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OBJECTIVE: COVID-19 would kill fewer people if health programmes can predict who is at higher risk of mortality because resources can be targeted to protect those people from infection. We predict mortality in a very large population in Mexico with machine learning using demographic variables and pre-existing conditions. DESIGN: Cohort study. SETTING: March 2020 to November 2021 in Mexico, nationally represented. PARTICIPANTS: 1.4 million laboratory-confirmed patients with COVID-19 in Mexico at or over 20 years of age. PRIMARY AND SECONDARY OUTCOME MEASURES: Analysis is performed on data from March 2020 to November 2021 and over three phases: (1) from March to October in 2020, (2) from November 2020 to March 2021 and (3) from April to November 2021. We predict mortality using an ensemble machine learning method, super learner, and independently estimate the adjusted mortality relative risk of each pre-existing condition using targeted maximum likelihood estimation. RESULTS: Super learner fit has a high predictive performance (C-statistic: 0.907), where age is the most predictive factor for mortality. After adjusting for demographic factors, renal disease, hypertension, diabetes and obesity are the most impactful pre-existing conditions. Phase analysis shows that the adjusted mortality risk decreased over time while relative risk increased for each pre-existing condition. CONCLUSIONS: While age is the most important predictor of mortality, younger individuals with hypertension, diabetes and obesity are at comparable mortality risk as individuals who are 20 years older without any of the three conditions. Our model can be continuously updated to identify individuals who should most be protected against infection as the pandemic evolves.

IntroductionSarcoidosis is a pulmonary and systemic granulomatous disease with a wide range of potential outcomes, from spontaneous resolution to end-stage organ damage and death. Currently, clinicians have no easy-to-use risk stratification tools for important clinical outcomes in sarcoidosis, such as progressive lung disease. This study will address two clinical practice needs: (1) development of a risk calculator that provides an estimate of the likelihood of pulmonary progression in sarcoidosis patients during the follow-up period and (2) determine the optimal interval for serial clinical monitoring (eg, 6, 12, 18 months) using these risk prediction tools.Methods and analysisThe Risk Indicators of Sarcoidosis Evolution-Unified Protocol study is a National Institutes of Health-sponsored, longitudinal observational study of adults with pulmonary sarcoidosis who will be enrolled at five US tertiary care centres. Participants will be evaluated at approximately 6-month intervals for up to 60 months with collection of lung function, blood samples and clinical data. The target sample size is 557 and the primary objective is to determine which clinical features measured during a routine clinic visit carry the most prognostic information for predicting clinical progression of pulmonary sarcoidosis over the follow-up period. The primary outcome measure will be quantified by a clinically meaningful change in forced vital capacity, forced expiratory volume in 1 s or diffusing capacity of the lung for carbon monoxide. The secondary objective is to determine if blood biomarkers measured during a routine clinic visit can improve the risk assessment modelling for progression of pulmonary sarcoidosis over the follow-up period.Ethics and disseminationThe study protocol has been approved by the Institutional Review Boards at each centre and the reliance Institutional Review Board overseeing the study (WCG, Protocol #20222400). Participants will provide informed consent prior to enrolment. Results will be disseminated via publication in a relevant peer-reviewed journal.Trial registration numberNCT05567133.

Purpose Bar charts of numerical data, often known as dynamite plots, are unnecessary and misleading. Their tendency to alter the perception of mean's position through the within-the-bar bias and their lack of information on the distribution of the data are two of numerous reasons. The machine learning tool, Barzooka, can be used to rapidly screen for different graph types in journal articles. We aim to determine the proportion of original research articles using dynamite plots to visualize data, and whether there has been a change in their use over time. Methods Original research articles in nine surgical fields of research were sampled based on MeSH terms and then harvested using the Python-based biblio-glutton-harvester tool. After harvesting, they were analysed using Barzooka. Over 40 000 original research articles were included in the final analysis. The results were adjusted based on previous validation data with 95% confidence bounds. Kendall τ coefficient with the Mann–Kendall test for significance was used to determine the trend of dynamite plot use over time. Results Eight surgical fields of research showed a statistically significant decrease in use of dynamite plots over 10 years. Oral and maxillofacial surgery showed no significant trend in either direction. In 2022, use of dynamite plots, dependent on field and 95% confidence bounds, ranges from ~30% to 70%. Conclusion Our results show that the use of dynamite plots in surgical research has decreased over time; however, use remains high. More must be done to understand this phenomenon and educate surgical researchers on data visualization practices.   What is already known on this topic   Although it is well established that dynamite plots are misleading data visualizations for numerical data, the frequency of their use in surgical research is yet to be investigated. What this study adds   Following this study, we now know that dynamite plots are frequently used in most fields of surgical research. Use over time is decreasing; however, more must be done. How this study might affect research, practice or policy   This study further reiterates the importance of good data visualizations in research. It also emphasizes the need for educational initiatives for surgeons involved in research, as well as the need for journal policies that encourage better data visualization practices. [ABSTRACT FROM AUTHOR]

IntroductionThe burden of Alzheimer's disease (AD) and AD-related dementias (ADRD) is increasing nationally and globally, with disproportionate impacts on lower-income, lower education and systematically marginalised older adults. Presence of inequalities in neighbourhood factors (eg, social context, physical and built environments) may affect risk of cognitive decline and be key for intervening on AD/ADRD disparities at the population level. However, existing studies are limited by a dearth of longitudinal, detailed neighbourhood measures linked to rich, prospective cohort data. Our main objective is to identify patterns of neighbourhood change related to prevalence of-and disparities in-cognitive decline and dementia.Methods and analysesWe describe the process of collecting, processing and linking extensive neighbourhood data to the Multi-Ethnic Study of Atherosclerosis (MESA), creating a 25+ years dataset. Within the MESA parent study, the MESA Neighborhoods and Aging cohort study will characterise dynamic, longitudinal neighbourhood social and built environment variables relevant to cognition for residential addresses of MESA participants. This includes administering new surveys, expanding residential address histories, calculating new measures derived from spatial data and implementing novel deep learning algorithms on street-level imagery. Applying novel statistical techniques, we will examine associations of neighbourhood environmental characteristics with cognition and clinically relevant AD/ADRD outcomes. We will investigate determinants of disparities in outcomes by socioeconomic position and race/ethnicity and assess the contribution of neighbourhood environments to these disparities. This project will provide new evidence about pathways between neighbourhood environments and cognitive outcomes, with implications for policies to support healthy ageing.Ethics and disseminationThis project was approved by the University of Washington and Drexel University Institutional Review Boards (protocols #00009029 and #00014523, and #180900605). Data will be distributed through the MESA Coordinating Center. Findings will be disseminated in peer-reviewed scientific journals, briefs, presentations and on the participant website.

BackgroundOlder multimorbid adults have a high risk of mortality and a short life expectancy (LE). Providing high-value care and avoiding care overuse, including of preventive care, is a serious challenge among multimorbid patients. While guidelines recommend to tailor preventive care according to the estimated LE, there is no tool to estimate LE in this specific population. Our objective is therefore to develop an LE estimator for older multimorbid adults by transforming a mortality prognostic index, which will be developed and internally validated in a prospective cohort.Methods and analysisWe will analyse data of the Optimising Therapy to Prevent Avoidable Hospital Admissions in Multimorbid Older People cohort study in Bern, Switzerland. 822 participants were included at hospitalisation with age of 70 years or older, multimorbidity (three or more chronic medical conditions) and polypharmacy (use of five drugs or more for >30 days). All-cause mortality will be assessed during 3 years of follow-up. We will apply a flexible parametric survival model with backward stepwise selection to identify the mortality risk predictors. The model will be internally validated using bootstrapping techniques. We will derive a point-based risk score from the regression coefficients. We will transform the 3-year mortality prognostic index into an LE estimator using the Gompertz survival function. We will perform a qualitative assessment of the clinical usability of the LE estimator and its application. We will conduct the development and validation of the mortality prognostic index following the Prognosis Research Strategy (PROGRESS) framework and report it following the Transparent Reporting of a Multivariable Prediction Model for Individual Prognosis or Diagnosis (TRIPOD) statement.Ethics and disseminationWritten informed consent by patients themselves or, in the case of cognitive impairment, by a legal representative, was required before enrolment. The local ethics committee (Kantonale Ethikkommission Bern) has approved the study. We plan to publish the results in peer-reviewed journals and present them at national and international conferences.

