Study Design Characteristics Research Articles

The Danish National Health Survey 2023: study design and participant characteristics.

To describe the design of the Danish National Health Survey (DNHS) 2023, participants' demographic characteristics and differences in demographic and selected health-related characteristics between respondents invited by web-mode and paper-mode. A sample of 25,000 residents in Denmark aged 16 years or above was invited to participate in the DNHS 2023 using a mixed-mode approach (web/paper mode). Web-mode invited were additionally invited to participate in an accelerometer study. The self-administered questionnaire included 83 questions about health, health behaviour and morbidity. Descriptive statistics were used to describe characteristics associated with response and invitation mode. The response proportion was 40.8%. Non-response was more frequent among men, individuals of the youngest age groups, individuals with non-Western backgrounds, unmarried and individuals from densely populated areas. The response proportion was higher among web-mode invited (42.0%) than paper-mode invited (22.6%). Paper-mode invited respondents were more often women, aged 80 years or older, and widowed compared with web-mode invited respondents. The DNHS 2023 is a national health survey including adult residents in Denmark. Non-response was more pronounced among some subgroups; however, calibrated weights were calculated to minimise non-response bias. The survey is essential for public health surveillance and can be used in health planning and policy development. Furthermore, the data from the survey can be used for research on the population's health and health behaviour. For future waves of the DNHS, it should be considered whether resources should be used to invite people unsubscribed from digital-post due to the low response proportion.

Study Design and Baseline Characteristics of ALIGN, a Randomized Controlled Study of Atrasentan in Patients With IgA Nephropathy

IntroductionEndothelin A (ETA) receptor activation is a driver of proteinuria, kidney inflammation and fibrosis in IgA nephropathy (IgAN). Atrasentan, a selective ETA receptor antagonist, has potential to reduce proteinuria and preserve kidney function in IgAN. ALIGN (NCT04573478) is a phase 3, randomized, double-blind, placebo-controlled clinical trial of atrasentan in patients with IgAN at high risk of kidney function loss. MethodsEligibility criteria for the ALIGN main stratum included patients with biopsy-proven IgAN, total protein excretion ≥1g/d, eGFR ≥30 mL/min/1.73m2, receiving a maximally-tolerated stable dose of a renin-angiotensin system inhibitor (RASi). An exploratory stratum enrolled participants also receiving a stable dose of sodium glucose cotransporter-2 inhibitors (SGLT2i). Participants were randomized to 0.75 mg atrasentan or placebo orally daily for 132 weeks followed by a 4-week wash-out period. The primary outcome is proteinuria change from baseline (24h urine protein–creatinine ratio UPCR) to Week 36 in the main stratum. Key secondary endpoints include eGFR change from baseline to Week 136, safety and tolerability. ResultsEnrolment occurred across 20 countries; 404 patients are randomized: 340 in the main stratum and 64 in the SGLT2i stratum. At baseline in the main stratum, mean age was 44.7 years, 42.4% were female, mean eGFR and median UPCR were 58.9 mL/min/1.73 m2 and 1.4 g/g, respectively. ConclusionThe ongoing ALIGN trial evaluates the efficacy and safety of atrasentan in a representative global IgAN population at risk for disease progression despite optimized RASi therapy and includes an exploratory stratum evaluating atrasentan use in combination with an SGLT2i.

79 - Study Design and Baseline Characteristics of Older Medicaid Recipients Enrolled in the Incontinence Helping Others Manage at Home (Incon@Home) Implementation Study

79 - Study Design and Baseline Characteristics of Older Medicaid Recipients Enrolled in the Incontinence Helping Others Manage at Home (Incon@Home) Implementation Study

New cancer medicines in Europe 2010–2020: comparison of medicines with or without extensions of indications

IntroductionDuring the last decade, extensions of therapeutic indications have been one of the most common methods to extend the lifecycle of a medical product in the post-authorisation phase and to...

Prospective Cohort Investigation on Physical Activity of Osteoporosis Outcomes (PAOPO) in Jidong: Objectives, Study Design, and Baseline Characteristics.

