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Introduction: Individuals’ comprehension of the information provided in consent forms should fundamentally influence whether to participate initially in a study and later whether to remain a participant. Existing evidence, however, suggests that participants do not thoroughly read, comprehend, or recall the information in consent forms. This study aimed to better understand how well participants recalled trial procedure information in the consent materials they received prior to taking part in a trial of a digital alcohol intervention. Method: This study was nested within an online effectiveness trial. The study included a contrast between two layout approaches to present the trial procedure information: one where all materials were shown on the same page (One page) and one where participants had to click on links to get materials for certain parts of the study information (Active request). Recall of trial procedures was measured 2 months post-randomization with four questions. Participants were also asked to leave a comment after each question. Result: Of the 2437 individuals who registered interest in the parent trial, 1197 were randomized to One page and 1240 were randomized to Active request. Approximately 90% consented to participate and 53% of the participants responded to the recall questionnaire. Contrasting the consent layout showed no marked differences between groups in three out of the four questions on recall of trial procedures. There was, however, evidence that recall of aspects of how personal data would be handled during the trial did differ between the two groups, with the Active request group reporting less recall than the One page group. Free-text comments were used to give nuance to the quantitative analysis. Conclusion: Participants exposed to different layouts of trial procedure information exhibited varying levels of information recall 2 months after consenting. The findings highlight the influence of the presentation of consent forms, which, This study was conducted under the auspices of the Alcohol Research Council of the Swedish Alcohol Retailing Monopoly (grant numbers 2019-0056 and 2020-0043) and the Swedish Research Council for Health, Working Life, and Welfare (grant number 2022-00193)

The gender and politics literature offers diverse views on the causes of gendered practices and the best methodologies for studying them. This article advances efforts to take stock of and systematize this diversity by grounding the feminist institutionalist perspective in critical realism. The article posits that gendered institutions are real entities with independent powers, while also emphasizing the crucial role that human ideas play in upholding and contesting gendered practices. To faithfully capture gendered institutions and their relationship with human agency, the article promotes the use of the abductive-retroductive research design. This approach allows feminist institutionalist scholars to construct and test multiple competing theories about gendered institutions, drawing from various empirical manifestations of institutional power. These expressions range from observable actions to codified rules, socially shared norms, and other subtle discourses. By shedding light on the principles at the heart of realist-oriented feminist research, this work paves the way for a more standardized and transparent approach to feminist inquiries.

The gender and politics literature offers diverse views on the causes of gendered practices and the best methodologies for studying them. This article advances efforts to take stock of and systematize this diversity by grounding the feminist institutionalist perspective in critical realism. The article posits that gendered institutions are real entities with independent powers, while also emphasizing the crucial role that human ideas play in upholding and contesting gendered practices. To faithfully capture gendered institutions and their relationship with human agency, the article promotes the use of the abductive-retroductive research design. This approach allows feminist institutionalist scholars to construct and test multiple competing theories about gendered institutions, drawing from various empirical manifestations of institutional power. These expressions range from observable actions to codified rules, socially shared norms, and other subtle discourses. By shedding light on the principles at the heart of realist-oriented feminist research, this work paves the way for a more standardized and transparent approach to feminist inquiries.

Introduction: Individuals’ comprehension of the information provided in consent forms should fundamentally influence whether to participate initially in a study and later whether to remain a participant. Existing evidence, however, suggests that participants do not thoroughly read, comprehend, or recall the information in consent forms. This study aimed to better understand how well participants recalled trial procedure information in the consent materials they received prior to taking part in a trial of a digital alcohol intervention. Method: This study was nested within an online effectiveness trial. The study included a contrast between two layout approaches to present the trial procedure information: one where all materials were shown on the same page (One page) and one where participants had to click on links to get materials for certain parts of the study information (Active request). Recall of trial procedures was measured 2 months post-randomization with four questions. Participants were also asked to leave a comment after each question. Result: Of the 2437 individuals who registered interest in the parent trial, 1197 were randomized to One page and 1240 were randomized to Active request. Approximately 90% consented to participate and 53% of the participants responded to the recall questionnaire. Contrasting the consent layout showed no marked differences between groups in three out of the four questions on recall of trial procedures. There was, however, evidence that recall of aspects of how personal data would be handled during the trial did differ between the two groups, with the Active request group reporting less recall than the One page group. Free-text comments were used to give nuance to the quantitative analysis. Conclusion: Participants exposed to different layouts of trial procedure information exhibited varying levels of information recall 2 months after consenting. The findings highlight the influence of the presentation of consent forms, which, This study was conducted under the auspices of the Alcohol Research Council of the Swedish Alcohol Retailing Monopoly (grant numbers 2019-0056 and 2020-0043) and the Swedish Research Council for Health, Working Life, and Welfare (grant number 2022-00193)