ObjectivesPeople who inject drugs (PWID) play an integral role in facilitating the entry of others into injection drug use (IDU). We sought to assess factors influencing PWID in providing IDU initiation assistance across three distinct North American settings and to generate pooled measures of risk.DesignWe employed data from three PWID cohort studies participating in PReventing Injecting by Modifying Existing Responses (PRIMER), for this cross-sectional analysis.SettingTijuana, Mexico; San Diego, USA; Vancouver, Canada.ParticipantsA total of 2944 participants were included in this study (Tijuana: n=766, San Diego: n=353, Vancouver: n=1825).MeasurementsThe outcome was defined as recently (ie, past 6 months) assisting in an IDU initiation event. Independent variables of interest were identified from previous PRIMER analyses. Site-specific multiple modified Poisson regressions were fit. Pooled relative risks (pRR) were calculated and heterogeneity across sites was assessed via linear random effects models.ResultsEvidence across all three sites indicated that having a history of providing IDU initiation assistance (pRR: 4.83, 95% CI: 3.49 to 6.66) and recently being stopped by law enforcement (pRR: 1.49, 95% CI: 1.07 to 2.07) were associated with a higher risk of providing assistance with IDU initiation; while recent opioid agonist treatment (OAT) enrolment (pRR: 0.64, 95% CI: 0.43 to 0.96) and no recent IDU (pRR: 0.21, 95% CI: 0.07 to 0.64) were associated with a lower risk. We identified substantial differences across site in the association of age (I2: 52%), recent housing insecurity (I2: 39%) and recent non-injection heroin use (I2: 78%).ConclusionWe identified common and site-specific factors related to PWID's risk of assisting in IDU initiation events. Individuals reporting a history of assisting IDU initiations, being recently stopped by law enforcement, and recently injecting methamphetamine/speedball were more likely to have recently assisted an IDU initiation. Whereas those who reported not recently engaging in IDU and those recently enrolled in OAT were less likely to have done so. Interventions and harm reduction strategies aimed at reducing the harms of IDU should incorporate context-specific approaches to reduce the initiation of IDU.

ObjectiveTo assess existing knowledge related to methodological considerations for linking population-based surveys and health facility data to generate effective coverage estimates. Effective coverage estimates the proportion of individuals in need of an intervention who receive it with sufficient quality to achieve health benefit.DesignSystematic review of available literature.Data sourcesMedline, Carolina Population Health Center and Demographic and Health Survey publications and handsearch of related or referenced works of all articles included in full text review. The search included publications from 1 January 2000 to 29 March 2021.Eligibility criteriaPublications explicitly evaluating (1) the suitability of data, (2) the implications of the design of existing data sources and (3) the impact of choice of method for combining datasets to obtain linked coverage estimates.ResultsOf 3805 papers reviewed, 70 publications addressed relevant issues. Limited data suggest household surveys can be used to identify sources of care, but their validity in estimating intervention need was variable. Methods for collecting provider data and constructing quality indices were diverse and presented limitations. There was little empirical data supporting an association between structural, process and outcome quality. Few studies addressed the influence of the design of common data sources on linking analyses, including imprecise household geographical information system data, provider sampling design and estimate stability. The most consistent evidence suggested under certain conditions, combining data based on geographical proximity or administrative catchment (ecological linking) produced similar estimates to linking based on the specific provider utilised (exact match linking).ConclusionsLinking household and healthcare provider data can leverage existing data sources to generate more informative estimates of intervention coverage and care. However, existing evidence on methods for linking data for effective coverage estimation are variable and numerous methodological questions remain. There is need for additional research to develop evidence-based, standardised best practices for these analyses.

Patient-reported outcomes (PROs) are used in clinical trials to provide valuable evidence on the impact of disease and treatment on patients' symptoms, function and quality of life. High-quality PRO data from trials can inform shared decision-making, regulatory and economic analyses and health policy. Recent evidence suggests the PRO content of past trial protocols was often incomplete or unclear, leading to research waste. To address this issue, international, consensus-based, PRO-specific guidelines were developed: the Standard Protocol Items: Recommendations for Interventional Trials (SPIRIT)-PRO Extension. The SPIRIT-PRO Extension is a 16-item checklist which aims to improve the content and quality of aspects of clinical trial protocols relating to PRO data collection to minimise research waste, and ultimately better inform patient-centred care. This SPIRIT-PRO explanation and elaboration (E&E) paper provides information to promote understanding and facilitate uptake of the recommended checklist items, including a comprehensive protocol template. For each SPIRIT-PRO item, we provide a detailed description, one or more examples from existing trial protocols and supporting empirical evidence of the item's importance. We recommend this paper and protocol template be used alongside the SPIRIT 2013 and SPIRIT-PRO Extension paper to optimise the transparent development and review of trial protocols with PROs.

ObjectivesFollowing well-established practices in demography, this article discusses several measures based on the number of COVID-19 deaths to facilitate comparisons over time and across populations.SettingsNational populations in 186 United Nations countries and territories and populations in first-level subnational administrative entities in Brazil, China, Italy, Mexico, Peru, Spain and the USA.ParticipantsNone (death statistics only).Primary and secondary outcome measuresAn unstandardised occurrence/exposure rate comparable to the Crude Death Rate; an indirectly age-and-sex standardised rate that can be derived even when the breakdown of COVID-19 deaths by age and sex required for direct standardisation is unavailable; the reduction in life expectancy at birth corresponding to the 2020 number of COVID-19 deaths.ResultsTo date, the highest unstandardised rate has been in New York, at its peak exceeding the state 2017 crude death rate. Populations compare differently after standardisation: while parts of Italy, Spain and the USA have the highest unstandardised rates, parts of Mexico and Peru have the highest standardised rates. For several populations with the necessary data by age and sex for direct standardisation, we show that direct and indirect standardisation yield similar results. US life expectancy is estimated to have declined this year by more than a year (-1.26 years), far more than during the worst year of the HIV epidemic, or the worst 3 years of the opioid crisis, and to reach its lowest level since 2008. Substantially larger reductions, exceeding 2 years, are estimated for Panama, Peru, and parts of Italy, Spain, the USA and especially, Mexico.ConclusionsWith lesser demand on data than direct standardisation, indirect standardisation is a valid alternative to adjust international comparisons for differences in population distribution by sex and age-groups. A number of populations have experienced reductions in 2020 life expectancies that are substantial by recent historical standards.

ObjectivesTo determine the proportion of patients with extremity sarcoma who would be willing to participate in a clinical trial in which they would be randomised to one of four different postoperative sarcoma surveillance regimens. Additionally, we assessed patients' perspectives on the burden of cancer care, factors that influence comfort with randomisation and the importance of cancer research.DesignProspective, cross-sectional patient survey.SettingOutpatient sarcoma clinics in Canada, the USA and Spain between May 2017 and April 2020. Survey data were entered into a study-specific database.ParticipantsPatients with extremity sarcoma who had completed definitive treatment from seven clinics across Canada, the USA and Spain.Main outcome measuresThe proportion of patients with extremity sarcoma who would be willing to participate in a randomised controlled trial (RCT) that evaluates varying postoperative cancer surveillance regimens.ResultsOne hundred thirty complete surveys were obtained. Respondents reported a wide range of burdens related to clinical care and surveillance. The majority of patients (85.5%) responded that they would agree to participate in a cancer surveillance RCT if eligible. The most common reason to participate was that they wanted to help future patients. Those that would decline to participate most commonly reported that participating in research would be too much of a burden for them at a time when they are already feeling overwhelmed. However, most patients agreed that cancer research will help doctors better understand and treat cancer.ConclusionsThese results demonstrate that most participants would be willing to participate in an RCT that evaluates varying postoperative cancer surveillance regimens. Participants' motivation for trial participation included altruistic reasons to help future patients and deterrents to trial participation included the overwhelming burden of a cancer diagnosis. These results will help inform the development of patient-centred RCT protocols in sarcoma surveillance research.Level of evidenceV.

IntroductionOnly international studies can provide the full variability of built environments and accurately estimate effect sizes of relations between contrasting environments and health-related outcomes. The aims of the International Physical Activity and Environment Study of Adolescents (IPEN Adolescent) are to estimate the strength, shape and generalisability of associations of the community environment (geographic information systems (GIS)-based and self-reported) with physical activity and sedentary behaviour (accelerometer-measured and self-reported) and weight status (normal/overweight/obese).Methods and analysisThe IPEN Adolescent observational, cross-sectional, multicountry study involves recruiting adolescent participants (ages 11-19 years) and one parent/guardian from neighbourhoods selected to ensure wide variations in walkability and socioeconomic status using common protocols and measures. Fifteen geographically, economically and culturally diverse countries, from six continents, participated: Australia, Bangladesh, Belgium, Brazil, Czech Republic, Denmark, Hong Kong SAR, India, Israel, Malaysia, New Zealand, Nigeria, Portugal, Spain and USA. Countries provided survey and accelerometer data (15 countries), GIS data (11), global positioning system data (10), and pedestrian environment audit data (8). A sample of n=6950 (52.6% female; mean age=14.5, SD=1.7) adolescents provided survey data, n=4852 had 4 or more 8+ hours valid days of accelerometer data, and n=5473 had GIS measures. Physical activity and sedentary behaviour were measured by waist-worn ActiGraph accelerometers and self-reports, and body mass index was used to categorise weight status.Ethics and disseminationEthical approval was received from each study site's Institutional Review Board for their in-country studies. Informed assent by adolescents and consent by parents was obtained for all participants. No personally identifiable information was transferred to the IPEN coordinating centre for pooled datasets. Results will be communicated through standard scientific channels and findings used to advance the science of environmental correlates of physical activity, sedentary behaviour and weight status, with the ultimate goal to stimulate and guide actions to create more activity-supportive environments internationally.