The aim of this study was to investigate the prospective association between physical activity (PA), independently or in conjunction with other contributing factors, and osteoporosis (OP) outcomes. The Physical Activity in Osteoporosis Outcomes (PAOPO) study was a community-based cohort investigation. A structured questionnaire was used to gather the participants' sociodemographic characteristics. Bone mineral density (BMD) measurements were performed to assess OP outcomes, and the relationship between BMD and OP was evaluated within this cohort. From 2013 to 2014, 8,471 participants aged 18 years and older were recruited from Tangshan, China's Jidong community. Based on their PA level, participants were categorized as inactive, moderately active, or very active. Men showed higher physical exercise levels than women across the activity groups. BMD was significantly higher in the very active group than in the moderately active and inactive groups. Individuals aged > 50 years are at a higher risk of developing OP and osteopenia. The PAOPO study offers promising insights into the relationship between PA and OP outcomes, encouraging the implementation of PA in preventing and managing OP.

Conducting power analysis for meta-analysis with dependent effect sizes: Common guidelines and an introduction to the POMADE R package.

Sample size and statistical power are important factors to consider when planning a research synthesis. Power analysis methods have been developed for fixed effect or random effects models, but until recently these methods were limited to simple data structures with a single, independent effect per study. Recent work has provided power approximation formulas for meta-analyses involving studies with multiple, dependent effect size estimates, which are common in syntheses of social science research. Prior work focused on developing and validating the approximations but did not address the practice challenges encountered in applying them for purposes of planning a synthesis involving dependent effect sizes. We aim to facilitate the application of these recent developments by providing practical guidance on how to conduct power analysis for planning a meta-analysis of dependent effect sizes and by introducing a new R package, POMADE, designed for this purpose. We present a comprehensive overview of resources for finding information about the study design features and model parameters needed to conduct power analysis, along with detailed worked examples using the POMADE package. For presenting power analysis findings, we emphasize graphical tools that can depict power under a range of plausible assumptions and introduce a novel plot, the traffic light power plot, for conveying the degree of certainty in one's assumptions.

A Framework for the Use and Likelihood of Regulatory Acceptance of Single-Arm Trials.

Single-arm clinical trials (SAT) are common in drug and biologic submissions for rare or life-threatening conditions, especially when no therapeutic options exist. External control arms (ECAs) improve interpretation of SATs but pose methodological and regulatory challenges. Through narrative reviews and expert input, we developed a framework for considerations that might influence regulatory use and likelihood of regulatory acceptance of an SAT, identifying non-oncology first indication approvals as an area of interest. We systematically analyzed FDA and EMA approvals using SATs as pivotal evidence. The framework guided outcome abstraction on regulatory responses. We examined all non-oncology FDA and EMA drug and biologic approvals for first indications from 2019 to 2022 to identify those with SAT as pivotal safety or efficacy evidence. We abstracted outcomes, key study design features, regulator responses to SAT and (where applicable) ECA design, and product label content. Among 20 SAT-based FDA approvals and 17 SAT-based EMA approvals, most common indications were progressive rare diseases with high unmet need/limited therapeutic options and a natural history without spontaneous improvement. Of the types of comparators, most were natural history cohorts (45% FDA; 47% EMA) and baseline controls (40% FDA; 47% EMA). Common critiques were of non-contemporaneous ECAs, subjective endpoints, and baseline covariate imbalance between arms. Based on recent FDA and EMA approvals, the likelihood of regulatory success for SATs with ECAs depends on many design, analytic, and data quality considerations. Our framework is useful in early drug development when considering SAT strategies for evidence generation.

No negative effects of intra-abdominal bio-logger implantation under general anaesthesia on spatial cognition learning in a hibernator the edible dormouse.

The effect of hibernation on cognitive capacities of individuals is not fully understood, as studies provide conflicting results. Most studies focus on behavioural observations without taking the physiological state of individuals to account. To mechanistically understand the effect of hibernation on the brain, physiological parameters need to be included. The implantation of bio-loggers can provide insights on i.e. body temperature without further manipulation of the animals. Surgeries and anaesthesia, however, can harm animals' health and cause cognitive dysfunction, potentially biasing data collected through bio-loggers. We investigated the effects of bio-logger implantation surgery on cognitive performance and learning, controlling for animal and study design characteristics. First, juvenile dormice successfully learned to solve a spatial cognition task using a vertical maze. Distance, transitions, velocity, and duration were measured as indicators for performance. After training, bio-loggers were implanted intra-abdominally under general anaesthesia. Animals were re-tested in the maze two weeks after. We found no effect of bio-logger implantation and surgery on performance. This study is the first to show spatial cognition learning in edible dormice and provides a full description of the peri-anaesthetic management and a protocol for bio-logger implantation surgery in dormice. Importantly, measures were taken to mitigate common anaesthetic complications that could lead to post-operative cognitive dysfunction and influence animal behaviour. By pairing physiological measurements through bio-logger implantation with behaviour and cognition measurements, future research will significantly advance the understanding on mechanisms of learning and behaviour.

