Core Outcome Set Research Articles

Developing a core outcome set for acetabular fractures: a systematic review protocol

BackgroundClinical trials investigating acetabular fractures are heterogeneous in their investigated outcomes and their corresponding measurements. Standardization may facilitate comparability and pooling of research results, which would lead to an increase in knowledge about the optimal treatment of acetabular fractures, resulting in long-term evidence-based treatment decisions and improvements in patient care. The aim of this systematic review is to identify the reported outcomes and their measurements from studies on treatments for acetabular fractures to develop a core outcome set which contains the most relevant outcome measures to be included in future studies.MethodsStudies published in English and German including patients aged 16 years and older, with a surgically treated acetabular fracture, will be included. Studies with nonsurgical treatment, pathologic fractures, polytraumatized patients, and patients younger than 16 years of age will be excluded because other outcomes may be of interest in these cases. Any prospective and retrospective study will be included. Systematic reviews will be excluded, but their included studies will be screened for eligibility. The literature will be searched on MEDLINE, CENTRAL, Web of Science, ClinicalTrials.gov, and WHO ICTRP. Risk of selective reporting of outcomes will be assessed using the Outcome Reporting Bias in Trials classification system. Heterogeneously defined outcomes that measure the same outcome will be grouped and subsequently categorized into outcome domains using the taxonomy of the Core Outcome Measures in Effectiveness Trials Initiative.DiscussionIt is expected that a high number of studies will be included, and many outcomes will be identified using different definitions and measurement instruments. A limitation of this systematic review is that only previously investigated outcomes will be detected, thus disregarding potentially relevant outcomes.Systematic review registrationPROSPERO CRD42022357644

Feasibility and utilization of electronic patient-reported outcome measures for lung cancer in routine clinical care.

11107 Background: Prior studies have shown the value of Patient-Reported Outcomes (PRO) in cost burden, quality of life (QoL), and survival in cancer care. To facilitate focus on patient-centricity and value-based care, the International Consortium for Health Outcomes Measurements (ICHOM) and European Health Outcomes Observatory (H2O) developed a Core Outcomes Set (COS) in lung cancer. We explored the feasibility of deploying standardized PRO measures among patients with lung cancer in US clinical practice. Methods: A retrospective database study was conducted, with IRB waiver, using Carevive PROmPT, an electronic PRO (ePRO) remote symptom monitoring platform with weekly surveys comparable in scope to ICHOM/H2O COS, of symptomatic adverse events (PRO-CTCAE), physical function (PROMIS 4A Physical Function), and QoL items (EORTC QLQ-C30 items #29,30). Severe PRO-CTCAE symptom scores triggered care team alerts. From 9/2020 to 5/2023, 207 patients enrolled across 9 US cancer centers. Patients were ≥18 years, with lung cancer, and in current treatment. Patient engagement was measured by survey compliance, follow-up time, and completeness; clinician engagement was measured by platform utilization time, time to alert response, and clinical action taken. Results: Participants were 50% female, 86% White, 12% African American, median age 68, 81% NSCLC, and 26% with metastatic disease. Median survey persistence: 8 weeks, with 85% compliance over that time. Of domains assessed, 37 (58%) from Carevive data overlapped with ICHOM/H2O COS, with 19 measured over time. Overlapping domains included 15 clinical outcomes with 60% median completion rate and 22 PRO domains with 98% median completion. Of 19 domains assessed longitudinally, median compliance was 76%. Of patients triggering ≥1 ePRO symptom alert, 87% were within 4 weeks, with 46% requesting call-backs. Median clinician follow-up to alerts was 20 hours, with 69.3% addressed within 48 hours. Clinicians accessed the platform 2-3 times weekly, for median 2 minutes (range: 1-14 minutes) per login for nurses and Advanced Practitioners (AP). 89% of alerts were addressed by nurses and AP. Of alert responses, 66% indicated continuation of treatment, 12% were escalated to the clinical team, and 1% were escalated to hospitalization or ER visit. Conclusions: Results support the feasibility of integrating a standard set of multi-domain ePRO measures, comparable to the ICHOM/H2O COS, into routine cancer care, across multiple sites, with consistent patient engagement and provider utilization. One limitation is that clinical and financial impact were not directly assessed. Future research should explore direct clinician feedback, systematic implementation of PRO measures, and impacts on patient outcomes, resource utilization, and health systems.