IntroductionAs new topical and systemic treatments become available for atopic dermatitis (AD), there is a need to understand how treatments are being used in routine clinical practice, their comparative effectiveness and their long-term safety in diverse clinical settings.Methods and analysisThe TARGET-DERM AD cohort is a longitudinal, observational study of patients with AD of all ages, designed to provide practical information on long-term effectiveness and safety unobtainable in traditional registration trials. Patients with physician-diagnosed AD receiving prescription treatment (topical or systemic) will be enrolled at academic and community clinical centres. Up to 3 years of retrospective medical records, 5 years of prospective medical records, and optional biological samples and patient-reported outcomes will be collected. The primary aims include characterisation of AD treatment regimens, evaluation of response to therapy, and description of adverse events.Ethics and disseminationTARGET-DERM has been approved by a central IRB (Copernicus Group IRB, 5000 Centregreen Way Suite 200, Cary, North Carolina 27513) as well as local and institutional IRBs. No additional Ethics Committee reviews. Results will be reviewed by a publications committee and submitted to peer-reviewed journals.Trial registration numberNCT03661866, pre-results.

ObjectivesPreterm births (PTBs) represent significant health risks, and several studies have found associations between high outdoor temperatures and PTB. We estimated both the total and natural direct effects (independent of particulate matter, ozone and nitrogen dioxide air pollutants) of the prior 2-day mean apparent temperature (AT) on PTB. We evaluated effect modification by maternal age, race, education, smoking status and prenatal care.Design and settingWe obtained birth records and meteorological data for the Detroit, Michigan, USA area, for the warm months (May to September), 1991 to 2001. We used a time series Poisson regression with splines of AT, wind speed, solar radiation and citywide average precipitation to estimate total effects. To accommodate multiple mediators and exposure-mediator interactions, AT inverse odds weights, predicted by meteorological and air pollutant covariates, were added in a subsequent model to estimate direct effects.ResultsAt 24.9°C relative to 18.6°C, 10.6% (95% CI: 3.8% to 17.2%) of PTBs were attributable to the total effects of AT, and 10.4% (95% CI: 2.2% to 17.5%) to direct effects. Relative excess risks of interaction indicated that the risk of PTB with increasing temperature above 18.6°C was significantly lower among black mothers and higher among mothers less than 19, older than 30, with late or no prenatal care and who smoked.ConclusionThis additional evidence of a direct association between high temperature and PTB may motivate public health interventions to reduce extreme heat exposures among pregnant women, particularly among those who may have enhanced vulnerability.

The essential principles of statistics as applied to surveys, studies, sampling, epidemiology, screening, and trials are described and explained. [ABSTRACT FROM AUTHOR]

Introduction: Climate change impacts the circumpolar region (including northern Canada) at a greater magnitude than other parts of the world. This affects food (in)security as well as food sovereignty. This scoping review aims to map the methods of measuring food (in)security and food sovereignty across northern Canada and the circumpolar region in support of the Yukon Government's climate change adaptation strategy., Methods and Analysis: We will adhere to the Preferred Reporting Items for Systematic Reviews and Meta-Analyses extension for Scoping Reviews, and work will be conducted according to the Joanna Briggs Institute (JBI) manual chapter on scoping reviews. Academic librarians develop the academic literature and grey literature search strategies, and the search strategies are further revised through iterative stages of peer review. The search strategy includes 7 academic literature databases, 11 grey literature databases, over 50 websites and the University of Toronto Libraries catalogue. Covidence, an evidence synthesis software, will be used for screening and extraction. The extraction chart will be developed and piloted by our team. A minimum of two reviewers will conduct screening, and conflicts will be resolved through discussion. Data will be extracted by one reviewer and verified by a second. Conflicts will be resolved through discussion or by a third reviewer., Ethics and Dissemination: This project does not require ethical approval as it is secondary research; data will be extracted from published academic research papers, dissertations, and publicly available reports and documents. Our dissemination plan includes presentations at conferences, submission to international peer-reviewed journals and a workshop on the search strategies., Competing Interests: Competing interests: None declared., (© Author(s) (or their employer(s)) 2024. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.)

Objectives: Target trial emulation is an approach that is increasingly used to improve transparency in observational studies and help mitigate biases. For studies declaring that they emulated a target trial, we aimed to evaluate the specification of the target trial, examine its consistency with the observational emulation and assess the risk of bias in the observational analysis., Design: Methodological systematic review reported in accordance with the Preferred Reporting Items for Systematic Reviews and Meta-Analyses statement., Data Sources: The database MEDLINE (Medical Literature Analysis and Retrieval System Online) was interrogated for all studies published from 1 January 2021 to 3 July 2022. We performed an additional manual search of 20 general medical and specialised journals that spanned the same period., Eligibility Criteria: All studies that declared emulating a hypothetical or real randomised trial were eligible., Data Extraction and Synthesis: Two independent reviewers performed the whole systematic review process (screening and selection of studies, data extraction and risk of bias assessment). The main outcomes were the definition of the key protocol components of the target trial and its emulation, consistency between the target trial and its emulation and risk of bias according to the ROBINS-I (Risk Of Bias In Non-randomised Studies - of Interventions) tool., Results: Among the selected sample of 100 studies, 24 (24%) did not specify the target trial. Only 40 studies (40%) provided detailed information on all components of the target trial protocol. Eligibility criteria, intervention strategies and outcomes were consistent between the target trial and its emulation in 35 studies (46% of those specifying the target trial). Overall, 28 studies (28%) exhibited serious risk of bias and 41 (41%) had misalignments in the timing of eligibility assessment, treatment assignment and the start of follow-up (time-zero). As compared with studies that did not specify the target trial, those that did specify the trial less frequently seemed to have both time-zero issues (39% vs 52%) and serious risk of bias (26% vs 33%)., Conclusions: One-quarter of studies declaring that they emulated a target trial did not specify the trial. Target trials and their emulations were particularly inconsistent for studies emulating a real randomised trial. Risk of methodological issues seemed lower in observational analyses that specified versus did not specify the target trial., Competing Interests: Competing interests: All authors have completed the ICMJE uniform disclosure form at www.icmje.org/disclosure-of-interest/ and declare that they have no competing interest related to the study. GLM declares consulting fees from Synapse Medicine, unrelated to the study., (© Author(s) (or their employer(s)) 2024. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.)

Objective: Pilot and feasibility studies are intended to ensure that subsequent randomised controlled trials (RCTs) are feasible, economical and rigorous, especially in a challenging research environment such as emergency medicine (EM). We aimed to evaluate the methodological quality in conducting and reporting randomised pilot and feasibility studies in the EM literature and propose recommendations to improve their quality., Design: Methodological systematic review., Data Sources and Eligibility: We searched MEDLINE and Embase (2018-29 September 2023) for pilot or feasibility RCTs published as full texts in the five top-ranked and other first-quartile EM journals according to Scimago., Data Extraction and Analysis: We assessed their methodological features and reporting quality primarily based on the Consolidated Standards of Reporting Trials (CONSORT) extension., Results: A total of 24 randomised trials identified as pilot (n=13), feasibility (n=3) or both (n=8) were included. At least one feasibility outcome was assessed in 9 trials (feasibility trials), while 15 others only focused on treatment efficacy (efficacy trials). Only three (12.5%) studies progressed to the main trials. Among 12 feasibility trials, 55.6% reported their outcomes with uncertainty estimates, and 33.3% had clear progression criteria. Efficacy trials tended to draw clinical implications on their results. Studies from the five top-ranked journals had better methodological and reporting quality than those from other first-quartile journals., Conclusion: Main methodological concerns for pilot and feasibility studies in first-quartile EM literature include misconceptions, misuses and suboptimal design and reporting quality. These issues were more prominent in lower-ranked first-quartile journals. Our findings highlight the need for resources and training for researchers, journal editors and peer reviewers on the value, objectives and appropriate conduct of pilot and feasibility studies. The conceptual framework and standardised methodological components should be emphasised. EM journals should reinforce the reporting standards and support their publication. These actions can lead to more methodologically rigorous pilot and feasibility studies in EM., Prospero Registration Number: CRD42023468437., Competing Interests: Competing interests: None declared., (© Author(s) (or their employer(s)) 2024. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.)