Cohort profile: study design and baseline characteristics of an observational longitudinal weight loss cohort and biorepository of patients undergoing sleeve gastrectomy in the USA

PurposeWe developed a comprehensive sleeve gastrectomy (SG) weight loss study cohort and biorepository to uncover mechanisms, biomarkers and predictive factors of weight loss, weight maintenance and amelioration of obesity-related comorbidities....

Risk Factors in Pregnant Women as Predictors of Postpartumhaemorrhage: A Systematic Review Aligned with SDG Goals

Objective: The objective of this study is to examine the risk factors affecting mothers that serve as predictors of postpartum haemorrhage, with the aim of identifying key predictors to improve clinical outcomes and reduce maternal mortality, aligning with the Sustainable Development Goals (SDGs). Theoretical Framework: In this topic, the main concepts and theories that underpin the research are presented. The framework includes understanding the pathophysiology of postpartum haemorrhage, maternal health indicators, and predictive analytics models. The Sustainable Development Goals (SDGs) for reducing Maternal Mortality Ratio (MMR) provide a solid basis for understanding the context of the investigation. Method: The methodology adopted for this research comprises a systematic review of the literature. A comprehensive search was conducted in PubMed, SpringerLink, ScienceDirect, Neliti, and Research Gate using the keywords "risk factor as predictor for postpartum haemorrhage." The search yielded 3587 articles, which were screened based on inclusion criteria: published between 2014 and 2024, original articles, full-text accessible, in English or Indonesian, and relevant to risk factors predicting postpartum haemorrhage. This process narrowed the selection to 805 articles. Further screening based on exclusion criteria, such as discrepancies in research titles, article inaccessibility, and duplication, resulted in six articles. These align with the PICOS framework: Population (women giving birth with or without comorbidities), Intervention (risk factors for postpartum haemorrhage), Outcome (predictors of postpartum haemorrhage), and Study (observational studies like cohorts, case controls, and cross-sectional studies). Results and Discussion: The results obtained revealed 33 risk factors that could be used as predictors of postpartum haemorrhage. In the discussion section, these results are contextualized in light of the theoretical framework, highlighting the implications and relationships identified. Possible discrepancies and limitations of the study are also considered in this section, including variations in study design and population characteristics. Research Implications: The practical and theoretical implications of this research are discussed, providing insights into how the results can be applied or influence practices in the field of maternal health. These implications could encompass clinical practice guidelines, development of predictive tools, and policy-making aimed at reducing MMR in line with SDGs Originality/Value: This study contributes to the literature by highlighting the originality of the research through the identification of multiple risk factors as clinical predictors of postpartum haemorrhage. The relevance and value of this research are evidenced by the potential to develop scoring systems, nomograms, and XGBoost classification models, which may significantly impact maternal healthcare practices and outcomes.

Escape room design in training crew resource management in acute care: a scoping review

