IMMPACT-recommended outcome measures and tools of assessment in burning mouth syndrome RCTs: an international Delphi survey protocol.
ABSTRACT: BACKGROUND:A core outcome set (COS) represents the agreed minimum set of domains and measurement instruments that should be measured and reported in any clinical trial for a given condition. In BMS randomized controlled trials (RCTs), the outcomes identified in the existing literature regarding the efficacy of therapeutic interventions are numerous and diverse. Although the standardized IMMPACT core outcome domains has been developed for measurement of outcomes in chronic pain RCTs, no BMS-specific COS have been adopted and validated. With the evolving landscape of BMS management end points and the development of new therapies, a consensus on a COS for use in future BMS trials is paramount to reduce heterogeneity in outcome reporting. The aim of this study was to reach a consensus for adopting the standardized Initiative on Methods, Measurement, and Pain Assessment in Clinical Trials (IMMPACT) outcome domains, and their tools of assessment, for burning mouth syndrome (BMS) clinical trials and clinical practice. METHODS:A BMS-specific COS will be developed using the method recommended by the Core Outcome Measures in Effective Trials (COMET) initiative (Registration: http://www.comet-initiative.org/studies/details/1357 ). Selection of questionnaire outcome measures was informed by the IMMPACT consensus and previous systematic review of RCTs in BMS conducted by the consortium. An international group of clinicians and researchers will be invited to participate in several rounds of a Delphi survey. A consensus meeting will be held with the objective of ratifying the outcomes for inclusion in the COS. A finalized COS explanatory document will be drafted, including all outcomes and measurements as determined by the Delphi rounds and consensus meeting. DISCUSSION:A COS for the management of BMS will improve the quality of future RCTs, reduce outcome reporting heterogeneity, and facilitate more vigorous data synthesis of management interventions for systematic reviews and meta-analysis. This would ensure enhanced quality evidence for clinical management of the condition.
Project description:<h4>Background</h4>Pelvic Girdle Pain (PGP) is an important cause of disability and economic cost worldwide. There is a need for effective preventative and management strategies. Emerging studies measure a variety of outcomes rendering synthesis and translation to clinical practice difficult. A Core Outcome Set (COS) can address this problem by ensuring that data are relevant, useful and usable for making well-informed healthcare choices. The aim of this study is to develop a consensus-based PGP-COS, including agreement on methods (e.g. instruments) for measuring the construct outcomes in the COS for use in research and clinical practice. Furthermore, as there is uncertainty as to whether incorporating stakeholder interviews in addition to conducting a systematic review to determine an initial list of outcomes for the Delphi survey, or, whether using different rating scales in a Delphi survey impacts on the final COS, we propose to embed two methodological studies within the PGP-COS development process to address these questions.<h4>Methods</h4>The PGP-COS study will include five phases: (1) A systematic review of the literature and semi-structured interviews with 15 patients (three countries) to form the initial list of outcomes for the Delphi survey; (2) A 3-round Delphi including patients, clinicians, researchers and service providers; (3) A systematic review of methods for measuring the outcomes in the preliminary PGP-COS identified in the Delphi survey; (4) A face-to-face consensus meeting to agree on the final PGP-COS and methods for measuring the COS; (5) Global dissemination. To address the methodological questions, we will assess the number and type of outcomes, in the final PGP-COS, that were exclusively derived from the interviews. Secondly, we will randomise Delphi survey participants to either a 5-point or 9-point importance rating scale, and examine potential differences in 'important' ratings between the groups.<h4>Discussion</h4>There is currently no COS for measuring/monitoring PGP in trials and clinical practice. A PGP-COS will ensure that relevant outcomes are measured using appropriate measurement instruments for patients with PGP globally.<h4>Core outcome set registration</h4>This PGP-COS was registered with COMET (Core Outcome Measures for Effectiveness Trials) in January 2017 ( http://www.comet-initiative.org/studies/details/958 ).
Project description:<h4>Background</h4> 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. <h4>Methods</h4> 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. <h4>Results</h4> 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. <h4>Conclusion</h4> This COS is recommended to be used in clinical trials aimed at improving AMS in care homes. <h4>Supplementary Information</h4> The online version contains supplementary material available at 10.1186/s13756-021-00925-8.