Objective: The objective of this study is to examine the feasibility of using survey data to identify factors that predict commute mode choice., Design: The study design is cross-sectional., Setting: Survey data from the Finnish Public Sector study (2020) were used., Participants: 42 574 public sector employees, of whom 10 983 were selected for the final sample. These included employees with 5 km or less commuting distances and those working full-time onsite or partly remotely. The mean age was 46 (SD 11) years, and 84% were women., Primary Outcomes: Commute by (1) bike or foot (an active mode) during summer and winter weather and (2) by car (a passive mode) during summer and winter weather., Methods: Using logistic Lasso (least-absolute-shrinkage-and-selection-operator) regression, we developed and tested a prediction model for short commutes of 5 km or less to identify the characteristics of employees most likely to commute actively during summer and winter weather and passively during summer and winter weather., Results: All models had a good predictive ability with a C-index of 0.82, 0.77, 0.72 and 0.71. Cycling and walking during summer weather were predicted by shorter commutes, higher physical activity, lower body mass index (BMI), female sex and higher team psychological safety. Predictors of cycling and walking during winter weather were shorter commute length, higher physical activity, lower BMI and higher age. Commuting by car during summer weather was predicted by longer journey length, higher BMI, lower physical activity, male sex and having children 7-18 years old living at home. Predictors of driving during winter weather were almost identical, but the male sex was replaced by having a spouse., Conclusions: We identified the correlates of active and passive commute choice in different weather conditions with eight variables. This information can be used to develop and target interventions to promote sustainable and healthy commuting modes., Competing Interests: Competing interests: None declared., (© Author(s) (or their employer(s)) 2024. Re-use permitted under CC BY. Published by BMJ.)

Background: Clinical trials are fundamental to healthcare, however, they also contribute to anthropogenic climate change. Following previous work to develop and test a method and guidance to calculate the carbon footprint of clinical trials, we have now applied the guidance to 10 further UK and international, academically sponsored clinical trials to continue the identification of hotspots and opportunities for lower carbon trial design., Methods: 10 collaborating clinical trial units (CTUs) self-identified and a trial was selected from their portfolio to represent a variety of designs, health areas and interventions. Trial activity data was collated by trial teams across 10 modules spanning trial setup through to closure, then multiplied by emission factors provided in the guidance to calculate the carbon footprint. Feedback was collected from trial teams on the process, experience and ease of use of the guidance., Results: We footprinted 10 trials: 6 investigational medicinal product trials, 1 nutritional, 1 surgical, 1 health surveillance and one complex intervention trial. Six of these were completed and four ongoing (two in follow-up and two recruiting). The carbon footprint of the 10 trials ranged from 16 to 765 tonnes CO 2 e. Common hotspots were identified as CTU emissions, trial-specific patient assessments and trial team meetings and travel. Hotspots for specific trial designs were also identified. The time taken to collate activity data and complete carbon calculations ranged from 5 to 60 hours. The draft guidance was updated to include new activities identified from the 10 trials and in response to user feedback., Discussion: There are opportunities to reduce the impact of trials across all modules, particularly trial-specific meetings and travel, patient assessments and laboratory practice. A trial's carbon footprint should be considered at the design stage, but work is required to make this common place., Competing Interests: Competing interests: JG, LF, JB and PRW have received NIHR funding to undertake this study (support for the manuscript). FA is the paid chair of the Sustainable Healthcare Coalition. RA-SS reports clinical study grants from the British Heart Foundation and Chief Scientist Office Health Improvement, Protection and Services Research Committee Project Grant; consulting fees from Recursion Pharmaceuticals and Bioxodes, and payment from European Stroke Masters (European Stroke Organisation). RA-SS participates on the Novo Nordisk NN9931-4553 and NN9931-4554 endpoint adjudication committee and is the clinical director of the UK Clinical Research Collaboration network of registered Clinical Trials Units. CA is the Vice President of the World Stroke Organisation, Editor-in-chief of Cerebrovascular Diseases and Associate Editor of International Journal of Stroke; reports grants or contracts from National Health and Medical Research Council (NHMRC) of Australia, Medical Research Council (MRC) of the UK, Medical Research Foundation (MRF) of the UK, Penumbra and Takeda; consulting fees from AstraZeneca Advisory Board and participation on Dutch DIST trial. CG is supported by the NIHR CTU efficiency call. KG receives consulting fees from Boehringer & Ingelheim (unrelated to this work) and is Chair of the ICTMC 2024 Scientific Committee. KH is the Deputy Chair of the NIHR Research Professors panel and was previously a member of the NIHR HTA General Committee. CN is an unpaid information specialist in a priority setting partnership for adults living with type 1 diabetes. SP reports grants or contracts from ViiV Healthcare, Gilead Sciences, NIH, MRC, EDCTP and Janssen-Cilag and is a DSMB member for the academic NIHR–funded trial TIPAL. SO’R, LM and JM are supported by funding from Health Research Board (HRB) Ireland MRSy is an independent unpaid member of many Independent Data Monitoring Committees for academic sponsors; reports grants or contracts from Astellas, Clovis Oncology, Janssen, Pfizer, Novartis and Sanofi-Aventis; consulting fees from Eli Lilly; speaker fees from Janssen and AstraZeneca, and payment from Eisai, all unrelated to the topic of this comment. EM, AB, DC, FY, LW, XC, CM, PD, NM, AP, KM, EB, FD and CK report no competing interests., (© Author(s) (or their employer(s)) 2024. Re-use permitted under CC BY. Published by BMJ.)

Introduction: Clean intermittent catheterisation (CIC) is the standard of care for treating neurogenic lower urinary tract dysfunction (NLUTD), the most common bladder dysfunction in children diagnosed with spinal dysraphism (SD) and spinal cord injury (SCI). Failure to follow the prescribed CIC regimen results in urinary tract infections, incontinence and renal insufficiency. Adherence to CIC is suboptimal, with reported non-adherence rates of 18%-66%. Despite the efficacy of CIC, the research on CIC adherence is not well defined in the literature and even less for caregivers of children on CIC protocols., Methods: This proposed study aims to identify caregiver CIC adherence and determinants while exploring the personal experiences of performing CIC from the perspective of caregivers of children with NLUTD due to SD and SCI. This cross-sectional, correlational, convergent mixed methods study design in which qualitative and quantitative data will be collected simultaneously will be used to study the level of adherence and the relationship of caregiver determinants to CIC in children with SD and SCI and adherence to the CIC protocol. Convenience sampling will be used to identify 60 adult caregivers who can read and write English or Spanish and have a child diagnosed with SD and SCI who is currently prescribed CIC by a urology provider., Analysis: The adherence data will be reported as frequency and percentages. A correlation analysis will be computed to assess the association between determinants measured by the Clean Intermittent Catheterization-Caregiver Questionnaire and adherence levels measured with the Intermittent Catheterization Adherence Scale. Thematic analysis will be used to analyse and interpret the interview data. A comparison joint display will be developed to compare quantitative and qualitative data results., Ethical and Dissemination: Institutional review board approval was obtained from the Children's Mercy Kansas City (Study00003003) and the University of Missouri-Kansas City (#2100185). The study's main results will be disseminated to caregiver participants, published in peer-reviewed journals and presented at conferences., Competing Interests: Competing interests: None declared., (© Author(s) (or their employer(s)) 2024. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.)

Introduction: Youth participatory evaluation is one model for monitoring global outcomes and assessing interventions to improve young people's health equity and well-being while embracing principles of participation and empowerment. Little is known about the use of this approach in practise. This scoping review will identify and synthesise descriptions of how youth participatory evaluation is enacted, to what extent it occurs, and describe the relationship between context and inclusion., Methods and Analysis: Scoping review methods will adhere to those outlined by Arksey and O'Malley. The study will also follow the Preferred Reporting Items for Systematic Reviews and Meta-Analyses Extension for Scoping Reviews. The review will use publicly available evaluation reports (grey literature) for programmes funded by the US National Science Foundation through the Advancing Informal science, technology, engineering and mathematics (STEM) Learning programme and whose reports are archived in the repository hosted by the Reimagining Equity and Values in Informal STEM education (REVISE) Center. This scoping review is limited to education, one of the domains of the social determinants of health, more precisely STEM education, due to the report publication parameters set by the REVISE Center repository. A research team member will download citations for and PDFs of reports. These citations and reports will be managed using Zotero and exported to Covidence, a web-based program designed to manage systematic and scoping reviews. Evaluation report selection will occur in a two-step process by trained coders with clear criteria. Inclusion criteria will include: (1) report is for an evaluation study; (2) evaluation has a focus on young people, aged 10-24; (3) evaluation is for a programme serving young people, aged 10-24; and (4) report written and uploaded to the REVISE Center repository between 2017 and 2022. All reports hosted on the REVISE Center repository are based in the USA and written in English. Data charting will also be done by trained coders and facilitated by Covidence and a codebook. Several procedures will be used to uphold rigour and consistency during this process. Data analysis will be done with Dedoose., Ethics and Dissemination: Human subjects research approval will not be required. This scoping review will rely on publicly available evaluation reports. No human research participants will be involved in this review. Findings will be shared through dissemination strategies, such as peer-reviewed journals, international and national conferences, and social media affiliated with academic institutions and professional associations., Study Registration: This study is preregistered on Open Science Framework (https://osf.io/23jdx/). Registration DOI: https://doi.org/10.17605/OSF.IO/K6J98., Competing Interests: Competing interests: None declared., (© Author(s) (or their employer(s)) 2024. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.)