BackgroundEffective teamwork is crucial to providing safe and high-quality patient care, especially in acute care. Crew Resource Management (CRM) principles are often used for training teamwork in these situations, with escape rooms forming a promising new tool. However, little is known about escape room design characteristics and their effect on learning outcomes. We investigated the current status of design characteristics and their effect on learning outcomes for escape room-based CRM/teamwork training for acute care professionals. We also aimed to identify gaps in literature to guide further research.MethodsMultiple databases were searched for studies describing the design and effect of escape rooms aimed training CRM/teamwork in acute care professionals and in situations that share characteristics. A standardized process was used for screening and selection. An evidence table that included study characteristics, design characteristics and effect of the escape room on learning outcomes was used to extract data. Learning outcomes were graded according to IPE expanded typology of Kirkpatrick’s levels of learning outcome and Medical Education Research Study Quality Instrument (MERSQI) scores were calculated to assess methodology.ResultsFourteen studies were included. Common design characteristics were a team size of 4–6 participants, a 40-minute time limit, linear puzzle organization and use of briefing and structured debriefing. Information on alignment was only available in five studies and reporting on several other educational and escape room design characteristics was low. Twelve studies evaluated the effect of the escape room on teamwork: nine evaluated reaction (Kirkpatrick level 1; n = 9), two evaluated learning (Kirkpatrick level 2) and one evaluated both. Overall effect on teamwork was overtly positive, with little difference between studies. Together with a mean MERSQI score of 7.0, this precluded connecting specific design characteristics to the effect on learning outcomes.ConclusionsThere is insufficient evidence if and how design characteristics affect learning outcomes in escape rooms aimed at training CRM/teamwork in acute care professionals. Alignment of teamwork with learning goals is insufficiently reported. More complete reporting of escape rooms aimed at training CRM/teamwork in acute care professionals is needed, with a research focus on maximizing learning potential through design.

Analysis of risk of bias assessments in a sample of intervention systematic reviews, Part II: focus on risk of bias tools reveals few meet current appraisal standards

ObjectivesRisk of bias (RoB) assessment is a critical part of any systematic review (SR). There are multiple tools available for assessing RoB of the studies included in a SR. The conduct of these assessments in intervention SRs are addressed by three items in AMSTAR-2, considered the preferred tool for critically appraising an intervention SR. This study focuses attention on item 9, which assesses the ability of a RoB tool to adequately address sources of bias, particularly in randomized trials (RCTs) and nonrandomized studies of interventions (NRSI). Our main objective is to report the detailed results of our examination of both Cochrane and non-Cochrane RoB tools and distinguish those that meet AMSTAR-2 item 9 appraisal standards. Study Design and SettingWe identified critical appraisal tools reported in a sample of 126 SRs reporting on interventions for persons with cerebral palsy published from 2014 to 2021. Eligible tools were those that had been used to assess the primary studies included in these SRs and for which assessment results were reported in enough detail to allow appraisal of the tool. We identified the version of the tool applied as original, modified, or novel and established the applicable study designs as intended by the tools’ developers. We then evaluated the potential ability of these tools to assess the four sources of bias specified in AMSTAR-2 item 9 for RCTs and NRSI. We adapted item 9 to appraise tools applied to single-case experimental designs, which we also encountered in this sample of SRs. ResultsMost of the eligible tools are recognized by name in the published literature and were applied in the original or modified form. Modifications were applied with considerable variability across the sample. Of the 37 tools we examined, those judged to fully meet the appraisal standards for RCTs included all the Cochrane tools, the original and modified Downs and Black Checklist, and the quality assessment standard for a cross-over study by Ding et al; for NRSI, these included all the Cochrane tools, the original and modified Downs and Black Checklist, and the Research Triangle Institute item bank on Risk of Bias and Precision of Observational Studies for NRSI. In general, tools developed for a specific study design were judged to meet the appraisal standards fully or partially for that design. These results suggest it is unlikely that a single tool will be adequate by AMSTAR-2 item 9 appraisal standards for an intervention SR that includes studies of various designs. ConclusionTo our knowledge, this is the first resource providing SR authors with practical information about the appropriateness and adequacy of RoB tools by the appraisal standards specified in AMSTAR-2 item 9 for RCTs and NRSI. We propose similar methods for appraisal of tools applied to single-case experimental design. We encourage authors to seek contemporary RoB tools developed for use in healthcare-related intervention SRs and designed to evaluate relevant study design features. The tools should address attributes unique to the review topic and research question but not be subjected to unjustified and excessive modifications. We promote recognition of the potential shortcomings of both Cochrane and non-Cochrane RoB tools, even those that perform well by AMSTAR-2 item 9 appraisal standards.

Alectinib vs. Lorlatinib in the Front-Line Setting for ALK-Rearranged Non-Small-Cell Lung Cancer (NSCLC): A Deep Dive into the Main Differences across ALEX and CROWN Phase 3 Trials.

