scholarly journals Establishing a core outcome set for mucopolysaccharidoses (MPS) in children: study protocol for a rapid literature review, candidate outcomes survey, and Delphi surveys

Trials ◽  
2021 ◽  
Vol 22 (1) ◽  
Author(s):  
Alison H. Howie ◽  
Kylie Tingley ◽  
Michal Inbar-Feigenberg ◽  
John J. Mitchell ◽  
Nancy J. Butcher ◽  
...  
Keyword(s):  
Literature Review ◽  
Health Care Providers ◽  
Metabolic Diseases ◽  
Outcome Measurement ◽  
Core Outcome Set ◽  
Core Outcome ◽  
Care Providers ◽  
Measurement Instruments ◽  
Two Phase ◽  
Outcome Set

Abstract Background Mucopolysaccharidoses (MPS) are a group of inherited metabolic diseases characterized by chronic, progressive multi-system manifestations with varying degrees of severity. Disease-modifying therapies exist to treat some types of MPS; however, they are not curative, underscoring the need to identify and evaluate co-interventions that optimize functioning, participation in preferred activities, and quality of life. A Canadian pediatric MPS registry is under development and may serve as a platform to launch randomized controlled trials to evaluate such interventions. To promote the standardized collection of patient/family-reported and clinical outcomes considered important to patients/families, health care providers (HCPs), and policymakers, the choice of outcomes to include in the registry will be informed by a core outcome set (COS). We aim to establish a patient-oriented COS for pediatric MPS using a multi-stakeholder approach. Methods In step 1 of the six-step process to develop the COS, we will identify relevant outcomes through a rapid literature review and candidate outcomes survey. A two-phase screening approach will be implemented to identify eligible publications, followed by extraction of outcomes and other pre-specified data elements. Simultaneously, we will conduct a candidate outcomes survey with children with MPS and their families to identify outcomes most important to them. In step 2, HCPs experienced in treating patients with MPS will be invited to review the list of outcomes generated in step 1 and identify additional clinically relevant outcomes. We will then ask patients/families, HCPs, and policymakers to rate the outcomes in a set of Delphi Surveys (step 3), and to participate in a subsequent consensus meeting to finalize the COS (step 4). Step 5 involves establishing a set of outcome measurement instruments for the COS. Finally, we will disseminate the COS to knowledge users (step 6). Discussion The proposed COS will inform the choice of outcomes to include in the MPS registry and, more broadly, promote the standardized collection of patient-oriented outcomes for pediatric MPS research. By involving patients/families from the earliest stage of the research, we will ensure that the COS will be relevant to those who will ultimately benefit from the research. Trial registration PROSPERO CRD42021267531, COMET

scholarly journals Development of a Core Outcome Set for Effectiveness Studies of Breech Birth at Term (Breech-COS)- An International Multi-Stakeholder Delphi Study: Study Protocol

2021 ◽  
Author(s):  
Shawn Walker ◽  
Tisha Dasgupta ◽  
Andrew Shennan ◽  
Jane Sandall ◽  
Catey Bunce ◽  
...  
Keyword(s):  
Literature Review ◽  
Core Outcome Set ◽  
Delphi Survey ◽  
Neonatal Outcomes ◽  
Core Outcome ◽  
Care Providers ◽  
Outcome Set ◽  
Increased Risk ◽  
Breech Birth ◽  
Effectiveness Studies

Abstract Background: Women pregnant with a breech-presenting foetus at term are at increased risk of adverse pregnancy outcomes. The most common intervention used to improve neonatal outcomes is planned delivery by caesarean section. But this is not always possible, and some women prefer to plan a vaginal birth. A number of providers have proposed alternative interventions, such as delivery protocols or specialist teams, but heterogeneity in reported outcomes and their measurements prevents meaningful comparisons. The aim of this paper is to present a protocol for a study to develop a Breech Core Outcome Set (Breech-COS) for studies evaluating the effectiveness of interventions to improve outcomes associated with term breech birth.Methods: The development of a Breech-COS includes three phases. First, a systematic literature review will be conducted to identify outcomes previously used in effectiveness studies of breech birth at term. A focus group discussion will be conducted with the study’s pre-established Patient and Public Involvement (PPI) group, to enable service user perspectives on the results of literature review to influence the design of Delphi survey instrument. Second, an international Delphi survey will be conducted to prioritize outcomes for inclusion in the Breech-COS from the point of view of key stakeholders, including perinatal care providers and families who have experienced a term breech pregnancy. Finally, a consensus meeting will be held with stakeholders to ratify the Breech-COS and disseminate findings for application in future effectiveness studies.Discussion: The expectation is that the Breech-COS will always be collected in all clinical trials, audits of practice and other forms of observation research that concern breech birth at term, along with other outcomes of interest. This will facilitate comparing, contrasting and combining studies with the ultimate goal of improved maternal and neonatal outcomes. Trial registration: Core Outcome Measures in Effectiveness Trials (COMET): #1749; Core Outcomes in Women’s and Newborn Health (CROWN) Initiative.