Laparoscopic cholecystectomy versus conservative management for adults with uncomplicated symptomatic gallstones: the C-GALL RCT.

Gallstone disease is a common gastrointestinal disorder in industrialised societies. The prevalence of gallstones in the adult population is estimated to be approximately 10-15%, and around 80% remain asymptomatic. At present, cholecystectomy is the default option for people with symptomatic gallstone disease. To assess the clinical and cost-effectiveness of observation/conservative management compared with laparoscopic cholecystectomy for preventing recurrent symptoms and complications in adults presenting with uncomplicated symptomatic gallstones in secondary care. Parallel group, multicentre patient randomised superiority pragmatic trial with up to 24 months follow-up and embedded qualitative research. Within-trial cost-utility and 10-year Markov model analyses. Development of a core outcome set for uncomplicated symptomatic gallstone disease. Secondary care elective settings. Adults with symptomatic uncomplicated gallstone disease referred to a secondary care setting were considered for inclusion. Participants were randomised 1: 1 at clinic to receive either laparoscopic cholecystectomy or observation/conservative management. The primary outcome was quality of life measured by area under the curve over 18 months using the Short Form-36 bodily pain domain. Secondary outcomes included the Otago gallstones' condition-specific questionnaire, Short Form-36 domains (excluding bodily pain), area under the curve over 24 months for Short Form-36 bodily pain domain, persistent symptoms, complications and need for further treatment. No outcomes were blinded to allocation. Between August 2016 and November 2019, 434 participants were randomised (217 in each group) from 20 United Kingdom centres. By 24 months, 64 (29.5%) in the observation/conservative management group and 153 (70.5%) in the laparoscopic cholecystectomy group had received surgery, median time to surgery of 9.0 months (interquartile range, 5.6-15.0) and 4.7 months (interquartile range 2.6-7.9), respectively. At 18 months, the mean Short Form-36 norm-based bodily pain score was 49.4 (standard deviation 11.7) in the observation/conservative management group and 50.4 (standard deviation 11.6) in the laparoscopic cholecystectomy group. The mean area under the curve over 18 months was 46.8 for both groups with no difference: mean difference -0.0, 95% confidence interval (-1.7 to 1.7); p-value 0.996; n = 203 observation/conservative, n = 205 cholecystectomy. There was no evidence of differences in quality of life, complications or need for further treatment at up to 24 months follow-up. Condition-specific quality of life at 24 months favoured cholecystectomy: mean difference 9.0, 95% confidence interval (4.1 to 14.0), p < 0.001 with a similar pattern for the persistent symptoms score. Within-trial cost-utility analysis found observation/conservative management over 24 months was less costly than cholecystectomy (mean difference -£1033). A non-significant quality-adjusted life-year difference of -0.019 favouring cholecystectomy resulted in an incremental cost-effectiveness ratio of £55,235. The Markov model continued to favour observation/conservative management, but some scenarios reversed the findings due to uncertainties in longer-term quality of life. The core outcome set included 11 critically important outcomes from both patients and healthcare professionals. The results suggested that in the short term (up to 24 months) observation/conservative management may be a cost-effective use of National Health Service resources in selected patients, but subsequent surgeries in the randomised groups and differences in quality of life beyond 24 months could reverse this finding. Future research should focus on longer-term follow-up data and identification of the cohort of patients that should be routinely offered surgery. This trial is registered as ISRCTN55215960. This award was funded by the National Institute for Health and Care Research (NIHR) Health Technology Assessment programme (NIHR award ref: 14/192/71) and is published in full in Health Technology Assessment; Vol. 28, No. 26. See the NIHR Funding and Awards website for further award information.

Development of a core outcome set for cardiac rehabilitation in patients with myocardial infarction: a study protocol

IntroductionHeterogeneous outcome reporting is common in clinical trials focused on cardiac rehabilitation for myocardial infarction (MI); this practice often results in the exclusion of data from clinical trials in systematic...

Core outcome Set for Cardio-oncology (COS-CO): development of a set of outcomes for the cardiovascular assessment and monitoring of cancer patients and survivors.