The gender and politics literature offers diverse views on the causes of gendered practices and the best methodologies for studying them. This article advances efforts to take stock of and systematize this diversity by grounding the feminist institutionalist perspective in critical realism. The article posits that gendered institutions are real entities with independent powers, while also emphasizing the crucial role that human ideas play in upholding and contesting gendered practices. To faithfully capture gendered institutions and their relationship with human agency, the article promotes the use of the abductive-retroductive research design. This approach allows feminist institutionalist scholars to construct and test multiple competing theories about gendered institutions, drawing from various empirical manifestations of institutional power. These expressions range from observable actions to codified rules, socially shared norms, and other subtle discourses. By shedding light on the principles at the heart of realist-oriented feminist research, this work paves the way for a more standardized and transparent approach to feminist inquiries.

The gender and politics literature offers diverse views on the causes of gendered practices and the best methodologies for studying them. This article advances efforts to take stock of and systematize this diversity by grounding the feminist institutionalist perspective in critical realism. The article posits that gendered institutions are real entities with independent powers, while also emphasizing the crucial role that human ideas play in upholding and contesting gendered practices. To faithfully capture gendered institutions and their relationship with human agency, the article promotes the use of the abductive-retroductive research design. This approach allows feminist institutionalist scholars to construct and test multiple competing theories about gendered institutions, drawing from various empirical manifestations of institutional power. These expressions range from observable actions to codified rules, socially shared norms, and other subtle discourses. By shedding light on the principles at the heart of realist-oriented feminist research, this work paves the way for a more standardized and transparent approach to feminist inquiries.

BACKGROUND: The hospital's physical environment can impact health and well-being. Patients spend most of their time in their hospital rooms. However, little experimental evidence supports specific physical design variables in these rooms, particularly for people poststroke. The study aimed to explore the influence of patient room design variables modeled in virtual reality using a controlled experimental design. METHODS: Adults within 3 years of stroke who had spent >2 nights in hospital for stroke and were able to consent were included (Melbourne, Australia). Using a factorial design, we immersed participants in 16 different virtual hospital patient rooms in both daytime and nighttime conditions, systematically varying design attributes: patient room occupancy, social connectivity, room size (spaciousness), noise (nighttime), greenery outlook (daytime). While immersed, participants rated their affect (Pick-A-Mood Scale) and preference. Mixed-effect regression analyses were used to explore participant responses to design variables in both daytime and nighttime conditions. Feasibility and safety were monitored throughout. Australian New Zealand Clinical Trials Registry, Trial ID: ACTRN12620000375954. RESULTS: Forty-four adults (median age, 67 [interquartile range, 57.3-73.8] years, 61.4% male, and a third with stroke in the prior 3-6 months) completed the study in 2019-2020. We recorded and analyzed 701 observations of affective responses (Pick-A-Mood Scale) in the daytime (686 at night) and 698 observations of preference responses in the daytime (685 nighttime) while continuously immersed in the virtual reality scenarios. Although single rooms were most preferred overall (daytime and nighttime), the relationship between affective responses differed in response to different combinations of nighttime noise, social connectivity, and greenery outlook (daytime). The virtual reality scenario intervention was feasible and safe for stroke participants. CONCLUSIONS: Immed

