Development of a core outcome set for the evaluation of interventions to enhance trial participation decisions on behalf of adults who lack capacity to consent: a mixed methods study (COnSiDER Study)

Abstract BackgroundTrials involving adults who lack capacity to provide consent for themselves rely on proxy or surrogate decision-makers, usually a family member, to make decisions about participation. Following decades of innovations to improve informed consent in trials, the first interventions to enhance proxy decisions about trial participation are now being developed. However, a lack of standardised outcome measurement in the evaluation of these novel interventions will impede comparisons between their effectiveness. The aim of this study was to establish an agreed standardised core outcome set (COS) for use when evaluating interventions to improve proxy decisions about trial participation on behalf of adults who lack capacity to consent.MethodsWe used established methods to develop the COS including a consensus study with key stakeholder groups comprising those who will use the COS in research (researchers and healthcare professionals) and patients or their representatives. Following a scoping review to identify candidate items, we used a modified two-round Delphi survey to achieve consensus on core outcomes, with equivocal items taken to a consensus meeting for discussion. The COS was finalised following an online consensus meeting in October 2020.ResultsA total of 28 UK stakeholders (5 researchers, 10 trialists, 3 patient/family representatives, 7 recruiters and 3 advisors/approvers) participated in the online Delphi survey to rank candidate items. Items were broadly grouped into three categories: how family members make decisions, their experiences of making decisions, and the personal aspects that influence the decision. Following the Delphi survey, 27 items were included and ten items exhibited no consensus which required discussion at the consensus meeting. Sixteen participants attended the meeting, including additional patient/family representatives invited to increase representation from this key group. We reached consensus for the inclusion of 28 outcome items, including one selected at the consensus meeting.ConclusionsThe study identified outcomes that should be measured as a minimum in all evaluations of interventions to enhance proxy decisions about trials. Further work is required to identify appropriate measures and timing of outcome measurement. Enhancing the quality of proxy decisions will help improve trial participation decisions for these vulnerable groups. Trial registration: The study is registered on the COMET database (https://www.comet-initiative.org/Studies/Details/1409)

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Development of a core outcome set for the evaluation of interventions to enhance trial participation decisions on behalf of adults who lack capacity to consent: a mixed methods study (COnSiDER Study)

Trials ◽

10.1186/s13063-021-05883-5 ◽

2021 ◽

Vol 22 (1) ◽

Author(s):

V. Shepherd ◽

F. Wood ◽

M. Robling ◽

E. Randell ◽

K. Hood

Keyword(s):

Decision Making ◽

Outcome Measures ◽

Scoping Review ◽

Core Outcome Set ◽

Consensus Meeting ◽

Delphi Survey ◽

Additional Patient ◽

Trial Participation ◽

Core Outcome ◽

Outcome Set

Abstract Background Trials involving adults who lack capacity to provide consent rely on proxy or surrogate decision-makers, usually a family member, to make decisions about participation. Interventions to enhance proxy decisions about trial participation are now being developed. However, a lack of standardised outcome measures limits evaluation of these interventions. The aim of this study was to establish an agreed standardised core outcome set (COS) for use when evaluating interventions to improve proxy decisions about trial participation. Methods We used established methods to develop the COS including a consensus study with key stakeholder groups comprising those who will use the COS in research (researchers and healthcare professionals) and patients or their representatives. Following a scoping review to identify candidate items, we used a modified two-round Delphi survey to achieve consensus on core outcomes, with equivocal items taken to a consensus meeting for discussion. The COS was finalised following an online consensus meeting in October 2020. Results A total of 28 UK stakeholders (5 researchers, 10 trialists, 3 patient/family representatives, 7 recruiters and 3 advisors/approvers) participated in the online Delphi survey to rank candidate items from the scoping review (n = 36) and additional items proposed by participants (n = 1). Items were broadly grouped into three categories: how family members make decisions, their experiences of making decisions, and the personal aspects that influence the decision. Following the Delphi survey, 27 items were included and ten items exhibited no consensus which required discussion at the consensus meeting. Sixteen participants attended the meeting, including additional patient/family representatives invited to increase representation from this key group (n = 2). We reached consensus for the inclusion of 28 outcome items, including one selected at the consensus meeting. Conclusions The study identified outcomes that should be measured as a minimum in all evaluations of interventions to enhance proxy decisions about trials. These relate to the process of decision-making, proxies’ experience of decision-making, and factors that influence decision-making such as understanding. Further work with people with impairing conditions and their families is needed to explore their views about the COS and to identify appropriate outcome measures and timing of measurement. Trial registration The study is registered on the COMET database (https://www.comet-initiative.org/Studies/Details/1409)