There is an increasing awareness of the evidence-based selection of outcomes to be measured in clinical trials and clinical practice. Currently, there is no core outcome set (COS) for cardio-oncology, which may hinder the (inter)national comparison of the effectiveness of research and the quality of cardio-oncology care. The aim of this study is to develop a standard and pragmatic patient-centred outcome set to assess and monitor cancer patients and survivors at risk of or with cardiovascular diseases. A list of outcome domains was generated through a review of registries and guidelines, and six patient interviews. The project team reviewed and refined the outcome domains prior to starting a two-round Delphi procedure conducted between January-June 2022. The panellists, including healthcare providers and researchers, were invited to rate the importance of the outcomes. 26 experts from 11 countries rated a list of 93 outcomes (round 1) and 63 outcomes (round 2) to gain consensus on a list of outcome measures, and of demographic factors, health status and treatment variables. The final COS includes 15 outcome measures, reflecting four core areas: life impact (n=2), pathophysiological manifestations (n=9), resource use/economic impact (n=1), and mortality/survival (n=3). Next, six demographic factors, 21 health status, three cardiovascular and nine cancer variables were included. This is the first international development of a COS for cardio-oncology. This set aims to facilitate (inter)national comparison in cardio-oncology care, using standardised parameters and meaningful patient-centred outcomes for research and quality of care assessments.

#2535 Development of a patient-centered outcome strategy for the CONVINCE trial

Abstract Background and Aims Patient-centered outcomes play an important role when assessing treatment effects. Patient-reported outcome (PRO) measures assessing symptoms and impacts are important tools to capture the patient perspective in clinical trials. However, careful consideration is needed when choosing PROs and PRO measures. Aspects such as the conceptual model, measurement model, validity, reliability, and responsiveness, as well as translation, interpretability of scores and burden for participants and investigators have to be considered when developing a patient-centered outcome strategy. The CONVINCE randomized controlled trial was funded by the European Union (HORIZON2020) to assess effects of high-dose hemodiafiltration (HDF) versus high-flux hemodialysis (HD). The primary outcome is all-cause mortality and one of the secondary outcomes is the experienced health status of the patients as measured by patient-reported outcomes. The aim of this study is to describe the approach for the development of a patient-centered outcome strategy using a domain-based approach that was implemented in the CONVINCE trial. Method The development of the patient-centered outcome strategy for the CONVINCE trial followed three consecutive steps: (1) targeted literature review to identify core outcome sets, as well as concepts and PRO measures in the field of dialysis; (2) semi-structured interviews and focus groups with dialysis patients (n = 13) and clinicians (n = 4) to determine health aspects, i.e. symptoms and impacts most relevant to patient population; (3) triangulation of results to determine the most relevant health domains to assess within the context of the CONVINCE trial and determination of PRO measures in due consideration of psychometric properties of respective instruments. Results We identified 97 PRO measures reported in previous dialysis studies, assessing more than 11 health domains and 41 disease-specific symptoms/problems relevant for dialysis patients. Core outcome sets, such as the SONG-HD and the ICHOM standard set for chronic kidney disease, indicated in total 19 outcomes that we classified as potential PROs. Interviews with patients and clinicians confirmed health domains and outcomes that were identified in the literature and provided further insights in terms of the level of importance of health domains. To balance the comprehensiveness of assessments and respondent burden, we decided to concentrate on 9 domains and 20 symptoms/problems according to the objectives of the trial. For the more proximal outcomes, i.e. recovery time and recovery time pattern, new items were developed by the CONVINCE consortium applying established state-of-the-art methods. For generic outcomes, e.g. fatigue, physical function, we decided to apply psychometrically advanced tools from the PROMIS® initiative, validated within a general population as well as various chronic health conditions. Conclusion Building upon core outcome sets by confirming health concepts most relevant for dialysis patients within the specific context of the CONVINCE trial, enabled us to develop a robust patient-centered outcome measurement strategy for the CONVINCE trial that assesses relevant health domains of dialyses patients in granular fashion using a domain-based approach. Further validation of the PRO measures should be considered, especially establishing meaningful change estimates to support interpretation of change in PRO measurement scores. Conference Abstract ERA EDTA; May 23-26, 2024; Stockholm, Sweden Kathrin I. Fischer, Peter J. Blankestijn, Krister Cromm, Michiel L. Bots, Claudia Barth, Bernard Canaud, Jörgen Hegbrant, Giovanni Strippoli, Andrew Davenport, Mark Woodward, Matthias Rose for the CONVINCE Scientific Committee and CONVINCE Investigators

Critical outcomes to be included in the Core Outcome Set for nutritional intervention studies in older adults with malnutrition or at risk of malnutrition: a modified Delphi Study