Project description:BACKGROUND:A core outcome set (COS) is a standardised collection of outcomes to be collected and reported in all trials within a research area. A COS can reduce reporting bias and facilitate evidence synthesis. This is currently unavailable for use in community-based bipolar trials. This research aimed to develop such a COS, with input from a full range of stakeholders. METHODS:A co-production approach was used throughout. A longlist of outcomes was derived from focus groups with people with a bipolar diagnosis and carers, interviews with healthcare professionals and a rapid review of outcomes listed in bipolar trials on the Cochrane database. An expert panel with personal and/or professional experience of bipolar participated in a modified Delphi process and the COS was finalised at a consensus meeting. RESULTS:Fifty participants rated the importance of each outcome. Sixty-six outcomes were included in Round 1 of the questionnaire; 13 outcomes were added by Round 1 participants and were rated in Round 2. Seventy-six percent of participants (n = 38) returned to Round 2 and 60 outcomes, including 4 outcomes added by participants in Round 1, received a rating of 7-9 by >70% and 1-3 by <25% of the sample. Fourteen participants finalised a COS containing 11 outcomes at the consensus meeting: personal recovery; connectedness; clinical recovery of bipolar symptoms; mental health and wellbeing; physical health; self-monitoring and management; medication effects; quality of life; service outcomes; experience of care; and use of coercion. CONCLUSIONS:This COS is recommended for use in community-based bipolar trials to ensure stakeholder-relevant outcomes, facilitate data synthesis, and transparent reporting. The COS includes guidance notes for each outcome to allow the identification of suitable measurement instruments. Further validation is recommended for use with a wide range of communities and to achieve standardised measurement.
Project description:Inherited metabolic diseases (IMD) are a large group of rare single-gene disorders that are typically diagnosed early in life. There are important evidence gaps related to the comparative effectiveness of therapies for IMD, which are in part due to challenges in conducting randomized controlled trials (RCTs) for rare diseases. Registry-based RCTs present a unique opportunity to address these challenges provided the registries implement standardized collection of outcomes that are important to patients and their caregivers and to clinical providers and healthcare systems. Currently there is no core outcome set (COS) for studies evaluating interventions for paediatric IMD. This protocol outlines a study that will establish COS for each of two relatively common IMD in children, phenylketonuria (PKU) and medium-chain acyl-CoA dehydrogenase (MCAD) deficiency.This two-part study is registered with the Core Outcome Measures in Effectiveness Trials (COMET) initiative. Part 1 includes a rapid review and development of an evidence map to identify a comprehensive listing of outcomes reported in past studies of PKU and MCAD deficiency. The review follows established methods for knowledge synthesis, including a comprehensive search strategy, two stages of screening citations against inclusion/exclusion criteria by two reviewers working independently, and extraction of important data elements from eligible studies, including details of the outcomes collected and outcome measurement instruments. The review findings will inform part 2 of our study, a set of Delphi surveys to establish consensus on the highest priority outcomes for each condition. Healthcare providers, families of children with PKU or MCAD deficiency, and health system decision-makers will be invited to participate in two to three rounds of Delphi surveys. The design of the surveys will involve parents of children with IMD who are part of a family advisory forum.This protocol is a crucial step in developing the capacity to launch RCTs with meaningful outcomes that address comparative effectiveness questions in the field of paediatric IMD. Such trials will contribute high-quality evidence to inform decision-making by patients and their family members, clinicians, and policy-makers.
Project description:<h4>Background</h4>Colorectal cancer (CRC) is a major cause of worldwide morbidity and mortality. Surgical treatment is common, and there is a great need to improve the delivery of such care. The gold standard for evaluating surgery is within well-designed randomized controlled trials (RCTs); however, the impact of RCTs is diminished by a lack of coordinated outcome measurement and reporting. A solution to these issues is to develop an agreed standard "core" set of outcomes to be measured in all trials to facilitate cross-study comparisons, meta-analysis, and minimize outcome reporting bias. This study defines a core outcome set for CRC surgery.<h4>Methods and findings</h4>The scope of this COS includes clinical effectiveness trials of surgical interventions for colorectal cancer. Excluded were nonsurgical oncological interventions. Potential outcomes of importance to patients and professionals were identified through systematic literature reviews and patient interviews. All outcomes were transcribed verbatim and categorized into domains by two independent researchers. This informed a questionnaire survey that asked stakeholders (patients and professionals) from United Kingdom CRC centers to rate the importance of each domain. Respondents were resurveyed following group feedback (Delphi methods). Outcomes rated as less important were discarded after each survey round according to predefined criteria, and remaining outcomes were considered at three consensus meetings; two involving international professionals and a separate one with patients. A modified nominal group technique was used to gain the final consensus. Data sources identified 1,216 outcomes of CRC surgery that informed a 91 domain questionnaire. First round questionnaires were returned from 63 out of 81 (78%) centers, including 90 professionals, and 97 out of 267 (35%) patients. Second round response rates were high for all stakeholders (>80%). Analysis of responses lead to 45 and 23 outcome domains being retained after the first and second surveys, respectively. Consensus meetings generated agreement on a 12 domain COS. This constituted five perioperative outcome domains (including anastomotic leak), four quality of life outcome domains (including fecal urgency and incontinence), and three oncological outcome domains (including long-term survival).<h4>Conclusion</h4>This study used robust consensus methodology to develop a core outcome set for use in colorectal cancer surgical trials. It is now necessary to validate the use of this set in research practice.