The environment is everything around us. The environment provides fresh air, clean water and productive fertile soil for the manufacture of food etc., But in the current global scenario, it has been observed that human activities are continuously and constantly impacting our mother nature in a negative way. As we navigate the complexities of the 21st century, it becomes increasingly evident that our actions today profoundly impact the world we will inhabit tomorrow. In the 21st century our environment is facing a lot of problems like climate change, depletion of ozone layer, acid rain, population explosion, loss of biodiversity due to over exploitation of nature and natural resources. Therefore, there is an urgent need and it is high time to rescue our environment thereby to maintain a harmonious relationship between man and nature in a sustainable way. In the pursuit of progress and prosperity, humanity has often overlooked the critical importance of environmental sustainability. This book, "Sustainable Development in 21st Century through Clean Environment," explores the nexus between sustainable development and environmental preservation. Our collective challenge lies in fostering development that uplifts communities without compromising the integrity of these ecosystems upon which all life depends. Through a multidisciplinary approach, this book seeks to unravel the multifaceted dimensions of sustainable development. The various chapters included in this book are from different areas of environment on the current issues of environmental challenges and possible sustainable ways to overcome those issues. We sincerely hope that this book will be a great help and support to all the students, teachers, researchers and other environmental activists who wants to do some meaningful contributions towards the environment. We would also like to express our heartfelt gratitude to our publisher Mrs. Rajani Adam for her immense love and moral support and timely help in bringin

The environment is everything around us. The environment provides fresh air, clean water and productive fertile soil for the manufacture of food etc., But in the current global scenario, it has been observed that human activities are continuously and constantly impacting our mother nature in a negative way. As we navigate the complexities of the 21st century, it becomes increasingly evident that our actions today profoundly impact the world we will inhabit tomorrow. In the 21st century our environment is facing a lot of problems like climate change, depletion of ozone layer, acid rain, population explosion, loss of biodiversity due to over exploitation of nature and natural resources. Therefore, there is an urgent need and it is high time to rescue our environment thereby to maintain a harmonious relationship between man and nature in a sustainable way. In the pursuit of progress and prosperity, humanity has often overlooked the critical importance of environmental sustainability. This book, "Sustainable Development in 21st Century through Clean Environment," explores the nexus between sustainable development and environmental preservation. Our collective challenge lies in fostering development that uplifts communities without compromising the integrity of these ecosystems upon which all life depends. Through a multidisciplinary approach, this book seeks to unravel the multifaceted dimensions of sustainable development. The various chapters included in this book are from different areas of environment on the current issues of environmental challenges and possible sustainable ways to overcome those issues. We sincerely hope that this book will be a great help and support to all the students, teachers, researchers and other environmental activists who wants to do some meaningful contributions towards the environment. We would also like to express our heartfelt gratitude to our publisher Mrs. Rajani Adam for her immense love and moral support and timely help in bringin

The environment is everything around us. The environment provides fresh air, clean water and productive fertile soil for the manufacture of food etc., But in the current global scenario, it has been observed that human activities are continuously and constantly impacting our mother nature in a negative way. As we navigate the complexities of the 21st century, it becomes increasingly evident that our actions today profoundly impact the world we will inhabit tomorrow. In the 21st century our environment is facing a lot of problems like climate change, depletion of ozone layer, acid rain, population explosion, loss of biodiversity due to over exploitation of nature and natural resources. Therefore, there is an urgent need and it is high time to rescue our environment thereby to maintain a harmonious relationship between man and nature in a sustainable way. In the pursuit of progress and prosperity, humanity has often overlooked the critical importance of environmental sustainability. This book, "Sustainable Development in 21st Century through Clean Environment," explores the nexus between sustainable development and environmental preservation. Our collective challenge lies in fostering development that uplifts communities without compromising the integrity of these ecosystems upon which all life depends. Through a multidisciplinary approach, this book seeks to unravel the multifaceted dimensions of sustainable development. The various chapters included in this book are from different areas of environment on the current issues of environmental challenges and possible sustainable ways to overcome those issues. We sincerely hope that this book will be a great help and support to all the students, teachers, researchers and other environmental activists who wants to do some meaningful contributions towards the environment. We would also like to express our heartfelt gratitude to our publisher Mrs. Rajani Adam for her immense love and moral support and timely help in bringin