IntroductionAs part of the development of an agreed minimum set of outcomes or Core Outcome Set (COS) for future nutritional intervention trials in older adults with malnutrition or at risk of malnutrition, this work reports on the Delphi surveys and final consensus.MethodsOutcomes from a scoping review were incorporated into a two-round Delphi survey. Researchers and healthcare professionals experienced in malnutrition in older adults were invited to take part in an online survey to rate 38 selected outcomes on a nine-point Likert scale ranging from ‘not important’ to ‘critical’ for their setting (community, hospital, or long-term care). Consensus for inclusion was reached when ≥75% (or ≥60% if a patient-reported outcome) of the participants scored the outcome as ‘critical’ and <15% as ‘not important’. Resulting outcomes were voted for inclusion or exclusion in the COS in a final online consensus meeting.ResultsNinety-three and 72 participants from diverse professional backgrounds and countries participated in the 1st and 2nd Delphi round, respectively. After both rounds eleven outcomes met the inclusion criteria, largely irrespective of setting. Fifteen participants, representing academia, health care, health policy, industry, and PPI, voted in a final online consensus meeting resulting in ten outcomes: malnutrition status, dietary intake, appetite, body weight or BMI, muscle strength, muscle mass, functional performance, functional limitations, quality of life, and acceptability of the intervention.ConclusionsTen outcomes will form the COS which is intended to be used by the scientific community in all future nutritional intervention studies for older adults with malnutrition or at risk of malnutrition. The subsequent phase will establish the appropriate methods to measure these outcomes.

Fostering connections at OMERACT 2023: A collaborative approach to Core Outcome Set development

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Development of an International Standard Set of Outcomes and Measurement Methods for Routine Practice for Infants, Children, and Adolescents with Epilepsy: The International Consortium for Health Outcomes Measurement Consensus Recommendations.

At present, there is no internationally accepted set of core outcomes or measurement methods for epilepsy clinical practice. The International Consortium for Health Outcomes Measurement (ICHOM) convened an international working group of experts in epilepsy, people with epilepsy, and their representatives to develop minimum sets of standardized outcomes and outcome measurement methods for clinical practice. Using modified Delphi consensus methods with consecutive rounds of online voting over 12 months, a core set of outcomes and corresponding measurement tool packages to capture the outcomes were identified for infants, children, and adolescents with epilepsy. Consensus methods identified 20 core outcomes. In addition to the outcomes identified for the ICHOM Epilepsy adult standard set, behavioral, motor, and cognitive/language development outcomes were voted as essential for all infants and children with epilepsy. The proposed set of outcomes and measurement methods will facilitate the implementation of the use of patient-centered outcomes in daily practice.

Effect of Probiotic Supplements on the Oral Microbiota-A Narrative Review.

Data from systematic reviews and meta-analyses show that probiotics positively impact clinical parameters of oral diseases such as gingivitis, dental caries, and periodontitis. However, the working mechanism of probiotics is not fully understood, but is hypothesized to be mediated by direct and indirect interactions with the oral microbiota and the human host. In the present narrative review, we focused on the microbiological effect of probiotic supplements based on data retrieved from randomized clinical trials (RCTs). In addition, we assessed to what extent contemporary molecular methods have been employed in clinical trials in the field of oral probiotics. Multiple RCTs have been performed studying the potential effect of probiotics on gingivitis, dental caries, and periodontitis, as evaluated by microbial endpoints. In general, results are conflicting, with some studies reporting a positive effect, whereas others are not able to record any effect. Major differences in terms of study designs and sample size, as well as delivery route, frequency, and duration of probiotic consumption, hamper comparison across studies. In addition, most RCTs have been performed with a limited sample size using relatively simple methods for microbial identification, such as culturing, qPCR, and DNA-DNA checkerboard, while high-throughput methods such as 16S sequencing have only been employed in a few studies. Currently, state-of-the-art molecular methods such as metagenomics, metatranscriptomics, and metaproteomics have not yet been used in RCTs in the field of probiotics. The present narrative review revealed that the effect of probiotic supplements on the oral microbiota remains largely uncovered. One important reason is that most RCTs are performed without studying the microbiological effect. To facilitate future systematic reviews and meta-analyses, an internationally agreed core outcome set for the reporting of microbial endpoints in clinical trials would be desirable. Such a standardized collection of outcomes would most likely improve the quality of probiotic research in the oral context.