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The outcomes of Perthes’ disease

The Bone & Joint Journal ◽

10.1302/0301-620x.102b5.bjj-2020-0072 ◽

2020 ◽

Vol 102-B (5) ◽

pp. 611-617

Author(s):

Donato G. Leo ◽

Helen Jones ◽

Rebecca Murphy ◽

Justin Wei Leong ◽

Tina Gambling ◽

...

Keyword(s):

Systematic Review ◽

Clinical Research ◽

Qualitative Interviews ◽

Core Outcome Set ◽

Consensus Meeting ◽

Delphi Survey ◽

Perthes Disease ◽

Core Outcome ◽

Outcome Set ◽

Life Impact

Aims To identify a suite of the key physical, emotional, and social outcomes to be employed in clinical practice and research concerning Perthes' disease in children. Methods The study follows the guidelines of the COMET-Initiative (Core Outcome Measures in Effectiveness Trials). A systematic review of the literature was performed to identify a list of outcomes reported in previous studies, which was supplemented by a qualitative study exploring the experiences of families affected by Perthes’ disease. Collectively, these outcomes formed the basis of a Delphi survey (two rounds), where 18 patients with Perthes’ disease, 46 parents, and 36 orthopaedic surgeons rated each outcome for importance. The International Perthes Study Group (IPSG) (Dallas, Texas, USA (October 2018)) discussed outcomes that failed to reach any consensus (either ‘in’ or ‘out’) before a final consensus meeting with representatives of surgeons, patients, and parents. Results In total, 23 different outcome domains were identified from the systematic review, and a further ten from qualitative interviews. After round one of the Delphi survey, participants suggested five further outcome domains. A total of 38 outcomes were scored in round two of the Delphi. Among these, 16 outcomes were scored over the prespecified 70% threshold for importance (divided into six main categories: adverse events; life impact; resource use; pathophysiological manifestations; death; and technical considerations). Following the final consensus meeting, 14 outcomes were included in the final Core Outcome Set (COS). Conclusion Core Outcome Sets (COSs) are important to improve standardization of outcomes in clinical research and to aid communication between patients, clinicians, and funding bodies. The results of this study should be a catalyst to develop high-quality clinical research in order to determine the optimal treatments for children with Perthes’ disease. Cite this article: Bone Joint J 2020;102-B(5):611–617.

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Development of a core outcome set for clinical trials aimed at improving antimicrobial stewardship in care homes

Antimicrobial Resistance and Infection Control ◽

10.1186/s13756-021-00925-8 ◽

2021 ◽

Vol 10 (1) ◽

Author(s):

Hoa Q. Nguyen ◽

Declan T. Bradley ◽

Michael M. Tunney ◽

Carmel M. Hughes

Keyword(s):

Clinical Trials ◽

Antimicrobial Stewardship ◽

Outcome Measurement ◽

Evidence Synthesis ◽

Core Outcome Set ◽

Delphi Survey ◽

Care Homes ◽

Core Outcome ◽

Outcome Set ◽

Days Of Therapy

Abstract Background Diverse outcomes reported in clinical trials of antimicrobial stewardship (AMS) interventions in care homes have hindered evidence synthesis. Our main objective was to develop a core outcome set (COS) for use in trials aimed at improving AMS in care homes. Methods A refined inventory of outcomes for AMS interventions in care homes, compiled from a previous study, was rated in a three-round international Delphi survey with 82 participants, using a nine-point Likert scale (from 1, unimportant, to 9, critical). This was followed by an online consensus exercise with 12 participants from Northern Ireland to finalise the COS content. Subsequently, a suitable outcome measurement instrument (OMI) was selected for each outcome in the COS by: identifying existing OMIs through a literature search and experts’ suggestions, assessing the quality of OMIs, and selecting one OMI for each core outcome via a two-round international Delphi survey with 59 participants. Results Of 14 outcomes initially presented, consensus was reached for inclusion of five outcomes in the COS after the three-round Delphi survey and the online consensus exercise, comprising the total number of antimicrobial courses prescribed, appropriateness of antimicrobial prescribing, days of therapy per 1000 resident-days, rate of antimicrobial resistance, and mortality related to infection. Of 17 potential OMIs identified, three were selected for the two-round Delphi exercise after the quality assessment. Consensus was reached for selection of two OMIs for the COS. Conclusion This COS is recommended to be used in clinical trials aimed at improving AMS in care homes.