Various next-generation ALK TKIs are available as first-line options for ALK-positive NSCLC, with alectinib and lorlatinib being commonly preferred. However, no direct comparison between them has been conducted, making it impossible to pick a winner. We performed an analytic, 'non-comparative' assessment of the two phase 3 pivotal clinical trials showing superiority of alectinib (ALEX) and lorlatinib (CROWN) in comparison to crizotinib. Overall, the two studies were very similar in the study design and patient characteristics, with the exception of the selection and evaluation of brain metastases. PFS hazard ratios numerically favored lorlatinib, both according to the investigator and to BICR. Notably, the 3-year PFS rate was numerically higher with lorlatinib (64%) than with alectinib (46.4%). Despite similar response rates and overall intracranial response, the rate of complete intracranial response was higher with lorlatinib, with a cumulative incidence risk of CNS disease progression at 12 months of 9.4% with alectinib and 2.8% with lorlatinib. The peculiar toxicities of lorlatinib were related to lipidic profile alterations, peripheral oedema and cognitive effects, with no impact on cardiovascular risk nor impairment in quality of life versus crizotinib. Furthermore, the rate of permanent treatment discontinuation due to adverse events was numerically higher with alectinib (26%) than with lorlatinib (7%). In conclusion, despite the immature OS data for both drugs, the efficacy of lorlatinib appears higher than alectinib while maintaining a manageable toxicity profile.

Scoping Literature Review of the Impact of COVID-19 on Burnout Syndrome Among Critical Care Nurses

Background: This scoping literature review examines burnout among critical care nurses, specifically focusing on the impact of the COVID-19 pandemic, a pervasive concern in healthcare professions. Aim: The primary objective of this study is to assess the prevalence of burnout syndrome among critical care nurses, with a specific focus on the pandemic's influence. The ultimate goal is to facilitate evidence-based interventions that can effectively alleviate burnout and safeguard the well-being of critical care nurses. Methods: We conducted a scoping review of the literature spanning from 2018 to 2023, utilizing PubMed and Hrčak databases and targeting original research papers, systematic reviews, and review articles. Employing a structured guideline, we outlined objectives, defined the study scope, and established inclusion/exclusion criteria. For data extraction, a protocol systematically captured key details, including study design and participant characteristics. The Maslach Burnout Inventory Scale, along with other measurement tools, guided burnout categorization. Our data synthesis followed a methodical approach outlined in the guideline, ensuring transparency and reliability, ultimately enhancing the overall robustness of our review. Results: This literature review comprehensively reviewed 15 articles. The findings are that burnout rates have risen in healthcare professionals, including nurses, with pandemic-related challenges amplifying the risk among critical care nurses. Contributing factors include demographic variables, job-related stressors, and insufficient workplace support. Conclusion: The findings distinctly highlight the exacerbating impact of the COVID-19 pandemic on burnout, introducing unique stressors that contribute to heightened levels among healthcare professionals. Demographic variables, job-related stressors, and insufficient workplace support emerge as consistent contributors to burnout, necessitating tailored interventions. The studies express the need for urgent, targeted interventions to safeguard the well-being of critical care nurses and maintain the quality of patient care in both routine healthcare and crisis settings.

Study design features increase replicability in cross-sectional and longitudinal brain-wide association studies.

Brain-wide association studies (BWAS) are a fundamental tool in discovering brain-behavior associations. Several recent studies showed that thousands of study participants are required for good replicability of BWAS because the standardized effect sizes (ESs) are much smaller than the reported standardized ESs in smaller studies. Here, we perform analyses and meta-analyses of a robust effect size index using 63 longitudinal and cross-sectional magnetic resonance imaging studies from the Lifespan Brain Chart Consortium (77,695 total scans) to demonstrate that optimizing study design is critical for increasing standardized ESs and replicability in BWAS. A meta-analysis of brain volume associations with age indicates that BWAS with larger variability in covariate have larger reported standardized ES. In addition, the longitudinal studies we examined reported systematically larger standardized ES than cross-sectional studies. Analyzing age effects on global and regional brain measures from the United Kingdom Biobank and the Alzheimer's Disease Neuroimaging Initiative, we show that modifying longitudinal study design through sampling schemes improves the standardized ESs and replicability. Sampling schemes that improve standardized ESs and replicability include increasing between-subject age variability in the sample and adding a single additional longitudinal measurement per subject. To ensure that our results are generalizable, we further evaluate these longitudinal sampling schemes on cognitive, psychopathology, and demographic associations with structural and functional brain outcome measures in the Adolescent Brain and Cognitive Development dataset. We demonstrate that commonly used longitudinal models can, counterintuitively, reduce standardized ESs and replicability. The benefit of conducting longitudinal studies depends on the strengths of the between- versus within-subject associations of the brain and non-brain measures. Explicitly modeling between- versus within-subject effects avoids averaging the effects and allows optimizing the standardized ESs for each separately. Together, these results provide guidance for study designs that improve the replicability of BWAS.