Fetal lower urinary tract obstruction: international Delphi consensus on management and core outcome set.

To reach an international expert consensus on the diagnosis, prognosis and management of fetal lower urinary tract obstruction (LUTO) by means of a Delphi procedure, and to use this to define a core outcome set (COS). A three-round Delphi procedure was conducted among an international panel of experts in fetal LUTO. The panel was provided with a list of literature-based parameters to consider for the diagnosis, prognosis, management and outcomes of LUTO. A parallel procedure was conducted with patient groups during the development of the COS. A total of 168 experts were approached, of whom 99 completed the first round and 80/99 (80.8%) completed all three rounds of the study questionnaires. Consensus was reached that, in the first trimester, an objective measurement of longitudinal bladder diameter of ≥ 7 mm should be used to suspect LUTO. In the second trimester, imaging parameters suggestive of LUTO could include enlarged bladder, keyhole sign, bladder wall thickening, bilateral hydronephrosis, bilateral hydroureteronephrosis and male sex. There was 79% agreement that the current prognostic scoring systems in the literature should not be used clinically. However, experts agreed on the value of amniotic fluid volume (at < 24 weeks) to predict survival and that the value of fetal intervention is to improve the chance of neonatal survival. Experts endorsed sonographic parameters suggestive of renal dysplasia, at least one vesicocentesis, and renal biochemistry for prognosis and counseling, but these items did not reach a consensus for determining candidacy for fetal intervention. On the other hand, imaging parameters suggestive of LUTO, absence of life-limiting structural or genetic anomalies, gestational age of ≥ 16 weeks and oligohydramnios (defined as deepest vertical pocket < 2 cm) should be used as candidacy criteria for fetal intervention based on expert consensus. If bladder refill was evaluated, it should be assessed subjectively. Vesicoamniotic shunt should be the first line of fetal intervention. In the presence of suspected fetal renal failure, serial amnioinfusion should be offered only as an experimental procedure under research protocols. A COS for future LUTO studies was agreed upon. International consensus on the diagnosis, prognosis and management of fetal LUTO, as well as the COS, should inform clinical care and research to optimize perinatal outcomes. © 2024 The Author(s). Ultrasound in Obstetrics & Gynecology published by John Wiley & Sons Ltd on behalf of International Society of Ultrasound in Obstetrics and Gynecology.

Key factors in the choice of appropriate outcomes for clinical trials.

Health research is the foundation of medical knowledge and healthcare system recommendations. Therefore, choosing appropriate outcomes in studies of therapeutic interventions is a fundamental step in producing evidence and, subsequently, for decision-making. In this article, we propose three key factors for the choice of outcomes: the inclusion of patient-reported outcomes, since they focus on the patient's perception of their health status and quality of life; the consideration of clinically relevant outcomes, which are direct measurements of the patient's health status and, therefore, will be decisive in decision-making; and the use of core outcome sets, a tool that standardizes the measurement and interpretation of outcomes, facilitating the production and synthesis of appropriate evidence for the evidence ecosystem. The correct choice of outcomes will help health decision-makers and clinicians deliver appropriate patient-centered care and optimize the use of resources in healthcare and clinical research.

Development of an International Standard Set of Outcomes and Measurement Methods for Routine Practice for Adults with Epilepsy: The International Consortium for Health Outcomes Measurement Consensus Recommendations.

At present, there is no internationally accepted set of core outcomes or measurement methods for epilepsy clinical practice. Therefore, the International Consortium for Health Outcomes Measurement (ICHOM) convened an international working group of experts in epilepsy, people with epilepsy and their representatives to develop minimum sets of standardized outcomes and outcomes measurement methods for clinical practice that support patient-clinician decision-making and quality improvement. Consensus methods identified 20 core outcomes. Measurement tools were recommended based on their evidence of strong clinical measurement properties, feasibility, and cross-cultural applicability. The essential outcomes included many non-seizure outcomes: anxiety, depression, suicidality, memory and attention, sleep quality, functional status, and the social impact of epilepsy. The proposed set will facilitate the implementation of the use of patient-centered outcomes in daily practice, ensuring holistic care. They also encourage harmonization of outcome measurement, and if widely implemented should reduce the heterogeneity of outcome measurement, accelerate comparative research, and facilitate quality improvement efforts.

Core Outcome Set for Necrotizing Enterocolitis Treatment Trials.