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Developing a core outcome set for children with protracted bacterial bronchitis

ERJ Open Research ◽

10.1183/23120541.00344-2019 ◽

2020 ◽

Vol 6 (1) ◽

pp. 00344-2019

Author(s):

Francis J. Gilchrist ◽

Imran Ali ◽

Malcolm Brodlie ◽

Will D. Carroll ◽

Bridget Kemball ◽

...

Keyword(s):

Clinical Trials ◽

Core Outcome Set ◽

Consensus Meeting ◽

Delphi Survey ◽

Respiratory Pathogen ◽

Core Outcome ◽

Potential Core ◽

Outcome Set ◽

Protracted Bacterial Bronchitis ◽

Wet Cough

BackgroundProtracted bacterial bronchitis (PBB) is a chronic endobrochial infection and a leading cause of chronic wet cough in children. There is an urgent need for a randomised controlled trial to investigate the optimal treatment but there is no core outcome set (COS) to inform choice of outcomes. A COS is a standardised set of outcomes representing the minimum that should be measured and reported in clinical trials of a specific condition. We have developed a COS for PBB.MethodsPotential core outcomes were collated from a systematic review, interviews with parents and a clinician survey. A two-round Delphi survey of healthcare professionals identified which outcomes had consensus for inclusion. The final COS was agreed at a consensus meeting of parent representatives and clinicians.Results20 outcomes were identified for the Delphi survey. After two rounds, 10 reached consensus. These were combined and edited at the consensus meeting into the final six: 1) Resolution of cough assessed using a cough score/diary recorded daily by parent(s) during treatment; 2) relapse of chronic wet cough and/or cumulative antibiotic treatment during ≥12 months follow-up; 3) change in child's quality of life (parent-proxy reporting for young children); 4) emergence of antibiotic resistance; 5) development of bronchiectasis diagnosed on clinically indicated computed tomography scans; and 6) microbiological clearance of identified respiratory pathogen if samples readily available.ConclusionsWe have developed a COS for PBB which will reduce the outcome heterogeneity and bias of future clinical trials, as well as promoting comparison between studies.

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A Core Outcome Set to Evaluate Physical Activity Interventions for People Living With Dementia

The Gerontologist ◽

10.1093/geront/gnz100 ◽

2019 ◽

Cited By ~ 3

Author(s):

Ana-Carolina Gonçalves ◽

Dinesh Samuel ◽

Mary Ramsay ◽

Sara Demain ◽

Alda Marques

Keyword(s):

Physical Activity ◽

Core Outcome Set ◽

Consensus Meeting ◽

Delphi Survey ◽

Core Outcome ◽

Card Sorting ◽

Modified Delphi ◽

Outcome Set ◽

Group 2 ◽

The Moment

Abstract Background and Objectives A core outcome set (COS) to evaluate physical activity (PA) interventions for people living with dementia is needed, as the development of guidelines is currently limited by important heterogeneity in this field of research. Development of COS often includes Delphi surveys, but people living with dementia are often excluded. This study aimed to reach consensus on this COS using a modified Delphi survey to enable the participation of people living with dementia. Research Design and Methods Two stakeholders groups took part in a Delphi survey (Group 1: people living with dementia and family caregivers; Group 2: professionals from different backgrounds, including physiotherapists, occupational therapists, and researchers). Caregivers and professionals completed the survey remotely. Participants living with dementia took part face-to-face, using a card sorting strategy. The consensus process was finalized with a consensus meeting. Results Ninety-five participants of both groups completed the modified Delphi. Of those, 11 attended the consensus meeting. The card sorting strategy was successful at including people living with dementia. Seven outcomes reached consensus: preventing falls; doing what you can do; staying healthy and fit; walking better, being able to stand up and climb stairs; feeling brighter; enjoying the moment; and, feeling useful and having a purpose. Discussion and Implications Robust and innovative methodological strategies were used to reach a consensus on a COS (what to measure) to evaluate PA for people living with dementia. Future work will focus on the selection of the most appropriate tools to measure these outcomes (how to measure).