Introduction: Pilot or feasibility trials examine the feasibility, viability and recruitment potential of larger, main trials. Specifically, a pilot trial can be instrumental in identifying methodological issues essential to the development of an effective research protocol. However, numerous studies published as pilot or feasibility studies have demonstrated notable inconsistencies in the nature of information reported, resulting in poor-quality and incomplete reporting. It is unclear whether such low quality or incompleteness of reporting is also prevalent in arthroplasty pilot trials., Methods and Analysis: This protocol outlines a methodological survey examining the completeness of reporting among hip and knee arthroplasty pilot trials in accordance with the Consolidated Standards of Reporting Trials (CONSORT) 2010 extension to pilot trials. Secondary objectives include: (1) determining the prevalence of 'spin' practices, defined as: (a) placing a focus on statistical significance rather than feasibility, (b) presenting results that show the trial to be non-feasible as feasible or (c) emphasising the effectiveness or potential intervention benefits rather than feasibility; (2) determining factors associated with incomplete reporting, and 'spin'. A search of PubMed will be conducted for pilot trials in hip or knee arthroplasty published between 01 January 2017 and 31 December 2023. Following screening, appropriate data will be extracted from eligible publications and reported as descriptive statistics, encompassing elements of the CONSORT checklist associated with completeness of reporting. Logistic regression analysis and Poisson regression will be used to analyse factors associated with completeness of reporting and spin., Ethics and Dissemination: This methodological review does not require formal ethical approval, as it will solely involve the use of published and publicly reported literature. The results of this study will be disseminated through submission to peer-reviewed journals and academic conference presentations. Study details will be sent to McMaster University's media coordinators to be shared through the institution's research-focused platforms., Competing Interests: Competing interests: None declared., (© Author(s) (or their employer(s)) 2024. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.)

Objective: We assessed the association of early statin initiation with inpatient mortality among hospitalised COVID-19 patients., Design, Setting and Participants: This observational study emulated a hypothetical target trial using electronic health records data from Northwestern Medicine Health System, Illinois, 2020-2022. We included patients who were ≥40 years, admitted ≥48 hours for COVID-19 from March 2020 to August 2022 and had no evidence of statin use before admission., Interventions: Individuals who initiated any statins within 48 hours of admission were compared with individuals who did not initiate statins during this period., Primary Outcome Measures: Inpatient mortality at hospital days 7, 14, 21 and 28 were determined using hospital records. Risk differences between exposure groups were calculated using augmented inverse propensity weighting (AIPW) with SuperLearner., Results: A total of 8893 individuals (24.5% early statin initiators) were included. Early initiators tended to be older, male and have higher comorbidity burdens. Unadjusted day 28 mortality was higher in early initiators (6.0% vs 3.6%). Adjusted analysis showed slightly higher inpatient mortality risk at days 7 (RD: 0.5%, 95% CI: 0.2 to 0.8) and 21 (RD: 0.6%, 95% CI: 0.04 to 1.1), but not days 14 (RD: 0.4%, 95% CI: -0.03 to 0.9) and 28 (RD: 0.4%, 95% CI: -0.2 to 1.1). Sensitivity analyses using alternative modelling approaches showed no difference between groups., Conclusions: Early statin initiation was not associated with lower mortality contrasting with findings of previous observational studies. Trial emulation helped in identifying and addressing sources of bias incompletely addressed by previous work. Statin use may be indicated for other conditions but not COVID-19., Competing Interests: Competing interests: LP receives funds for unrelated research from Omron Healthcare Co. Other authors have no other conflicts to declare., (© Author(s) (or their employer(s)) 2024. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.)

Purpose: Significant methodological shortcomings limit the validity of prior research on pregnancy decision-making and the effects of 'unintended' pregnancies on people's health and well-being. The Attitudes and Decisions After Pregnancy Testing (ADAPT) study investigates the consequences for individuals unable to attain their pregnancy and childbearing preferences using an innovative nested prospective cohort design and novel conceptualisation and measurement of pregnancy preferences., Participants: This paper describes the characteristics of the ADAPT Study Cohort, comprised of 2015 individuals aged 15-34 years, assigned female at birth, recruited between 2019 and 2022 from 23 health facilities in the southwestern USA., Findings to Date: The cohort was on average 25 years old. About 59% identified as Hispanic/Latine, 21% as white, and 8% as black, 13% multiracial or another race. Over half (56%) were nulliparous. About 32% lived in a household with income <100% of the federal poverty level. A significant minority (37%) reported a history of a depressive, anxiety or other mental health disorder diagnosis, and 30% reported currently experiencing moderate or severe depressive symptoms. Over one-quarter (27%) had ever experienced physical intimate partner violence, and almost half (49%) had ever experienced emotional abuse. About half (49%) had been diagnosed with a chronic health condition, and 37% rated their physical health as fair or poor. The 335 (17%) participants who experienced incident pregnancy over 1 year were similar to selected non-pregnant matched comparison participants in terms of age, racial and ethnic identity, and parity but were more likely to live with a main partner than comparison participants., Future Plans: We will continue to follow participants who experienced incident pregnancy and non-pregnant comparison participants until 2026. Analyses will examine pregnancy decision-making and investigate differences in health and well-being by prepregnancy pregnancy desires and feelings after the discovery of pregnancy, offering new insights into the consequences of not attaining one's reproductive preferences., Trial Registration Number: NCT03888404., Competing Interests: Competing interests: None declared., (© Author(s) (or their employer(s)) 2024. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.)

Background: The WHO identifies climate change as the most significant threat to global health systems. Indigenous peoples, whose lives are deeply intertwined with nature, are particularly vulnerable to the impacts of these changes., Objective: This study aimed to understand the perspectives of Indigenous stakeholders and public services managers on the interconnectedness of climate change and Indigenous health., Design: A qualitative study with 22 Indigenous stakeholders and public service managers on climate change and perceived impact on Indigenous health., Setting and Participants: Indigenous stakeholders and public service managers on climate change and perceived impact on Indigenous health from Brazil. Data was collected through interviews incorporating two vignette videos depicting environmental and health scenarios. Thematic content analysis was used to analyse the data., Results: The analytical process yielded six subcategories that were further grouped into three overarching thematic macro-categories: environmental degradation and climate change in the context of Indigenous peoples; environment, vulnerability and impact on Indigenous mental health; and actions and public health policies for Indigenous peoples., Conclusion: The perspectives of Indigenous stakeholders and public service managers on the interconnectedness of climate change and Indigenous health were deeply entrenched in their lived experiences of loss of their lands from deforestation and environmental degradation. They argued strongly for the strengthening of public health policies aimed at the Indigenous peoples, to face many challenges, especially suicide, and to have a voice in decision-making. A sensitive approach that values Indigenous peoples' connections with nature is fundamental to promote their health and well-being., Competing Interests: Competing interests: None declared., (© Author(s) (or their employer(s)) 2024. Re-use permitted under CC BY. Published by BMJ.)

Gold standard treatments for military personnel seeking support for mental health difficulties are often standardised and manualised to ensure high levels of treatment fidelity. While manualised treatments are preferable to less evidence-based idiosyncratic approaches, they may not fully account for the differences in symptom profiles present in patients with the same psychological diagnosis. Indeed, recent findings have highlighted that a significant proportion of individuals do not benefit from the 'gold standard' treatments. This brief report discusses the utility of statistical techniques, specifically latent profile analysis and network analysis, to support the transition to more evidence-based idiosyncratic, personalised care for clinical military, and general, populations. Further incorporation of such analysis methods may support arriving at a framework to support the personalisation of care in terms of the selection and adaption of evidence-based approach treatments based on individual clinical need., Competing Interests: Competing interests: None declared., (© Author(s) (or their employer(s)) 2024. No commercial re-use. See rights and permissions. Published by BMJ.)

Objectives: We aim to evaluate estimated glomerular filtration rate (eGFR) patterns of progression in a multiethnic cohort of people with type I diabetes mellitus and with baseline eGFR ≥45 mL/min/1.73 m 2 ., Design: Observational cohort., Setting: People with a clinical diagnosis of type 1 diabetes, attending two university hospital-based outpatient diabetes clinics, in South London between 2004 and 2018., Participants: We studied 1495 participants (52% females, 81% white, 12% African-Caribbean and 7% others)., Primary and Secondary Outcome Measures: Clinical measures including weight and height, systolic blood pressure, diastolic blood pressure and laboratory results (such as serum creatinine, urine albumin to creatinine ratio (ACR), HbA1c were collected from electronic health records (EHRs) and eGFR was estimated by the Chronic Kidney Disease-Epidemiology Collaboration. Ethnicity was self-reported., Results: Five predominantly linear patterns/groups of eGFR trajectories were identified. Group I (8.5%) had a fast eGFR decline (>3 mL/min/1.73 m 2 year). Group II (23%) stable eGFR, group III (29.8%), groups IV (26.3%) and V (12.4%) have preserved eGFR with no significant fall. Group I had the highest proportion (27.6%) of African-Caribbeans. Significant differences between group I and the other groups were observed in age, gender, HbA1C, systolic and diastolic blood pressure, body mass index, cholesterol and urine ACR, p<0.05 for all. At 10 years of follow-up, 33% of group I had eGFR <30 and 16.5%<15 (mL/min/1.73 m 2 )., Conclusions: Distinct trajectories of eGFR were observed in people with type 1 diabetes. The group with the highest risk of eGFR decline had a greater proportion of African-Caribbeans compared with others and has higher prevalence of traditional modifiable risk factors for kidney disease., Competing Interests: Competing interests: None declared., (© Author(s) (or their employer(s)) 2024. Re-use permitted under CC BY. Published by BMJ.)