A multidomain lifestyle intervention to maintain optimal cognitive functioning in Dutch older adults—study design and baseline characteristics of the FINGER-NL randomized controlled trial

BackgroundEvidence on the effectiveness of multidomain lifestyle interventions to prevent cognitive decline in older people without dementia is mixed. Embedded in the World-Wide FINGERS initiative, FINGER-NL aims to investigate the effectiveness of a 2-year multidomain lifestyle intervention on cognitive functioning in older Dutch at risk individuals.MethodsMulti-center, randomized, controlled, multidomain lifestyle intervention trial with a duration of 24 months. 1210 adults between 60–79 years old with presence of ≥ 2 modifiable risk factors and ≥ 1 non-modifiable risk factor for cognitive decline were recruited between January 2022 and May 2023 via the Dutch Brain Research Registry and across five study sites in the Netherlands. Participants were randomized to either a high-intensity or a low-intensity intervention group. The multidomain intervention comprises a combination of 7 lifestyle components (physical activity, cognitive training, cardiovascular risk factor management, nutritional counseling, sleep counseling, stress management, and social activities) and 1 nutritional product (Souvenaid®) that could help maintain cognitive functioning. The high-intensity intervention group receives a personalized, supervised and hybrid intervention consisting of group meetings (on-site and online) and individual sessions guided by a trained lifestyle coach, and access to a digital intervention platform that provides custom-made training materials and selected lifestyle apps. The low-intensity intervention group receives bi-monthly online lifestyle-related health advice via the digital intervention platform. Primary outcome is 2-year change on a cognitive composite score covering processing speed, executive function, and memory.ResultsWithin 17 months, participant recruitment has been successfully completed (N = 1210; mean age: 67.7 years (SD: 4.6); 64% female). Modifiable risk factors commonly present at baseline were physical inactivity (89%), low mental/cognitive activity (50%), low social engagement (39%), hypertension (39%) and high alcohol consumption (39%). The mean body mass index of participants was 28.3 (SD: 4.2) and the total serum cholesterol was 5.4 mmol/L (SD: 1.2).ConclusionsBaseline lifestyle and clinical measurements showed successful recruitment of participants with sufficient potential for prevention. Results of FINGER-NL will provide further insight into the efficacy of a multidomain lifestyle intervention to prevent cognitive decline in older adults.Trial registrationClinicalTrials.gov (ID: NCT05256199)/2022–01-11.

Cost burden of cirrhosis and liver disease progression in metabolic dysfunction-associated steatohepatitis: A US cohort study.

Metabolic dysfunction-associated steatohepatitis (MASH), formerly nonalcoholic steatohepatitis, is characterized by fat accumulation and inflammation of the liver and may result in progression to cirrhosis and liver-related events. To characterize the impact of cirrhosis and progression to liver-related events on costs and health care resource use (HCRU) among MASH patients in the United States. The study cohort included patients with diagnosed nonalcoholic steatohepatitis (International Classification of Diseases, Tenth Revision, Clinical Modification code K75.81) in Optum's deidentified Clinformatics Data Mart Database (October 2015 to December 2022) and were stratified by baseline cirrhosis status. Among those without cirrhosis at baseline, patients were further stratified by status of progression to cirrhosis during follow-up. Total HCRU and costs per-person per-year (PPPY) were estimated and compared descriptively between the cohorts. In addition, gamma generalized linear models were used to compare costs PPPY between those with vs without cirrhosis at baseline, as well as with vs without progression during follow-up, while adjusting for baseline patient and disease characteristics. Annual costs per person were also longitudinally modeled using gamma generalized linear mixed models to understand longitudinal changes in costs PPPY while accounting for time correlations within individual patients. Lastly, a series of sensitivity analyses were conducted to assess the impact of study design features and clinical variations of total costs PPPY. A total of 28,576 adults were included, and 9,157 (32.0%) had baseline cirrhosis; of the 19,419 without baseline cirrhosis, a total of 4,235 (21.8%) progressed over follow-up. Mean (SD) HCRU and costs PPPY were higher among patients with cirrhosis ($110,403 [$226,037]) than without ($28,340 [$61,472]; P < 0.01) and among those with progression ($58,128 [$102,626]) than without ($20,031 [$39,740]; P < 0.01). Costs remained significantly greater when adjusted for covariates, with a risk ratio (95% CI) of 1.99 (1.89-2.09) when comparing with vs without baseline cirrhosis and 2.28 (2.15-2.42) when comparing with vs without progression over follow-up. Costs increased with each subsequent year, to 21% by year 6 among those with cirrhosis at baseline and 49% among those without baseline cirrhosis who progressed. The financial burden of MASH is substantial and significantly greater among those with cirrhosis or disease progression. Although patients without cirrhosis incur lower burden, the increase over time is greater and associated with progression. Therapies that slow progression may help alleviate the financial burden, and strategies are needed to identify patients with MASH at risk of progressing to cirrhosis.