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STANDARDIZING AND HARMONIZING OUTCOME MEASUREMENT IN MULTIMODAL PAIN THERAPY- VAPAIN CORE OUTCOME SET RECOMMENDATION

10.26226/morressier.598b05d8d462b80290b5360e ◽

2017 ◽

Author(s):

Ulrike Kaiser

Keyword(s):

Pain Therapy ◽

Outcome Measurement ◽

Core Outcome Set ◽

Core Outcome ◽

Multimodal Pain Therapy ◽

Outcome Set

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A core outcome set for localised prostate cancer effectiveness trials: protocol for a systematic review of the literature and stakeholder involvement through interviews and a Delphi survey

Trials ◽

10.1186/s13063-015-0598-0 ◽

2015 ◽

Vol 16 (1) ◽

Cited By ~ 33

Author(s):

Steven MacLennan ◽

Hendrika J Bekema ◽

Paula R Williamson ◽

Marion K Campbell ◽

Fiona Stewart ◽

...

Keyword(s):

Prostate Cancer ◽

Systematic Review ◽

Core Outcome Set ◽

Stakeholder Involvement ◽

Delphi Survey ◽

Core Outcome ◽

Review Of The Literature ◽

Outcome Set ◽

Effectiveness Trials

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How to evaluate the clinical outcome of joint-preserving treatment for osteonecrosis of the femoral head: development of a core outcome set

Journal of Orthopaedic Surgery and Research ◽

10.1186/s13018-019-1364-x ◽

2019 ◽

Vol 14 (1) ◽

Cited By ~ 1

Author(s):

Zhipeng Xue ◽

Jigao Sun ◽

Taixian Li ◽

Zeqing Huang ◽

Weiheng Chen

Keyword(s):

Femoral Head ◽

Core Outcome Set ◽

Consensus Meeting ◽

Walking Distance ◽

Core Outcome ◽

Outcome Indicators ◽

X Ray ◽

Outcome Set ◽

Core Outcomes ◽

Hip Flexion

Abstract Background This study aimed to develop a core outcome set (COS) for clinical trials of joint-preserving treatment for osteonecrosis of the femoral head (ONFH), that is, to define a minimal set of outcomes that should be reported in such trials. Methods A mixed research method was adopted in this study. First, clinical trials of hip preservation therapy were systematically researched and analyzed. Second, a three-round Delphi survey involving both doctors and patients was carried out to obtain the core outcome indicators. Round 1 was a modified Delphi questionnaire for doctors and patients to determine which outcomes are important to these stakeholders, round 2 determined what clinical evaluation core outcomes should be included for the joint-preserving treatment of ONFH, and round 3 determined how core outcomes should be measured. Finally, a consensus meeting was held to discuss and vote on the established COS. Results The results of the systematic review showed that 42 outcome indicators were classified according to common signs and symptoms, quality of life, long-term outcomes, radiological evaluation, blood biochemistry, and indexes of safety. The three rounds of Delphi surveys completed the selection of indicators for the COS and the determination of the corresponding measurements. A total of 73 orthopedic doctors and 103 patients participated in round 1, and the top 10 indicators selected were basically the same. In round 2, 32 experts identified the following indicators: pain, range of motion (ROM) of hip flexion, walking distance, and stable rating of X-ray images. In round 3, 35 experts defined the measurement of each indicator. Finally, the consensus meeting identified the four indicators aforementioned that constituted the COS. The scores for pain, ROM of hip flexion, and walking distance are from 0 to 10; 0 represents the best scores, while 10 represents the most serious impairment. The stable rating of X-ray images is determined by the morphology of the femoral head and the change in the density of the necrotic area. Conclusions We established a COS for hip-preserving treatment of ONFH that includes four indicators: pain, ROM of hip flexion, walking distance, and stable rating of X-ray images.

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