Introduction: Historically, diseases and non-battle injuries (DNBI) typically stand for 70%‒95% of all medical events during military missions. There is, however, no comprehensive compilation of medical statistics for Swedish soldiers during deployment., Method: During United Nations Multidimensional Integrated Stabilization Mission in Mali, climate data and medical outpatient health surveillance data were compiled for Swedish soldiers deployed to Timbuctoo, between 2015 and 2019. Correlations between climate data and medical outpatient health surveillance data were analysed., Results: Battle injuries accounted for 0.4% of the visits to healthcare, while diseases accounted for 53.6%, and non-battle injuries for 46%, the majority being musculoskeletal injuries. The combination of high temperature, humidity, sun radiation and good visibility, during summer rotation weeks, caused more events of injuries and heat stress than any other period., Conclusion: Musculoskeletal injuries were the major cause for visits to the Swedish camp hospital. Injuries and heat stress increased during periods of high temperature, humidity, sun radiation and good visibility. Lack of medical data, i.e. unknown number of unique patients seeking healthcare, cause codes not always connected to a primary diagnosis, and revisits not being connected to a diagnose, complicated interpretation of health risk factors., Competing Interests: Competing interests: None declared., (© Author(s) (or their employer(s)) 2024. Re-use permitted under CC BY. Published by BMJ.)

Cardiovascular disease (CVD) is one of the leading causes of death across the world. CVD can lead to angina, heart attacks, heart failure, strokes, and eventually, death; among many other serious conditions. The early intervention with those at a higher risk of developing CVD, typically with statin treatment, leads to better health outcomes. For this reason, clinical prediction models (CPMs) have been developed to identify those at a high risk of developing CVD so that treatment can begin at an earlier stage. Currently, CPMs are built around statistical analysis of factors linked to developing CVD, such as body mass index and family history. The emerging field of machine learning (ML) in healthcare, using computer algorithms that learn from a dataset without explicit programming, has the potential to outperform the CPMs available today. ML has already shown exciting progress in the detection of skin malignancies, bone fractures and many other medical conditions. In this review, we will analyse and explain the CPMs currently in use with comparisons to their developing ML counterparts. We have found that although the newest non-ML CPMs are effective, ML-based approaches consistently outperform them. However, improvements to the literature need to be made before ML should be implemented over current CPMs. [ABSTRACT FROM AUTHOR]

Objective: This study aims to (1) build and validate model-based case definitions for multiple sclerosis (MS) that use trends (ie, trend-based case definitions) and (2) to apply dynamic classification to identify the average number of data years needed for classification (ie, average trend needed)., Design: Retrospective cohort study design., Participants: 608 MS cases and 59 620 MS non-cases., Setting: Data from 1 April 2004 to 31 March 2022 were obtained from the Manitoba Population Research Data Repository. MS case status was ascertained from homecare records and linked to health data. Trend-based case definitions were constructed using multivariate generalised linear mixed models applied to annual numbers of general and specialist physician visits, hospitalisations and MS healthcare contacts or medication dispensations. Dynamic classification, which ascertains cases and non-cases annually, was used to estimate mean classification time. Classification accuracy performance measures, including sensitivity, specificity, positive predictive value (PPV), negative predictive value (NPV), proportion correctly classified (PCC) and F1-scores, were compared for trend-based case definitions and a deterministic case definition of 3+MS healthcare contacts or medication dispensations., Results: When applied to the full study period, classification accuracy performance measure estimates for all case definitions exceeded 0.90, except sensitivity and PPV for the trend-based dynamic case definition (0.88, 0.64, respectively). PCC was high for all case definitions (0.94-0.99); F1-scores were lower for the trend-based case definitions compared with the deterministic case definition (0.74-0.93 vs 0.96). Dynamic classification identified 5 years as the average trend needed. When applied to the average trend windows, accuracy estimates for trend-based case definitions were lower than the estimates from the full study period (sensitivity: 0.77-0.89; specificity: 0.90-0.97; PPV: 0.54-0.81; NPV: 0.97-0.99; F1-score: 0.64-0.84). Accuracy estimates for the deterministic case definition remained high, except sensitivity (0.42-0.80). F1-score was variable (0.59-0.89)., Conclusions: Trend-based and deterministic case definitions classifications were similar to a population-based clinician assessment reference standard for multiple measures of classification accuracy. However, accuracy estimates for both trend-based and deterministic case definitions varied as the years of data used for classification were reduced. Dynamic classification appears to be a viable option for identifying the average trend needed for trend-based case definitions., Competing Interests: Competing interests: RAM receives research funding from: CIHR, Research Manitoba, Multiple Sclerosis Society of Canada, Multiple Sclerosis Scientific Foundation, Crohn’s and Colitis Canada, National Multiple Sclerosis Society, CMSC and the US Department of Defense, and is a co-investigator on studies receiving funding from Biogen Idec and Roche Canada. LL receives research funding from CIHR, Canada Research Chairs Programme, Natural Sciences and Engineering Research Council of Canada, National Institutes of Health, and the Canadian Agency for Drugs and Technologies in Health., (© Author(s) (or their employer(s)) 2024. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.)

Introduction: The commonly used frequentist paradigm of null hypothesis statistics testing with its reliance on the p-value and the corresponding notion of 'statistical significance' has been under ongoing criticism. Misinterpretation and misuse of the p-value have contributed to publication bias, unreliable studies, frequent false positives, fraud and mistrust in results of scientific studies. While p-values themselves are still useful, part of the problem may be the confusion between statistical and clinical significance. In randomised controlled trials of health interventions, this confusion could lead to erroneous conclusions about treatment efficacy, research waste and compromised patient outcomes. The extent to which clinical and statistical significance of published randomised clinical trials do not match is not known. This is a protocol for a methodological study to understand the extent of the problem of disparities between statistical and clinical significance in published clinical trials, and to identify and assess the factors associated with discrepant results in these studies., Methods and Analysis: A methodological survey of published randomised controlled trials is planned. Trials published between 2018 and 2022 and their protocols will be searched and screened for inclusion, with a planned sample size of 500 studies. The reported minimum clinically important difference, the study effect size and confidence intervals will be used to assess clinical importance of trial results. Comparison of statistical significance and clinical importance of the trial results will be used to determine disparity. Data will be analysed to estimate the outcomes, and factors associated with disparate study results will be assessed using logistic regression analysis., Ethics and Dissemination: Ethical approval for the study has been granted by Stellenbosch University's Health Research Ethics Committee. This is part of a larger study towards a PhD in Biostatistics and will be disseminated as a thesis, conference abstract and peer-reviewed manuscript., Competing Interests: Competing interests: None declared., (© Author(s) (or their employer(s)) 2024. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.)

Context: In the context of iterative feedback loops to support real-time policy decision making, and an emphasis on speeding up adoption of evidence-based interventions, qualitative healthcare researchers are increasingly expected to produce rapid results and products. Traditional qualitative methods have been adapted for this purpose., Objective: To develop and apply a rapid analysis framework in a process evaluation for the VICTORION-Spirit study; a ground-breaking hybrid trial examining real-world delivery of inclisiran-a cholesterol-lowering treatment-in primary care., Design: We developed a rapid analysis framework, using a summary template, to analyse data from semistructured telephone interviews., Setting: Primary care in Greater Manchester, UK., Participants: Patients who had received inclisiran as part of the VICTORION-Spirit trial (56), providers delivering inclisiran (28) and representatives from the Academic Health Science Network (8) participated in the original study., Results: The rapid analysis framework we developed and applied comprised six steps: (1) creating a summary template based on the five Consolidated Framework for Implementation Research domains; (2) test-driving, refining and finalising the summary template; (3) completing the template soon after each interview using field notes; (4) discussing analysis as a team; (5) transferring summaries to a matrix; and (6) using the summary matrix to inform presentations and interim reports for stakeholders. Our rapid analysis framework saved time and improved efficiency, as we were able to feedback barriers to stakeholders in real time via presentations., Conclusions: Rapid analysis in applied healthcare research can produce timely and trustworthy findings. Our rapid analysis framework would be useful within studies where there is a need to feedback to stakeholders and adjust implementation strategies accordingly in real time. Thus, supporting successful implementation efforts and accelerating adoption., Trial Registration Number: NCT04807400, 19/03/2021., Competing Interests: Competing interests: None declared., (© Author(s) (or their employer(s)) 2024. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.)