Variability in outcome reporting in necrotizing enterocolitis (NEC) treatment trials hinders conducting meta-analyses and implementing novel treatments. We aimed to develop a core outcome set (COS) for NEC treatment trials including outcome measures most relevant to patients and physicians, from NEC diagnosis to adulthood. Clinicians and/or researchers from low-middle- and high-income countries were approached based on their scientific contributions to NEC literature, and patients and parents through local organizations. We presented participants with 45 outcomes used in NEC research, identified through a systematic review. To achieve consensus, outcomes were rated on a scale of 1 to 9 in 3 online Delphi rounds, and discussed at a final consensus meeting. Seventy-one participants from 25 countries completed all Delphi rounds, including 15 patients and family representatives. Thirteen outcomes reached consensus in one of the stakeholder groups and were included in the consensus meeting, 6 outcomes reached consensus in both groups. Twenty-seven participants from both high- and low-middle-income countries attended the online consensus meeting, including family representatives and NEC patients. After discussion and a final vote, 5 outcomes reached consensus to be included: mortality, NEC-related mortality, short bowel syndrome, quality of life, and neurodevelopmental impairment. This NEC COS includes 5 predominantly long-term outcomes agreed upon by clinicians, patients, and family representatives. Use of this international COS will help standardize outcome selection in clinical trials, ensure these are relevant to those most affected by NEC care, and, ultimately, improve the care of infants with NEC.

Developing Core Outcome Sets for Key Vascular Procedures: A Major Step Forward in Vascular Surgery

Developing Core Outcome Sets for Key Vascular Procedures: A Major Step Forward in Vascular Surgery

Supportive and palliative care needs in advanced non-malignant liver disease: systematic review

Advanced cirrhosis confers a significant symptom burden and has a 50% 2-year mortality rate in those with decompensated disease. There is increasing demand for supportive and palliative care (SAPC) for...

Systematic review of patient and service outcome measures of remote digital technologies for cochlear implant and hearing aid users

IntroductionThe use of telehealth for cochlear implant (CI) and hearing aid service provision has the potential to provide efficient, effective, and equitable services to users. However, clinicians require evidence that remote technologies provide care that is equal, or superior to, standard delivery. There are many outcome measures used across audiology, however there is little consensus for a standardized approach to assessment. This systematic review aims to identify the outcome measures to assess remote technologies for CI and hearing aid users, as a first step in a larger project to develop a core outcome set for remote technologies in CI users.MethodsA systematic search of seven electronic databases was conducted using a search strategy defined by PICOTS for the research question. Eligible studies were in English and published in 2012 onwards. Search strategy, selection, and data collection followed PRISMA 2020 guidelines.ResultsThis systematic literature review of 49 articles revealed over 250 discrete outcomes. Outcome measures were grouped into core areas, outcome domains, and outcome sub-domains. Studies assessing remote technology for CI users assessed significantly more outcomes in the ear and labyrinth domain (43% vs. 10%) and studies assessing remote technology in hearing aid users assessed significantly more outcomes in the cognitive (28% vs. 5%) and emotional (35% vs. 10%) functioning domains. Outcome measures within the auditory functioning domain were also significantly different, with CI studies utilizing more speech perception measures (95% vs. 21%) and hearing aid studies utilizing significantly more self-reported outcome measures (73% vs. 19%).DiscussionThe inclusion of hearing aid studies was to ensure that all key outcome domains used within remote hearing rehabilitation were captured, as well as to compare differences in outcome domains between the two user groups. There were significant differences between studies of remote technologies for CI and hearing aid users. These results will inform the ongoing development of a core outcome set for remote technologies in CI users.Systematic Review Registrationhttps://www.crd.york.ac.uk/prospero/display_record.php?RecordID=370171, identifier: CRD42022370171.

COS-PPA: protocol to develop a core outcome set for primary progressive aphasia

IntroductionThe term primary progressive aphasia (PPA) describes a group of language-led dementias. Disease-modifying treatments that delay, slow or reverse progression of PPA are currently lacking, though a number of interventions...