scholarly journals A Core Outcome Set for Nonpharmacological Community-Based Interventions for People Living With Dementia at Home: A Systematic Review of Outcome Measurement Instruments

2020 ◽  
Author(s):  
Andrew J E Harding ◽  
Hazel Morbey ◽  
Faraz Ahmed ◽  
Carol Opdebeeck ◽  
Ruth Elvish ◽  
...  
Keyword(s):  
Outcome Measurement ◽  
Core Outcome Set ◽  
Face Validity ◽  
Core Outcome ◽  
Measurement Instruments ◽  
Community Based Interventions ◽  
Community Based ◽  
Key Stakeholders ◽  
Outcome Set ◽  
Dementia Research

Abstract Background and Objectives It is questionable whether existing outcome measurement instruments (OMIs) in dementia research reflect what key stakeholders’ value. We attained consensus from more than 300 key stakeholders, including people living with dementia, and identified 13 core outcome items for use in nonpharmacological and community-based interventions for people with dementia living at home. In this systematic review, we review OMIs that have previously been used in dementia care research to determine how, or even if, the 13 core outcome items can be measured. Research Design and Methods We extracted self-reported OMIs from trials, reviews, and reports of instrument development. Searches were undertaken in the ALOIS database, MEDLINE, PsycINFO, CINAHL, SocINDEX, and COSMIN databases. We aimed to assess the psychometric properties of OMI items for face validity with the core outcome items, content validity, internal consistency, and responsiveness. We held a coresearch workshop involving people living with dementia and care partners in order to ratify the findings. Results In total 347 OMIs were located from 354 sources. Of these, 76 OMIs met the inclusion criteria. No OMIs were deemed to have sufficient face validity for the core outcome set (COS) items, and no OMIs proceeded to further assessment. The “best” available OMI is the Engagement and Independence in Dementia Questionnaire. Discussion and Implications This study provides a practical resource for those designing dementia research trials. Being able to measure the COS items would herald a paradigm shift for dementia research, be responsive to what key stakeholders value and enhance the ability to make comparisons.

scholarly journals How to select outcome measurement instruments for outcomes included in a “Core Outcome Set” – a practical guideline

Trials ◽  
2016 ◽  
Vol 17 (1) ◽  
Author(s):  
Cecilia A. C. Prinsen ◽  
Sunita Vohra ◽  
Michael R. Rose ◽  
Maarten Boers ◽  
Peter Tugwell ◽  
...  
Keyword(s):  
Outcome Measurement ◽  
Core Outcome Set ◽  
Core Outcome ◽  
Measurement Instruments ◽  
Practical Guideline ◽  
Outcome Set

scholarly journals Heterogeneity in outcome selection, definition and measurement in studies assessing the treatment of cryptoglandular anal fistula: findings from a systematic review

2021 ◽  
Author(s):  
A. J. H. M. Machielsen ◽  
N. Iqbal ◽  
M. L. Kimman ◽  
K. Sahnan ◽  
S. O. Adegbola ◽  
...  
Keyword(s):  
Systematic Review ◽  
Anal Fistula ◽  
Outcome Measurement ◽  
Core Outcome Set ◽  
Adult Patients ◽  
Cochrane Library ◽  
Core Outcome ◽  
Measurement Instruments ◽  
Outcome Reporting ◽  
Outcome Set