Introduction: Sociodemographic variables influence health outcomes, either directly (ie, gender identity) or indirectly (eg, structural/systemic racism based on ethnoracial group). Identification of how sociodemographic variables can impact the health of critically ill adults is important to guide care and research design for this population. However, despite the growing recognition of the importance of collecting sociodemographic measures that influence health outcomes, insufficient and inconsistent data collection of sociodemographic variables persists in critical care studies. We aim to develop a set of core data variables (CoDaV) for social determinants of health specific to studies involving critically ill adults., Methods and Analysis: We will conduct a scoping review to generate a list of possible sociodemographic measures to be used for round 1 of the modified Delphi processes. We will engage relevant knowledge users (previous intensive care unit patients and family members, critical care researchers, critical care clinicians and research co-ordinators) to participate in the modified Delphi consensus survey to identify the CoDaV. A final consensus meeting will be held with knowledge user representatives to discuss the final CoDaV, how each sociodemographic variable will be collected (eg, level of granularity) and how to disseminate the CoDaV for use in critical care studies., Ethics and Dissemination: The University of Calgary conjoint health research ethics board has approved this study protocol (REB22-1648)., Competing Interests: Competing interests: None declared., (© Author(s) (or their employer(s)) 2024. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.)

Introduction: Reviews of commercial and publicly available smartphone (mobile) health applications (mHealth app reviews) are being undertaken and published. However, there is variation in the conduct and reporting of mHealth app reviews, with no existing reporting guidelines. Building on the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines, we aim to develop the Consensus for APP Review Reporting Items (CAPPRRI) guidance, to support the conduct and reporting of mHealth app reviews. This scoping review of published mHealth app reviews will explore their alignment, deviation, and modification to the PRISMA 2020 items for systematic reviews and identify a list of possible items to include in CAPPRRI., Method and Analysis: We are following the Joanna Briggs Institute approach and Arksey and O'Malley's five-step process. Patient and public contributors, mHealth app review, digital health research and evidence synthesis experts, healthcare professionals and a specialist librarian gave feedback on the methods. We will search SCOPUS, CINAHL Plus, AMED, EMBASE, Medline, APA PsycINFO and the ACM Digital Library for articles reporting mHealth app reviews and use a two-step screening process to identify eligible articles. Information on whether the authors have reported, or how they have modified the PRISMA 2020 items in their reporting, will be extracted. Data extraction will also include the article characteristics, protocol and registration information, review question frameworks used, information about the search and screening process, how apps have been evaluated and evidence of stakeholder engagement. This will be analysed using a content synthesis approach and presented using descriptive statistics and summaries. This protocol is registered on OSF (https://osf.io/5ahjx)., Ethics and Dissemination: Ethical approval is not required. The findings will be disseminated through peer-reviewed journal publications (shared on our project website and on the EQUATOR Network website where the CAPPRRI guidance has been registered as under development), conference presentations and blog and social media posts in lay language., Competing Interests: Competing interests: None declared., (© Author(s) (or their employer(s)) 2024. Re-use permitted under CC BY. Published by BMJ.)

Introduction: African cities, particularly Abidjan and Johannesburg, face challenges of rapid urban growth, informality and strained health services, compounded by increasing temperatures due to climate change. This study aims to understand the complexities of heat-related health impacts in these cities. The objectives are: (1) mapping intraurban heat risk and exposure using health, socioeconomic, climate and satellite imagery data; (2) creating a stratified heat-health forecast model to predict adverse health outcomes; and (3) establishing an early warning system for timely heatwave alerts. The ultimate goal is to foster climate-resilient African cities, protecting disproportionately affected populations from heat hazards., Methods and Analysis: The research will acquire health-related datasets from eligible adult clinical trials or cohort studies conducted in Johannesburg and Abidjan between 2000 and 2022. Additional data will be collected, including socioeconomic, climate datasets and satellite imagery. These resources will aid in mapping heat hazards and quantifying heat-health exposure, the extent of elevated risk and morbidity. Outcomes will be determined using advanced data analysis methods, including statistical evaluation, machine learning and deep learning techniques., Ethics and Dissemination: The study has been approved by the Wits Human Research Ethics Committee (reference no: 220606). Data management will follow approved procedures. The results will be disseminated through workshops, community forums, conferences and publications. Data deposition and curation plans will be established in line with ethical and safety considerations., Competing Interests: Competing interests: MC, GM and CP have pension fund investments in the fossil fuel industry. The University of the Witwatersrand holds endowments and financial reserves invested in the same industry., (© Author(s) (or their employer(s)) 2024. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.)

Objectives: The main aim of this study was to demonstrate how ordered network analysis of video-recorded interactions combined with verbal response mode (VRM) coding (eg, edification, disclosure, reflection and interpretation) can uncover specific communication patterns that contribute to the development of shared understanding between physicians and nurses. The major hypothesis was that dyads that reached shared understanding would exhibit different sequential relationships between VRM codes compared with dyads that did not reach shared understanding., Design: Observational study design with the secondary analysis of video-recorded interactions., Setting: The study was conducted on two oncology units at a large Midwestern academic health care system in the USA., Participants: A total of 33 unique physician-nurse dyadic interactions were included in the analysis. Participants were the physicians and nurses involved in these interactions during patient care rounds., Primary and Secondary Outcome Measures: The primary outcome measure was the development of shared understanding between physicians and nurses, as determined by prior qualitative analysis. Secondary measures included the frequencies, orders and co-occurrences of VRM codes in the interactions., Results: A Mann-Whitney U test showed that dyads that reached shared understanding (N=6) were statistically significantly different (U=148, p=0.00, r=0.93) from dyads that did not reach shared understanding (N=25) in terms of the sequential relationships between edification and disclosure, edification and advisement, as well as edification and questioning. Dyads that reached shared understanding engaged in more edification followed by disclosure, suggesting the importance of this communication pattern for reaching shared understanding., Conclusions: This novel methodology demonstrates a robust approach to inform interventions that enhance physician-nurse communication. Further research could explore applying this approach in other healthcare settings and contexts., Competing Interests: Competing interests: None declared., (© Author(s) (or their employer(s)) 2024. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.)

Introduction: Many patients referred for suspicion of myelodysplastic neoplasm (MDS) are subjected to unnecessary discomfort from bone marrow aspiration, due to the low disease prevalence in this population. Flow cytometric analysis of peripheral blood neutrophil myeloperoxidase expression could rule out MDS with sensitivity and negative predictive value estimates close to 100%, ultimately obviating the need for bone marrow aspiration in up to 35% of patients. However, the generalisability of these findings is uncertain due to the limited sample size, the enrolment of patients at a single study site, and the reliability issues associated with laboratory-developed tests and varying levels of operator experience. This study aims to validate the accuracy attributes of peripheral blood neutrophil myeloperoxidase expression quantified by flow cytometric analysis in an independent multicentre sample., Methods and Analysis: The MPO-MDS-Valid project is a cross-sectional diagnostic accuracy study comparing an index test to a reference standard. Consecutive adult patients referred for suspicion of MDS are being recruited at seven university hospitals and one cancer centre in France. At each site, flow cytometric analysis of peripheral blood samples is performed by operators who are blinded to the reference diagnosis. A central adjudication committee whose members are unaware of the index test results will determine the reference diagnosis of MDS, based on cytomorphological evaluation of bone marrow performed in duplicate by experienced hematopathologists. The target sample size is 400 patients and the anticipated study recruitment completion date is 31 December 2025., Ethics and Dissemination: An institutional review board (Comité de Protection des Personnes Nord-Ouest III, Caen, France) approved the protocol, prior to the start of the study. Participants are recruited using an opt-out approach. Efforts will be made to publish the primary results within 6 months after study completion., Trial Registration Number: NCT05175469., Competing Interests: Competing interests: None declared., (© Author(s) (or their employer(s)) 2024. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.)

Introduction: Observational studies are fraught with several biases including reverse causation and residual confounding. Overview of reviews of observational studies (ie, umbrella reviews) synthesise systematic reviews with or without meta-analyses of cross-sectional, case-control and cohort studies, and may also aid in the grading of the credibility of reported associations. The number of published umbrella reviews has been increasing. Recently, a reporting guideline for overviews of reviews of healthcare interventions (Preferred Reporting Items for Overviews of Reviews (PRIOR)) was published, but the field lacks reporting guidelines for umbrella reviews of observational studies. Our aim is to develop a reporting guideline for umbrella reviews on cross-sectional, case-control and cohort studies assessing epidemiological associations., Methods and Analysis: We will adhere to established guidance and prepare a PRIOR extension for systematic reviews of cross-sectional, case-control and cohort studies testing epidemiological associations between an exposure and an outcome, namely Preferred Reporting Items for Umbrella Reviews of Cross-sectional, Case-control and Cohort studies (PRIUR-CCC). Step 1 will be the project launch to identify stakeholders. Step 2 will be a literature review of available guidance to conduct umbrella reviews. Step 3 will be an online Delphi study sampling 100 participants among authors and editors of umbrella reviews. Step 4 will encompass the finalisation of PRIUR-CCC statement, including a checklist, a flow diagram, explanation and elaboration document. Deliverables will be (i) identifying stakeholders to involve according to relevant expertise and end-user groups, with an equity, diversity and inclusion lens; (ii) completing a narrative review of methodological guidance on how to conduct umbrella reviews, a narrative review of methodology and reporting in published umbrella reviews and preparing an initial PRIUR-CCC checklist for Delphi study round 1; (iii) preparing a PRIUR-CCC checklist with guidance after Delphi study; (iv) publishing and disseminating PRIUR-CCC statement., Ethics and Dissemination: PRIUR-CCC has been approved by The Ottawa Health Science Network Research Ethics Board and has obtained consent (20220639-01H). Participants to step 3 will give informed consent. PRIUR-CCC steps will be published in a peer-reviewed journal and will guide reporting of umbrella reviews on epidemiological associations., Competing Interests: Competing interests: MS received honoraria/has been a consultant for Angelini, Lundbeck, Otsuka., (© Author(s) (or their employer(s)) 2024. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.)

