoma) at 8 days in 28,034 kidney transplant recipients with a kidney biopsy during the 2010-2019 period in France and compared them to 55,026 patients with a native kidney biopsy as controls.ResultsThe rate of major bleeding was low (angiographic intervention: 0.2%, hemorrhage/hematoma: 0.4%, nephrectomy: 0.02%, blood transfusion: 4.0%). A new bleeding risk score was developed (anemia = 1, female gender = 1, heart failure = 1, acute kidney failure = 2 points). The rate of bleeding varied: 1.6%, 2.9%, 3.7%, 6.0%, 8.0%, and 9.2% for scores 0 to 5, respectively, in kidney transplant recipients. The ROC AUC was 0.649 (0.634-0.664) in kidney transplant recipients and 0.755 (0.746-0.763) in patients who had a native kidney biopsy (rate of bleeding: from 1.2% for score = 0 to 19.2% for score = 5).ConclusionsThe risk of major bleeding is low in most patients but indeed variable. A new universal risk score can be helpful to guide the decision concerning kidney biopsy and the choice of inpatient vs. outpatient procedure both in native and allograft kidney recipients

Range and variability of outcomes reported in randomized trials conducted in patients with polycystic kidney disease: A systematic review

Rationale & Objective: Trials in autosomal dominant polycystic kidney disease (ADPKD) have increased, but their impact on decision making has been limited. Because heterogeneity in reported outcomes may be responsible, we assessed their range and variability in ADPKD trials. Study Design: Systematic review. Setting & Study Population: Adult participants in clinical trials in ADPKD. Selection Criteria for Studies: We included trials that studied adults and were published in English. For trials that enrolled patients without ADPKD, only those enrolling ≥50% of participants with ADPKD were included. Data Extraction: We extracted information on all discrete outcome measures, grouped them into 97 domains, and classified them into clinical, surrogate, and patient-reported categories. For each category, we choose the 3 most frequently reported domains and performed a detailed analysis of outcome measures. Analytical Approach: Frequencies and characteristics of outcome measures were described. Results: Among 68 trials, 1,413 different outcome measures were reported. 97 domains were identified; 41 (42%) were surrogate, 30 (31%) were clinical, and 26 (27%) were patient reported. The 3 most frequently reported domains were in the surrogate category: kidney function (54; 79% of trials; using 46 measures), kidney and cyst volumes (43; 63% of trials; 52 measures), and blood pressure (27; 40% of trials, 30 measures); in the clinical category: infection (10; 15%; 21 measures), cardiovascular events (9; 13%; 6 measures), and kidney failure requiring kidney replacement therapy (8; 12%; 5 measures); and in the patient-reported category: pain related to ADPKD (16; 24%; 26 measures), pain for other reasons (11; 16%; 11 measures), and diarrhea/constipation/gas (10; 15%; 9 measures). Limitations: Outcome measures were assessed for only the top 3 domains in each category. Conclusions: The outcomes in ADPKD trials are broad in scope and highly variable. Surrogate outcomes were most frequently reported. Patient-reported outcomes were uncommon. A consensus-based set of core outcomes meaningful to patients and clinicians is needed for future ADPKD trials

Establishing a core outcome set for peritoneal dialysis : report of the SONG-PD (standardized outcomes in nephrology-peritoneal dialysis) consensus workshop

Outcomes reported in randomized controlled trials in peritoneal dialysis (PD) are diverse, are measured inconsistently, and may not be important to patients, families, and clinicians. The Standardized Outcomes in Nephrology-Peritoneal Dialysis (SONG-PD) initiative aims to establish a core outcome set for trials in PD based on the shared priorities of all stakeholders. We convened an international SONG-PD stakeholder consensus workshop in May 2018 in Vancouver, Canada. Nineteen patients/caregivers and 51 health professionals attended. Participants discussed core outcome domains and implementation in trials in PD. Four themes relating to the formation of core outcome domains were identified: life participation as a main goal of PD, impact of fatigue, empowerment for preparation and planning, and separation of contributing factors from core factors. Considerations for implementation were identified: standardizing patient-reported outcomes, requiring a validated and feasible measure, simplicity of binary outcomes, responsiveness to interventions, and using positive terminology. All stakeholders supported inclusion of PD-related infection, cardiovascular disease, mortality, technique survival, and life participation as the core outcome domains for PD

Core outcome domains for trials in autosomal dominant polycystic kidney disease: An international Delphi survey

