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Journal ArticleDOI

Developing a Set of Core Outcomes for Trials in Hemodialysis: An International Delphi Survey.

TL;DR: A consensus-based prioritized list of core outcomes for trials in hemodialysis will improve the relevance, efficiency, and comparability of trial evidence to facilitate treatment decisions and patients/caregivers gave higher priority to lifestyle-related outcomes than health professionals.
About: This article is published in American Journal of Kidney Diseases.The article was published on 2017-10-01 and is currently open access. It has received 187 citations till now. The article focuses on the topics: Health care & Family caregivers.

Summary (3 min read)

INTRODUCTION

  • The enormous investment in biomedical research, particularly in randomized trials, may not have led to the improvements in health that were hoped for.
  • It has been estimated that 85% of the worldwide US $240 billion invested in research annually is wasted.
  • In nephrology, there has been substantial research investment into hemodialysis (HD), yet survival rates have not improved correspondingly over the past 40 years and quality of life remains poor even compared with patients with many cancers.
  • This may be partly attributable to what outcomes are selected and reported in trials – a challenge well-recognized across medical specialties.
  • 9,11,12 In HD, biochemical markers such as serum phosphorus, calcium, and parathyroid hormone, are commonly reported but are not strongly and consistently associated with mortality, cardiovascular disease (CVD), or quality of life.

Study design

  • The Delphi method is a technique for achieving consensus among a panel of experts.
  • This process involves sequential surveys, typically conducted over three rounds, answered anonymously and gives equal influence to all who participate.
  • It was first developed by the RAND Corporation in the 1950’s 25 and has since been increasingly used as a valid approach to develop consensus-based core outcomes for clinical trials in various medical specialty areas.

Participant selection and recruitment

  • Stakeholders including patients, caregivers/family members, nephrologists, surgeons, nurses, social workers, psychologists, dieticians, pharmacists, policy makers, researchers and industry, with experience or interest in HD were invited to join the Delphi Panel.
  • Participants worldwide were eligible if they were aged over 18 years and able to complete an online survey in English-language.
  • Using an opt-in, snowballing sampling frame, the authors recruited patients/caregivers through participating hospitals, patient/consumer organizations, and social media listed in Supplementary File S1.
  • Participants registered their email on www.songinitiative.org prior to the survey launch.

Data collection

  • The 34 outcome domains for the three-round Delphi survey were identified from a systematic review of outcomes reported in trials in HD, stakeholder interviews, and nominal group technique conducted with patients on HD and caregivers.
  • The Delphi survey was completed online via LimeSurvey between September and November, 2015.
  • Outcomes with a mean and median of less than 7 for patients/ caregivers and health professionals were not included in round 2.
  • Participants reviewed the group scores and their own score for each outcome and re-rated the 29 outcomes using the same 9-point Likert scale, also known as Round 2.
  • For each outcome, an optional comments box allowed participants to explain reasons for their rating.

Data analysis

  • The authors used SPSS (IBM; Version 22.0) to calculate descriptive statistics.
  • The authors calculated the median, mean, and proportion of participants (rating 7-9) for each outcome.
  • The scores were calculated separately for patients/caregivers and health professionals, with the difference in means considered significant at P<0.05 based on the t-test.
  • Any analysis of the total sample was weighted equally between patients/caregivers and health professionals.
  • Consensus was defined a priori based on the OMERACT definition using proportion scores.

Definition of consensus

  • Outcomes from round 1 with a mean and median score greater than or equal to 7 for patients/caregivers and health professionals were included in round 2.
  • This was validated against the proportion of critically important scores (rated 7-9) for each outcome to ensure that important outcomes were not excluded.
  • Outcomes from round 2 with a mean and median score greater than 7 for patients/caregivers and health professionals were included in round 3.
  • This was validated against the proportion of critically important scores (rated 7-9) for each outcome.

Delphi scores

  • The means, medians, and proportion of participants rating the outcome 7 – 9 (critical importance) for each of the 34 outcomes are shown in Supplementary Table S1, also known as Round 1.
  • The following outcomes were excluded from round 2 as they had a mean or median score of less than 7 (not of critical importance) among both patient/caregiver and health professional groups in round 1: nausea/vomiting, sexual function, restless legs syndrome, itching and cramps.
  • The differences in ratings between stakeholder groups are shown in Figure 3.