Project description:<h4>Background</h4>Pregestational diabetes mellitus (PGDM) is associated with adverse pregnancy outcomes including increased rates of caesarean section birth, macrosomia, congenital malformation, prematurity, admission to the neonatal intensive care unit and stillbirth. As a result, there has been an increase in interventions to improve outcomes in both mother and infant. To date, meaningful comparisons between these studies are limited due to heterogeneity in outcome selection and reporting. The aim of this study is to develop a core outcome set (COS) for randomised controlled trials evaluating the effectiveness of interventions for the treatment of pregnant women with PGDM.<h4>Methods</h4>The study consists of three steps. The first step is a systematic review of the literature to assess outcomes reported in randomised controlled trials assessing the effectiveness of interventions for the treatment of pregnant women with PGDM. The second step is a three round, online Delphi survey to prioritise these outcomes. In this step, stakeholders (including women with PGDM, healthcare workers, researchers and policymakers) will be asked to rank the importance of outcomes for inclusion in the COS using a 9-point Likert type scale. Outcomes that meet the inclusion criteria after completion of the Delphi surveys will be brought to the consensus meeting. The consensus meeting will be the third and final step, where the COS will be finalised. The consensus meeting will include members from each stakeholder group.<h4>Discussion</h4>This paper describes the process used to develop a COS for the reporting of studies evaluating the effectiveness of interventions in pregnant women with PGDM. The COS will enable greater comparison between and information synthesis across RCTs in the treatment of PGDM. In addition, this COS will also help improve trial reporting and minimise research waste by prioritising the collection and reporting of outcomes that matter to all relevant stakeholder groups.<h4>Trial registration</h4>This COS has been registered with the Core Outcome Measures in Effectiveness Trials (COMET) initiative ( http://www.comet-initiative.org/studies/details/1425 ) on the 4th of November 2019. The systematic review component of this study has also been registered with the International Prospective Register of Systematic Reviews (PROSPERO) ( https://www.crd.york.ac.uk/prospero/display_record.php?ID=CRD42020173549 ).
Project description:This masterclass introduces the topic of core outcome sets, describing rationale and methods for developing them, and providing some examples that are relevant for clinical research and practice.A core outcome set is a minimum consensus-based set of outcomes that should be measured and reported in all clinical trials for a specific health condition and/or intervention. Issues surrounding outcome assessment, such as selective reporting and inconsistency across studies, can be addressed by the development of a core set. As suggested by key initiatives in this field (i.e. OMERACT and COMET), the development requires achieving consensus on: (1) core outcome domains and (2) core outcome measurement instruments. Different methods can be used to reach consensus, including: literature systematic reviews to inform the process, qualitative research with clinicians and patients, group discussions (e.g. nominal group technique), and structured surveys (e.g. Delphi technique). Various stakeholders should be involved in the process, with particular attention to patients.Several COSs have been developed for musculoskeletal conditions including a longstanding one for low back pain, IMMPACT recommendations on outcomes for chronic pain, and OMERACT COSs for hip, knee and hand osteoarthritis. There is a lack of COSs for neurological, geriatric, cardio-respiratory and pediatric conditions, therefore, future research could determine the value of developing COSs for these conditions.
