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

https://doi.org/10.1053/j.ajkd.2016.11.029Get rights and content

Background

Survival and quality of life for patients on hemodialysis therapy remain poor despite substantial research efforts. Existing trials often report surrogate outcomes that may not be relevant to patients and clinicians. The aim of this project was to generate a consensus-based prioritized list of core outcomes for trials in hemodialysis.

Study Design

In a Delphi survey, participants rated the importance of outcomes using a 9-point Likert scale in round 1 and then re-rated outcomes in rounds 2 and 3 after reviewing other respondents’ scores. For each outcome, the median, mean, and proportion rating as 7 to 9 (critically important) were calculated.

Setting & Participants

1,181 participants (202 [17%] patients/caregivers, 979 health professionals) from 73 countries completed round 1, with 838 (71%) completing round 3.

Outcomes & Measurements

Outcomes included in the potential core outcome set met the following criteria for both patients/caregivers and health professionals: median score ≥ 8, mean score ≥ 7.5, proportion rating the outcome as critically important ≥ 75%, and median score in the forced ranking question < 10.

Results

Patients/caregivers rated 4 outcomes higher than health professionals: ability to travel, dialysis-free time, dialysis adequacy, and washed out after dialysis (mean differences of 0.9, 0.5, 0.3, and 0.2, respectively). Health professionals gave a higher rating for mortality, hospitalization, decrease in blood pressure, vascular access complications, depression, cardiovascular disease, target weight, infection, and potassium (mean differences of 1.0, 1.0, 1.0, 0.9, 0.9, 0.8, 0.7, 0.4, and 0.4, respectively).

Limitations

The Delphi survey was conducted online in English and excludes participants without access to a computer and internet connection.

Conclusions

Patients/caregivers gave higher priority to lifestyle-related outcomes than health professionals. The prioritized outcomes for both groups were vascular access problems, dialysis adequacy, fatigue, cardiovascular disease, and mortality. This process will inform a core outcome set that in turn will improve the relevance, efficiency, and comparability of trial evidence to facilitate treatment decisions.

Section snippets

Study Design

The Delphi method is a technique for achieving consensus among a panel of experts. This process involves sequential surveys, typically conducted over 3 rounds and answered anonymously, and gives equal influence to all who participate. It was first developed by the RAND Corporation in the 1950s25 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, 27, 28 The SONG-HD Delphi process is

Participant Characteristics

In total, 1,181 people from 73 countries participated in round 1 of the Delphi survey, including 202 (17%) patients/caregivers and 979 (83%) health professionals. Round 2 included 165 (17%) patients/caregivers and 784 (83%) health professionals from 63 countries. In the third and final round, 150 (18%) patients/caregivers and 688 (82%) health professionals participated. The full survey completion rate was 71%. Participant characteristics are provided in Tables 1 and 2.

In round 3, of the 150

Discussion

The highest priority outcomes shared among patients/caregivers and health professionals were vascular access problems, dialysis adequacy, fatigue, CVD, and mortality. Overall, most of these reflect common and high-impact outcomes in the context of HD, which have remained as major challenges in providing care for patients on HD therapy. Frequently reported biochemical outcomes in HD trials, such as phosphate, calcium, and parathyroid hormone levels, were consistently rated to be of lower

Acknowledgements

We thank the following organizations for their support: International: Cochrane Kidney and Transplant, International Society of Nephrology, KDIGO (Kidney Disease: Improving Global Outcomes), PKD International; Australia/New Zealand: Australian and New Zealand Society of Nephrology, Australian Kidney Trials Network, Christchurch Kidney Society, Kidney Health Australia, Kidney Health Australia–Caring for Australasians with Renal Impairment Guidelines, Kidney Health New Zealand, Renal Society of

References (39)

  • C.M. Bell et al.

    An off-the-shelf help list: a comprehensive catalog of preference scores from published cost-utility analyses

    Med Decis Making

    (2001)
  • A. Nissenson

    Improving outcomes for ESRD patients: shifting the quality paradigm

    Clin J Am Soc Nephrol

    (2014)
  • G. Wong et al.

    How do people with chronic kidney disease value cancer-related quality of life?

    Nephrology

    (2012)
  • M. Wyld et al.

    A systematic review and meta-analysis of utility-based quality of life in chronic kidney disease treatments

    PLoS Med

    (2012)
  • E. Gargon et al.

    Choosing important health outcomes for comparative effectiveness research: a systematic review

    PLoS One

    (2014)
  • G. Ghandi

    Patient-important outcomes in registered diabetes trials

    JAMA

    (2008)
  • S. Svensson et al.

    Surrogate outcomes in clinical trials: a cautionary tale

    JAMA Intern Med

    (2013)
  • J. Yudkin et al.

    The idolatory of the surrogate

    BMJ

    (2011)
  • J.M. Halimi et al.

    Renal endpoints in renal and cardiovascular randomized clinical trials: time for a consensus?

    Fundam Clin Pharmacol

    (2012)
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    Because an author of this article was an editor for AJKD at the time the manuscript was submitted, the peer-review and decision-making processes were handled entirely by an Associate Editor (Amit X. Garg, MD, MA, FRCPC, PhD) who served as Acting Editor-in-Chief. Details of the journal’s procedures for potential editor conflicts are given in the Information for Authors & Journal Policies.

    A list of the SONG-HD initiative investigators is available at www.songinitiative.org.

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