DISCUSSION

  • The highest-priority outcomes shared among patients/caregivers and health professionals were vascular access problems, dialysis adequacy, fatigue, CVD, and mortality.
  • The SONG-HD Delphi survey had a large sample size (five times greater than most similar Delphi surveys to establish core outcomes)23,38,39 and achieved a broad engagement of a range of stakeholders including patients/caregivers, multidisciplinary healthcare providers, policy makers and industry, across 73 countries, and retained a high response rate of 71% by round 3.
  • The process provided a transparent and systematic way to gain consensus on the importance of outcomes to consider for a core outcome set in HD.
  • The authors recognize that these outcomes may not be shared by those who did not participate in the study, or by individuals or groups within the study.
  • To effectively implement a core outcome set in chronic kidney disease, patients/caregivers and health professional participants suggested that outcome measures should be feasible and valid, and take patient priorities into account.

Contributions

  • Research idea and study design: all authors; Data acquisition: NE, AT, BM, BH, DCW, PT, SC, TH, WvB, WCW, HT, SY, SM, DCH, JCC; Data analysis/interpretation: all authors.
  • Each author contributed important intellectual content during manuscript drafting or revision and accepts accountability for the overall work by ensuring that questions pertaining to the accuracy or integrity of any portion of the work are appropriately investigated and resolved.
  • NE takes responsibility that this study has been reported honestly, accurately, and transparently; that no important aspects of the study have been omitted; and that any discrepancies from the study as planned (and, if relevant, registered) have been explained.

Financial Disclosure

  • David C Harris received fees from Roche Myanmar to give a lecture in Myanmar.
  • David W Johnson has received consultancy fees, research grants, travel sponsorships and speaker’s honoraria from Baxter Healthcare and Fresenius Medical Care.
  • Sreedhar Mandayam has received research funding from Keryx and is on the Advisory Board/consulting for Corp Medix.
  • He has received lecture fees from Fresenius, Amgen, Janssen, ZS Pharma and Vifor Fresenius.
  • All other authors have no financial interests to disclose.

TABLES AND FIGURES

  • Definitions of high rating outcome domains Table 3.
  • Mean scores for round 2 and 3 are not available for outcomes that were excluded in those rounds.
  • Figure 3. Difference in mean scores between patients/caregivers and health professionals (rounds 1-3) Note: Ordered by difference in mean scores between patients/caregivers and health professionals.

Outcome domain Definition

  • Vascular access problems Problems with fistula, graft, or catheter required for dialysis e.g. access infections, bleeding, bruising, pain, discomfort, clotting Death/Mortality Number of people on hemodialysis who have died, risk of death, how long the patient will live Cardiovascular disease Disease of the heart and blood vessels e.g. heart attack, stroke, blockage of blood vessels Dialysis adequacy.
  • How well the dialysis cleans the blood, clearance, Kt/V Fatigue/Energy Feeling tired with no energy for weeks, for most of the time Ability to travel Inclusion criteria for the core outcome set based on median, mean, proportion (7-9) and median rank scores Median score ≥.

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Citations
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Journal ArticleDOI
TL;DR: The need to move away from a "one-size-fits-all" approach to dialysis and provide more individualized care that incorporates patient goals and preferences while still maintaining best practices for quality and safety is represented during the KDIGO conference.

213 citations


Cites background from "Developing a Set of Core Outcomes f..."

  • ...Patients and clinicians can have divergent and sometimes conflicting goals for hemodialysis treatment,with clinicians focused onoutcomes such as mortality and biochemical markers and patients prioritizing their well-being and lifestyle.(9) For example, some patients on homehemodialysis have reported awillingness to trademonths of survival for ability to travel....

    [...]

Journal ArticleDOI
TL;DR: The current landscape of dialysis therapy is described from an epidemiological, economic, ethical and patient-centred framework, and initiatives that are aimed at stimulating innovations in the field to one that supports high-quality, high-value care are described.
Abstract: The development of dialysis by early pioneers such as Willem Kolff and Belding Scribner set in motion several dramatic changes in the epidemiology, economics and ethical frameworks for the treatment of kidney failure. However, despite a rapid expansion in the provision of dialysis — particularly haemodialysis and most notably in high-income countries (HICs) — the rate of true patient-centred innovation has slowed. Current trends are particularly concerning from a global perspective: current costs are not sustainable, even for HICs, and globally, most people who develop kidney failure forego treatment, resulting in millions of deaths every year. Thus, there is an urgent need to develop new approaches and dialysis modalities that are cost-effective, accessible and offer improved patient outcomes. Nephrology researchers are increasingly engaging with patients to determine their priorities for meaningful outcomes that should be used to measure progress. The overarching message from this engagement is that while patients value longevity, reducing symptom burden and achieving maximal functional and social rehabilitation are prioritized more highly. In response, patients, payors, regulators and health-care systems are increasingly demanding improved value, which can only come about through true patient-centred innovation that supports high-quality, high-value care. Substantial efforts are now underway to support requisite transformative changes. These efforts need to be catalysed, promoted and fostered through international collaboration and harmonization. Dialysis is a life-saving therapy; however, costs of dialysis are high, access is inequitable and outcomes are inadequate. This Review describes the current landscape of dialysis therapy from an epidemiological, economic, ethical and patient-centred framework, and describes initiatives that are aimed at stimulating innovations in the field to one that supports high-quality, high-value care.