Objectives: To develop a statistical approach that provides a quantitative index measuring the magnitude of the irregularity of the breathing response to exercise for the diagnosis of dysfunctional breathing., Design: Cross-sectional, retrospective, real-world study., Setting: Single-centre study., Participants: A population of 209 patients investigated with cardiopulmonary exercise testing in our institution for unexplained or disproportionate exertional dyspnoea between January and July 2022., Primary and Secondary Outcome Measures: A novel statistical approach providing a quantitative index-proportional tidal volume variation (PTVV)-was developed to measure the magnitude of the irregularity of the breathing response to exercise., Results: PTVV provided a reliable statistical readout for the objective assessment of DB with a prediction accuracy of 78% (95% CI: 72 to 83%). The prevalence of DB in the investigated population was high with more than half of the patients affected by moderate-to-severe DB., Conclusions: PTVV can easily be implemented in the clinical routine. Our study suggests a possible further simplification for the diagnosis of DB with two objective criteria including PTVV and one single criterion for hyperventilation., Competing Interests: Competing interests: None declared., (© Author(s) (or their employer(s)) 2024. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.)

Introduction: Due to a change in diagnostic prerequisites and the inclusion of novel diagnostic entities, the implementation of the 11th revision of the International Classification of Diseases (ICD-11) will presumably change prevalence rates of specific mental, behavioural or neurodevelopmental disorders and result in an altered prevalence rate for this grouping overall. This scoping review aims to summarise the characteristics of primary studies examining the prevalence of mental, behavioural or neurodevelopmental disorders based on ICD-11 criteria. The knowledge attained through this review will primarily characterise the methodological approaches of this research field and additionally assist in deciding which psychiatric diagnoses are-given the current literature-most relevant for subsequent systematic reviews and meta-analyses intended to approximate the magnitude of prevalence rates while providing a first glimpse of the range of expected (differences in) prevalence rates in these conditions., Methods and Analysis: MEDLINE, Embase, Web of Science and PsycINFO will be searched from 2011 to present without any language filters. This scoping review will follow the Preferred Reporting Items for Systematic Reviews and Meta-Analyses extension for Scoping Review guidelines.We will consider (a) cross-sectional and longitudinal studies (b) focusing on the prevalence rates of mental, behavioural or neurodevelopmental disorders (c) using ICD-11 criteria for inclusion. The omission of (a) case numbers and sample size, (b) study period and period of data collection or (c) diagnostic procedures on full-text level is considered an exclusion criterion.This screening will be conducted by two reviewers independently from one another and a third reviewer will be consulted with disagreements. Data extraction and synthesis will focus on outlining methodological aspects., Ethics and Dissemination: We intend to publish our review in a scientific journal. As the primary data are publicly available, we do not require research ethics approval., Competing Interests: Competing interests: None declared., (© Author(s) (or their employer(s)) 2024. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.)

Objective: The objective of this study was to compare ultrasound features and establish a predictive nomogram for distinguishing between triple-negative breast cancer (TNBC) and non-TNBC., Design: A retrospective cohort study., Setting: This study was conducted at Quanzhou First Hospital, a grade A tertiary hospital in Quanzhou, China, with the research data set covering the period from September 2019 to August 2023., Participants: The study included a total of 205 female patients with confirmed TNBC and 574 female patients with non-TNBC, who were randomly divided into a training set and a validation set at a ratio of 7:3., Main Outcome Measures: All patients underwent ultrasound examination and received a confirmatory pathological diagnosis. Nodules were classified according to the Breast Imaging-Reporting and Data System standard. Subsequently, the study conducted a comparative analysis of clinical characteristics and ultrasonic features., Results: A statistically significant difference was observed in multiple clinical and ultrasonic features between TNBC and non-TNBC. Specifically, in the logistic regression analysis conducted on the training set, indicators such as posterior echo, lesion size, presence of clinical symptoms, margin characteristics, internal blood flow signals, halo and microcalcification were found to be statistically significant (p<0.05). These significant indicators were then effectively incorporated into a static and dynamic nomogram model, demonstrating high predictive performance in distinguishing TNBC from non-TNBC., Conclusion: The results of our study demonstrated that ultrasound features can be valuable in distinguishing between TNBC and non-TNBC. The presence of posterior echo, size, clinical symptoms, margin, internal flow, halo and microcalcification was identified as predictive factors for this differentiation. Microcalcification, hyperechoic halo, internal flow and clinical symptoms emerged as the strongest predictive factors, indicating their potential as reliable indicators for identifying TNBC and non-TNBC., Competing Interests: Competing interests: None declared., (© Author(s) (or their employer(s)) 2024. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.)

Objectives: To assess the efficacy of a sustained educational intervention to affect diverse outcomes across the pregnancy and infancy timeline., Setting: A multi-arm cluster-randomised controlled trial in 99 villages in Honduras' Copán region, involving 16 301 people in 5633 households from October 2015 to December 2019., Participants: Residents aged 12 and older were eligible. A photographic census involved 93% of the population, with 13 881 and 10 263 individuals completing baseline and endline surveys, respectively., Intervention: 22-month household-based counselling intervention aiming to improve practices, knowledge and attitudes related to maternal, neonatal and child health., Primary and Secondary Outcome Measures: Primary outcomes were prenatal/postnatal care behaviours, facility births, exclusive breast feeding, parental involvement, treatment of diarrhoea and respiratory illness, reproductive health, and gender/reproductive norms. Secondary outcomes were knowledge and attitudes related to the primary outcomes., Results: Parents targeted for the intervention were 16.4% (95% CI 3.1%-29.8%, p=0.016) more likely to have their newborn's health checked in a health facility within 3 days of birth; 19.6% (95% CI 4.2%-35.1%, p=0.013) more likely to not wrap a fajero around the umbilical cord in the first week after birth; and 8.9% (95% CI 0.3%-17.5%, p=0.043) more likely to report that the mother breast fed immediately after birth. Changes in knowledge and attitudes related to these primary outcomes were also observed. We found no significant effect on various other practices., Conclusion: A sustained counselling intervention delivered in the home setting by community health workers can meaningfully change practices, knowledge and attitudes related to proper newborn care following birth, including professional care-seeking, umbilical cord care and breast feeding., Trial Registration Number: NCT02694679., Competing Interests: Competing interests: NAC reports grants from the Bill and Melinda Gates Foundation and the Tata-Yale Alliance. EMA reports grants from the National Science Foundation and Office of Naval Research. LF reports a grant from the National Institutes of Health. The Bill and Melinda Gates Foundation funded the intervention through a grant to IDB supervised by JN and EI. All other authors declare no competing interests., (© Author(s) (or their employer(s)) 2024. Re-use permitted under CC BY. Published by BMJ.)

Objectives: To map the available methodological guidelines and documents for conducting and reporting benefit-risk assessment (BRA) during health technologies' life cycle; and to identify methodological guidelines for BRA that could serve as the basis for the development of a BRA guideline for the context of health technology assessment (HTA) in Brazil., Design: Scoping review., Methods: Searches were conducted in three main sources up to March 2023: (1) electronic databases; (2) grey literature (48 HTA and regulatory organisations) and (3) manual search and contacting experts. We included methodological guidelines or publications presenting methods for conducting or reporting BRA of any type of health technologies in any context of the technology's life cycle. Selection process and data charting were conducted by independent reviewers. We provided a structured narrative synthesis of the findings., Results: From the 83 eligible documents, six were produced in the HTA context, 30 in the regulatory and 35 involved guidance for BRA throughout the technology's life cycle. We identified 129 methodological approaches for BRA in the documents. The most commonly referred to descriptive frameworks were the Problem, Objectives, Alternatives, Consequences, Trade-offs, Uncertainty, Risk and Linked decisions and the Benefit-Risk Action Team. Multicriteria decision analysis was the most commonly cited quantitative framework. We also identified the most cited metric indices, estimation and utility survey techniques that could be used for BRA., Conclusions: Methods for BRA in HTA are less established. The findings of this review, however, will support and inform the elaboration of the Brazilian methodological guideline on BRA for HTA., Trial Registration Number: https://doi.org/10.17605/OSF.IO/69T3V., Competing Interests: Competing interests: None declared., (© Author(s) (or their employer(s)) 2024. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.)