Rationale & Objective Outcomes reported in trials involving patients with autosomal dominant polycystic kidney disease (ADPKD) are heterogeneous and rarely include patient-reported outcomes. We aimed to identify critically important consensus-based core outcome domains to be reported in trials in ADPKD. Study Design An international 2-round online Delphi survey was conducted in English, French, and Korean languages. Setting & Participants Patients/caregivers and health professionals completed a 9-point Likert scale (7-9 indicating critical importance) and a Best-Worst Scale. Analytical Approach The absolute and relative importance of outcomes were assessed. Comments were analyzed thematically. Results 1,014 participants (603 [60%] patients/caregivers, 411 [40%] health professionals) from 56 countries completed round 1, and 713 (70%) completed round 2. The prioritized outcomes were kidney function (importance score, 8.6), end-stage kidney disease (8.6), death (7.9), blood pressure (7.9), kidney cyst size/growth (7.8), and cerebral aneurysm (7.7). Kidney cyst–related pain was the highest rated patient-reported outcome by both stakeholder groups. Seven themes explained the prioritization of outcomes: protecting life and health, directly encountering life-threatening and debilitating consequences, specificity to ADPKD, optimizing and extending quality of life, hidden suffering, destroying self-confidence, and lost opportunities. Limitations Study design precluded involvement from those without access to internet or limited computer literacy. Conclusions Kidney function, end-stage kidney disease, and death were the most important outcomes to patients, caregivers, and health professionals. Kidney cyst–related pain was the highest rated patient-reported outcome. Consistent reporting of these top prioritized outcomes may strengthen the value of trials in ADPKD for decision making

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

Strong Heterogeneity of Outcome Reporting in Systematic Reviews

International audienceOBJECTIVES: The Core Outcome Measures in Effectiveness Trial initiative aims at developing core outcome set (COS) for research. Consensus on a COS for renoprotection research is lacking. We performed a systematic review (SR) of SRs of renoprotection to identify outcomes used in renoprotection research and to assess how frequently these outcomes could be meta-analyzed in the SRs. STUDY DESIGN AND SETTING: We searched for SRs with meta-analyses of renoprotection treatments in the Database of Abstracts of Reviews of Effects and in MEDLINE and collected outcomes that were meta-analyzed. For each outcome and SR, we assessed the proportion of trials/patients that could be meta-analyzed. RESULTS: We retrieved 66 SRs. A total of 609 outcomes were extracted for 20 distinct renoprotection outcomes. The median (interquartile range) proportion of SRs in which these outcomes had been meta-analyzed was 8% (2%, 27%) (range 2-50%). The proportion of trials that had been aggregated was >75% for only 36% of the outcomes. CONCLUSION: The outcomes meta-analyzed in renoprotection trials are heterogeneous, with a low proportion of trials or patients pooled for each outcome and each SR. A COS is needed in renoprotection research to limit this waste of research. The outcomes identified in the present work could be a basis for this COS

Le recueil en routine de données de santé perçue à l’ère du paiement à la qualité : préconisations de la Commission épidémiologie et santé publique de la SFNDT

International audienceIn France, the method of financing is mainly based on the quantity of care produced. The fixed-rate financing of patients with chronic kidney disease at stage IV or V introduces the notion of payment to quality. Part of the quality assessment will focus on the patients' feelings about their care. The objective of this paper is to assess these indicators used in nephrology, markers in their own right of the quality of care. The patients reported outcomes measures considering the impact of illness or care and the Patient Reported Experience Measures considering their perception of their experience with the health care system or care pathway, are broader than quality of life. These PROs are measured using standardized and validated questionnaires, generic or specific. The Standardised Outcomes in Nephrology initiative has shown that PROs, too often neglected in favor of biological criteria, are instead favored by patients. In the context of a broad deployment of monitoring the quality of life for the purpose of evaluation of care, outside research protocol, the Commission recommends one of the following 2 tools: EuroQol 5D and 12-Item Short Form Health Survey, a compromise between feasibility and relevance and e-SATIS given its great use in health facilities, with an annual follow-up

The Scope and Consistency of Outcomes Reported in Trials in Patients with Systemic Sclerosis

International audienceOBJECTIVE: The core outcome set for trials in systemic sclerosis (SSc) was developed in 2008 and comprises eleven domains and thirty-one measures, leading to the development of a composite index (CRISS). We aimed to assess the scope, and consistency of outcomes reported in trials of SSc, and the uptake of this core set and CRISS. METHODS: MEDLINE, Cochrane CENTRAL, Embase and clinicaltrials gov were searched to identify randomized trials published from 2000 to 29 April 2018 in adults with limited or diffuse SSc. Outcomes and measures were recorded for each trial, classified into domains and the frequency of outcomes before after publication of the publication of the core set calculated. RESULTS: From 152 trials, 4193 outcomes were classified into 84 domains. The three most common domains were health-related quality of life and function (59%, 130 measures), skin (47%, 59 measures) and pulmonary (45%, 168 measures). After the publication of the core outcome set, no trial reported the complete core set with adherence to each of the eleven domains ranging from 6.1% to 54.4% and adherence to each of the thirty-one measures ranging from 0 to 48.1%. The five measures required for CRISS were reported completely in 11% of trials. CONCLUSION: Despite recognition that uniform acquisition and reporting of outcomes would enable a better evaluation of proposed SSc therapeutics, the outcome domains and measures reported in randomized trials in systemic sclerosis remain very inconsistent, with little impact of the core outcome set. This article is protected by copyright. All rights reserved