212 citations

Journal ArticleDOI
TL;DR: The overarching theme resulting from presentations and discussions was that managing BP and volume in dialysis involves weighing multiple clinical factors and risk considerations as well as patient lifestyle and preferences, all within a narrow therapeutic window for avoiding acute or chronic volume-related complications.

113 citations

Journal ArticleDOI
TL;DR: This report outlines implementation strategies and pathways to be established through partnership with stakeholders, which may bolster acceptance and reporting of core outcomes in trials, and encourage their use by end-users such as guideline producers and policymakers to help improve patient-important outcomes.

107 citations

Journal ArticleDOI
TL;DR: Low-dose aspirin does not prevent thrombovascular accidents in low-risk haemodialysis patients during treatment with recombinant human erythropoietin.
Abstract: . Int J Clin Pract 2010; 64: 1239–1244 94. Kaufman JS, Connor TZ, Zhang JH et al. Randomized controlled trial of clopidogrel plus aspirin to prevent hemodialysis access graft thrombosis. J Am Soc Nephrol 2003; 14: 2313–2321 95. Kooistra MP, van Es A, Marx JJ et al. Low-dose aspirin does not prevent thrombovascular accidents in low-risk haemodialysis patients during treatment with recombinant human erythropoietin. Nephrol Dial Transplant

95 citations


Cites background from "Developing a Set of Core Outcomes f..."

  • ...For patients, the ‘umbilical cord’ keeping them alive can be a constant source of stressful experiences [1]....

    [...]

References
More filters
Journal ArticleDOI
TL;DR: The DELPHI method was devised in order to obtain the most reliable opinion consensus of a group of experts by subjecting them to a series of questionnaires in depth interspersed with controlled opinion feedback as mentioned in this paper.
Abstract: This paper gives an account of an experiment in the use of the so-called DELPHI method, which was devised in order to obtain the most reliable opinion consensus of a group of experts by subjecting them to a series of questionnaires in depth interspersed with controlled opinion feedback.

4,856 citations

Journal ArticleDOI
TL;DR: “Research results should be easily accessible to people who need to make decisions about their own health...

1,535 citations

Journal ArticleDOI
TL;DR: This report discusses how avoidable waste can be considered when research priorities are set and recommends ways to improve the yield from basic research, and the transparency of processes by which funders prioritise important uncertainties should be increased.

1,069 citations


"Developing a Set of Core Outcomes f..." refers background in this paper

  • ...This may be partly attributable to what outcomes are selected and reported in trials – a challenge well-recognized across medical specialties.(1,8-10)...

    [...]

Journal ArticleDOI
TL;DR: In an unadjusted intention-to-treat analysis, cinacalcet did not significantly reduce the risk of death or major cardiovascular events in patients with moderate- to-severe secondary hyperparathyroidism who were undergoing dialysis.
Abstract: In an unadjusted intention-to-treat analysis, cinacalcet did not significantly reduce the risk of death or major cardiovascular events in patients with moderate-to-severe secondary hyperparathyroidism who were undergoing dialysis. (Funded by Amgen; EVOLVE ClinicalTrials.gov number, NCT00345839.).

796 citations

Journal ArticleDOI
TL;DR: The OMERACT Filter 2.0 explicitly describes a comprehensive conceptual framework and a recommended process to develop core outcome measurement sets for rheumatology likely to be useful as a template in other areas of health care.

646 citations


"Developing a Set of Core Outcomes f..." refers background or methods in this paper

  • ...It was first developed by the RAND Corporation in the 1950’s 25 and has since been increasingly used as a valid approach to develop consensus-based core outcomes for clinical trials in various medical specialty areas.(10,22,26-28) The SONG-HD Delphi process is shown in Supplementary Figure S1....

    [...]

  • ...Initiatives to develop core outcomes are seen in rheumatology and oncology, and have demonstrated improvements in consistent reporting of relevant outcomes.(10,23,24) As part of the international Standardized Outcomes in NephrologyHemodialysis (SONG-HD) initiative, this study aimed to generate a consensus-based prioritized list...

    [...]

  • ...This has been done successfully by the Outcome Measures in Rheumatology (OMERACT) initiative, which has improved the reporting and relevance of outcomes in rheumatology trials.(10,24) Recent analyses have shown that a higher percentage of trials are now incorporating the OMERACT core outcome set in rheumatology trials....

    [...]

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