The African American Dementia and Aging Project (AADAPt): An Oregon-based Longitudinal Study.

The vast majority of studies on aging, cognition, and dementia focus on non-Hispanic white subjects. This paper adds to the extant literature by providing insight into the African American aging experience. Here we describe the study design and baseline characteristics of the African American Dementia and Aging Project (AADAPt) study, which is exploring aging and cognition in African American older adults in Oregon. African American older adults (n=177) participated in AADAPt, a longitudinal study that collected data on cognitive, physical, and social functioning in annual visits since 2000. AADAPt participants had risk factors for developing dementia in future, such as hypertension and hyperlipidemia, but also reported protective factors such as high social engagement. The AADAPt project offers new insights into aging in older African Americans that includes data on cognition, social engagement, and physical health, which are crucial for understanding the experience of under-represented groups and making future studies more inclusive. These findings reflect a window of time for a geographically-focused cohort, and the lessons learned from this study likely have broader implications for shaping the health of these older African American adults. Keywords: African American, Dementia, Observational Study.

The Role of Fibroblast Activation Protein Inhibitor Positron Emission Tomography in Inflammatory and Infectious Diseases: An Updated Systematic Review.

The role of fibroblast activation protein inhibitor (FAPI) positron emission tomography/computed tomography (PET/CT) is emerging for the assessment of non-oncological diseases, such as inflammatory and infectious diseases, even if the evidence in the literature is still in its initial phases. We conducted a systematic search of Scopus, PubMed/MEDLINE, Embase, and Cochrane library databases for studies published before 31 December 2023 reporting infectious and inflammatory disease imaging with FAPI PET/CT. We included twenty-one studies for a total of 1046 patients. The most frequent disease studied was lung interstitial disease, investigated in six studies for a total of 200 patients, followed by bone and joint diseases in two studies and 185 patients, IgG4-related disease in 53 patients, and Crohn's disease in 30 patients. Despite the heterogeneity of studies in terms of study design and technical features, FAPI PET/CT showed a high detection rate and diagnostic role. Moreover, when compared with 2-[18F]FDG PET/CT (n = 7 studies), FAPI PET/CT seems to have better diagnostic performances. The presence of chronic inflammation and tissue remodeling, typical of immune-mediated inflammatory conditions, may be the underlying mechanism of FAPI uptake.

Impact of gender representativeness in online symptom survey and clinical trial participation among patients with myeloproliferative neoplasms

Patients with myeloproliferative neoplasms (MPNs) face chronic symptom burden. Online symptom assessment studies allow for recruitment of large numbers of motivated patients, but patient self-selection can lead to sampling bias. This study evaluated how gender representativeness in MPN symptom surveys and trials impacted symptom score mean estimates, using data from 4825 survey respondents and 291 trial participants with MPNs. The survey data showed that men participated at a rate roughly 50% less than what would be expected based on prevalence, and women reported higher scores than men on average for six of 10 symptoms. Together, this led to potential over estimation in six of 10 symptom score means (ranging from 5.8% to 15.3% overestimated). The trial data showed less gender-based sampling bias compared to the survey data. Studies utilizing online symptom surveys should implement study design features to recruit more men, assess for gender participation imbalances, and provide weighted estimates where appropriate.