Abstract Background Treatment for cryptoglandular anal fistula (AF) is challenging and a lack of uniform outcomes in the literature prevents direct comparison of treatments. This can be addressed by developing a core outcome set, a standardised set of outcomes reported in all interventional studies for a specific condition. The aim of this systematic review is to assess the range of outcomes, their definitions, and the measurement instruments currently utilised in interventional studies for adult patients with AF. This will inform the development of an AF core outcome set. Methods Medline, Embase and The Cochrane Library were searched to identify all patient- and clinician-reported outcomes in studies assessing medical, surgical or combination treatment of adult patients with AF published from January 2008 to May 2020. The resulting outcomes were categorized according to the Core Outcome Measurement in Effectiveness Trials (COMET) taxonomy to better understand their distribution. Results In total, 155 studies were included, 552 outcomes were extracted, with a median of three outcomes (interquartile range 2–5) per study. Only 25% of studies demonstrated high-quality outcome reporting. The outcomes were merged into 52 unique outcomes and structured into four core areas and 14 domains, with the majority in the domain of physiological or clinical (gastrointestinal) outcomes. The most commonly reported outcomes were healing (77%), incontinence (63%), and recurrence (40%), with no single outcome assessed across all studies. There was a wide variation in outcome definitions and measurement instruments used. Conclusions There is substantial heterogeneity in outcomes, definitions, and measurement instruments reported in interventional studies for cryptoglandular anal fistula. This emphasises the need for standardised outcome reporting and measurement.

scholarly journals Protocol for the development of a core outcome set for cauda equina syndrome: systematic literature review, qualitative interviews, Delphi survey and consensus meeting

BMJ Open ◽  
2019 ◽  
Vol 9 (4) ◽  
pp. e024002 ◽  
Author(s):  
Nisaharan Srikandarajah ◽  
Adam J Noble ◽  
Martin Wilby ◽  
Simon Clark ◽  
Paula R Williamson ◽  
...  
Keyword(s):  
Literature Review ◽  
Systematic Literature Review ◽  
Cauda Equina Syndrome ◽  
Qualitative Interviews ◽  
Core Outcome Set ◽  
Cauda Equina ◽  
Consensus Meeting ◽  
Core Outcome ◽  
The Core ◽  
Outcome Set

IntroductionCauda equina syndrome (CES) is a serious neurological condition most commonly due to compression of the lumbosacral nerve roots, which can result in significant disability. The evidence for acute intervention in CES is mainly from retrospective studies. There is heterogeneity in the outcomes chosen for analysis in these studies, which makes it difficult to synthesise the data across studies. This study will develop a core outcome set for use in future studies of CES, engaging with key stakeholders and using transparent methodology. This will help ensure that relevant outcomes are used in future and will facilitate attempts to summarise data across studies in systematic reviews.Methods and analysisA systematic literature review will document all the outcomes for CES after surgery mentioned in the literature. The qualitative interviews with patients with CES will be semistructured, audio recorded, transcribed and thematically analysed with the use of NVivo V.10 to identify outcomes and determine the themes described. The outcomes from the literature review and patient interviews will be combined and prioritised to determine what the most important outcomes are in CES research studies to patients and healthcare professionals. The prioritisation will be done through a two-round iterative Delphi survey and a consensus meeting. This process will decide the core outcome set for patients with CES.Ethics and disseminationREC and HRA approval was obtained on the 6/12/16 for the qualitative interviews from South Central—Hampshire A REC. REC reference 16/SC/0587. REC and HRA approval was obtained on 26/3/18 for the Delphi process and consensus meeting from North West—Greater Manchester Central REC. REC reference was 18/NW/0022. The final core outcome set will be published and freely available.Trial registration numberThis study is registered with the Core Outcome Measures in Effectiveness Trials database as study 824.

scholarly journals 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)

2021 ◽  
Author(s):  
Victoria Shepherd ◽  
Fiona Wood ◽  
Michael Robling ◽  
Elizabeth Randell ◽  
Kerenza Hood
Keyword(s):  
Outcome Measurement ◽  
Core Outcome Set ◽  
Consensus Meeting ◽  
Delphi Survey ◽  
Additional Patient ◽  
Trial Participation ◽  
Core Outcome ◽  
Capacity To Consent ◽  
Outcome Set ◽  
Participation Decisions

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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