Project description:Introduction:In recent years, there has been growing interest in alternatives to appendicectomy. In particular, non-operative treatment of appendicitis, with antibiotics alone, has been proposed as a potential treatment. A small number of randomised controlled trials (RCTs) in adults and, more recently, children suggest that antibiotic treatment may be a valid alternative to appendicectomy. However, there is currently insufficient data to justify its widespread use. Prior to performing further efficacy studies of the treatment of appendicitis in children, it is imperative to identify the most relevant outcome measures for inclusion in the design of comparative studies. This is of particular importance when evaluating a novel treatment approach since the outcomes of importance may differ from those commonly reported with traditional therapies.A review of the relevant literature and electronic resources failed to identify a core outcome set (COS) for children with appendicitis. We aim to define a COS for the measurement of treatment interventions in children (<18 years) with acute appendicitis. Methods and analysis:This project will entail: (1) a systematic review to identify previously reported acute uncomplicated appendicitis treatment outcomes; (2) assembly of stakeholder panels (paediatric and adult surgeons, patients and parents); (3) a three-stage Delphi process; and (4) a final consensus meeting to complete the COS. Ethics and registration:COS development is part of CONservative TReatment of Appendicitis in Children - a randomised controlled Trial (Feasibility) (CONTRACT) study, for which full ethical approval for CONTRACT has been granted. The COS development study is registered with the COMET Initiative in May 2017 (http://www.comet-initiative.org/studies/details/987).
Project description:The key aim of the study is to establish an agreed standardised core outcome set (COS) for use when evaluating non-pharmacological health and social care interventions for people living at home with dementia.Drawing on the guidance and approaches of the Core Outcome Measures in Effectiveness Trials (COMET), this study uses a four-phase mixed-methods design: 1 Focus groups and interviews with key stakeholder groups (people living with dementia, care partners, relevant health and social care professionals, researchers and policymakers) and a review of the literature will be undertaken to build a long list of outcomes. 2 Two rounds of Delphi surveys will be used with key stakeholder groups. Statements for the Delphi surveys and participation processes will be developed and informed through substantial member involvement with people living with dementia and care partners. A consensus meeting will be convened with key participant groups to discuss the key findings and finalise the COS. 3 A systematic literature review will be undertaken to assess the properties of tools and instruments to assess components of the COS. Measurement properties, validity and reliability will be assessed using the Consensus-based Standards for the Selection of Health Measurement (COSMIN) and COMET guidance. 4 A stated preference survey will elicit the preferences of key stakeholders for the outcomes identified as important to measure in the COS.To the best of our knowledge, this study is the first to use a modified Delphi process to involve people living with dementia as a participant group. Though the study is confined to collecting data in the United Kingdom, use of the COS by researchers will enhance the comparability of studies evaluating non-pharmacological and community-based interventions.The study is registered on the COMET initiative, registered in 2014 at comet-initiative.org .
Project description:<h4>Introduction</h4>Myocardial infarction (MI) is the most dangerous complication in patients with coronary heart disease. In China, there is an increasing number of randomised controlled trials (RCTs) of traditional Chinese medicine (TCM) for treating MI. However, the inconsistency of outcome reporting means that a large number of clinical trials cannot be included in systematic reviews to provide the best evidence for clinical practice. The aim of this study is to develop a core outcome set (COS) for future TCM clinical trials of MI, which may improve the consistency of outcome reporting and facilitate the synthesis of data across studies in systematic reviews.<h4>Methods and analysis</h4>We will conduct a systematic review of MI clinical trials with any intervention. Semistructured interviews will be conducted to obtain the perspectives of patients with MI. The outcomes from the systematic review and semistructured interviews will be grouped and used to develop a questionnaire. The questionnaire will be developed as a supplement for the TCM syndromes of MI and will be constructed from the results of a systematic review, existing medical records and a cross-sectional study. Then two rounds of the Delphi survey will be conducted with different stakeholders (TCM experts and Western medicine experts in cardiovascular disease, methodologists, magazine editors and patients) to determine the importance of the outcomes. Only the TCM experts will need to response to the questionnaire for core TCM syndromes. A face-to-face consensus meeting will be conducted to create a final COS and recommend measurement time for each outcome.<h4>Ethics and dissemination</h4>This project has been approved by the Ethics Committee of Dongzhimen Hospital, Beijing University of Chinese Medicine. The final COS will be published and freely available.<h4>Trial registration number</h4>This study is registered with the Core Outcome Measures in Effectiveness Trials database as study 1243 (available at: http://www.comet-initiative.org/studies/details/1243).