Factors influencing use and choice of Core Outcome Sets and outcome measurement instruments in trials of interventions to prevent childhood obesity: A survey protocol

Background Two core outcome sets for childhood obesity prevention have been developed; standardised sets of outcome measurement instruments for these core outcome sets are currently being developed. Core outcome sets and standardised measurement sets can reduce heterogeneity and improve evidence syntheses for trials of interventions to prevent childhood obesity and/or interventions to improve child health behaviours related to childhood obesity. Such benefits are only realised if core outcome sets and standardised measurement sets are used in trials. The aims of this study are 1) to examine trialists’ awareness and attitudes towards the two existing core outcome sets and factors influencing their use; 2) to explore the characteristics of outcome measurement instruments that trialists currently use; and 3) to better understand how trialists choose outcome measurement instruments and the factors that influence those choices. Methods A cross-sectional online survey will be conducted with researchers involved in the design and/or conduct of trials of interventions to prevent childhood obesity and/or to improve child health behaviours related to childhood obesity, in children aged 0 to 5 years (trialists). Trialists will be recruited using purposive sampling, and will complete a 22-item survey examining trialist characteristics, awareness of the existing core outcome sets, factors influencing use of the existing core outcome sets, characteristics of measurement instruments, how trialists choose measurement instruments, and factors influencing choice of measurement instrument. Quantitative data will be analysed descriptively; responses to open-ended questions will be analysed using qualitative content analysis. Conclusions Findings from this study will inform approaches to maximising use of core outcome sets and standardised measurement sets for childhood obesity prevention. Use of standardised approaches to what and how outcomes are measured in this area will reduce heterogeneity and research waste and enhance evidence syntheses to better determine intervention effects.

The development of a Core Outcome Set for clinical trials targeting interventions aiming to improve adherence to appropriate polypharmacy in older people – A Delphi consensus study

Abstract Introduction Older people encounter challenges when taking polypharmacy. Different interventions have been applied to improve adherence to polypharmacy. However, there are few high-quality studies, and little consideration has been given to the outcomes selected to measure the effectiveness of interventions.[1] To produce high-quality studies and accurately report adherence-related outcomes, developing a Core Outcome Set (COS) is important.[2] A COS is the minimum number of outcomes that should be reported and measured in future trials in a specific health field to reduce outcomes’ inconsistencies across trials.[2] Aim This study aimed to develop a COS for use in clinical trials aiming at improving adherence to appropriate polypharmacy in older patients. Methods A list of outcomes related to adherence to polypharmacy (n=13) was compiled from previous semi-structured interviews with stakeholders. The Delphi panel consisted of experts in adherence, polypharmacy and gerontology, including journal editors, academics, non-National Health Service doctors, nurses and pharmacists who were identified by searching researchers’ publication profiles using Google Scholar and Scopus. Senior editors and editor-in-chief of peer-reviewed journals were identified by searching journal information. Public members were identified by searching for contact emails of staff members in public charities/organisations representing older people’s healthcare needs (e.g. AgeUK). Additionally, identified participants received invitation emails asking them to forward the email to other participants and experts who might be interested in this study (i.e. snowball sampling). The 13 outcomes were distributed in three online Delphi questionnaires between February and April 2023 using an online SoGoSurvey® platform. All outcomes were rated according to their degree of importance using a nine-point Likert scale (1 to 3 = unimportant; 4 to 6 = important but not critical; 7 to 9 = critical). Following each round, response rates and the distribution of scores were calculated to classify outcomes into ‘consensus in’, ‘consensus out’ and ‘no consensus’. An outcome was considered very important if ≥80% of participants scored it as ‘critical’ and ≤15% scored it as ‘unimportant’. Results The Delphi panel (Round 1, n=57; Round 2, n=53; Round 3, n=50) comprised different professionals, including 38 academics, 23 pharmacists, 12 doctors, six journal editors, three nurses and four public members, some of whom had multiple roles, e.g. doctor and academic. Participants were distributed over four continents, mostly from Europe (n=39). Both Australia and America had identical representation (n=8), while two participants represented Asia. Consensus was reached following the third round on seven outcomes, namely: medication adherence across multiple medications (100.0%), treatment burden (96.2%), health-related quality of life (96.2%), healthcare utilisation (83.0%), adverse events and side effects (83.0%), patient-carer satisfaction (82%), and cost-effectiveness (81.1%). Conclusion Seven outcomes were considered of critical importance to stakeholders. This study is the first to develop a COS for interventions targeting adherence to appropriate polypharmacy in older patients. However, most of our participants were academics and pharmacists from Europe, whilst other participant categories and continents were less well-represented. A future nominal group technique study will be conducted to discuss the results obtained from previous Delphi questionnaires, followed by selecting the appropriate outcome measurement instruments to measure these outcomes.