INTRODUCTION: Sepsis affects 25.2 million children per year globally and causes 3.4 million deaths, with an annual cost of hospitalisation in the USA of US$7.3 billion. Despite being common, severe and expensive, therapies and outcomes from sepsis have not substantially changed in decades. Variable case definitions, lack of a reference standard for diagnosis and broad spectrum of disease hamper efforts to evaluate therapies that may improve sepsis outcomes. This landscape analysis of community-acquired childhood sepsis in Australia and New Zealand will characterise the burden of disease, including incidence, severity, outcomes and cost. Sepsis diagnostic criteria and risk stratification tools will be prospectively evaluated. Sepsis therapies, quality of care, parental awareness and understanding of sepsis and parent-reported outcome measures will be described. Understanding these aspects of sepsis care is fundamental for the design and conduct of interventional trials to improve childhood sepsis outcomes. METHODS AND ANALYSIS: This prospective observational study will include children up to 18 years of age presenting to 12 emergency departments with suspected sepsis within the Paediatric Research in Emergency Departments International Collaborative network in Australia and New Zealand. Presenting characteristics, management and outcomes will be collected. These will include vital signs, serum biomarkers, clinician assessment of severity of disease, intravenous fluid administration for the first 24 hours of hospitalisation, organ support therapies delivered, antimicrobial use, microbiological diagnoses, hospital and intensive care unit length-of-stay, mortality censored at hospital discharge or 30 days from enrolment (whichever comes first) and parent-reported outcomes 90 days from enrolment. We will use these data to determine sepsis epidemiology based on existing and novel diagnostic criteria. We will also validate existing and novel sepsis risk stratification crit

Research stands as a fundamental pillar that recognizes the university as an entity of institutional and social relevance. This statement is particularly true in the field of health sciences, where educational institutions are expected not only to generate knowledge for the benefit of society but also to shape individuals capable of immersing themselves in research and strengthening this vital premise. In this context, intricate concepts that outline the nature and purpose of research in the academia emerge. Scientific research, on the one hand, arises as the driving force behind the advancement of knowledge, emphasizing rigorous and systematic methods to unravel the mysteries of reality. On the other hand, research training stands as a pivotal educational vehicle for nurturing analytical and curious minds, providing future professionals with the necessary tools to inquire and contribute to scientific progress. Similarly, formative research, although less known, plays a crucial role in integrating research into the learning experience, which fosters critical thinking skills and problem-solving abilities from early stages. In this educational and research journey, various actors and components take center stage. Research centers arise as fundamental pillars, as they provide the conducive environment to interdisciplinary collaboration and development of innovative ideas. Educators, on their part, play the role of counselors and mentors by sharing their experience and knowledge with the new generations of professionals. Furthermore, extracurricular or nonacademic activities in the area of research emerge as fertile grounds for the expansion of intellectual horizons, offering spaces for creative exploration and practical application of the knowledge acquired by students.However, these concepts and components are not free of challenges and controversies when integrating into the academic programs. Implementing these approaches at the undergraduate and graduate levels req, La investigación se erige como un pilar fundamental que distingue a la universidad como una entidad de relevancia institucional y social. Esta afirmación es particularmente verídica en el ámbito de las ciencias de la salud, donde no solo se espera que las instituciones educativas generen conocimiento en beneficio de la sociedad, sino que también moldeen individuos capacitados para sumergirse en la investigación y fortalecer esta premisa vital. En este contexto, emergen conceptos intrincados que delinean la naturaleza y el propósito de la investigación en el ámbito académico. La investigación científica, por un lado, se alza como el motor principal que impulsa el avance del conocimiento, y pone énfasis en el método riguroso y sistemático para desentrañar los misterios de la realidad. Por otro lado, la formación para la investigación se erige como un vehículo educativo primordial para cultivar mentes analíticas y curiosas, que dota a los futuros profesionales con las herramientas necesarias para indagar y contribuir al progreso científico. Asimismo, la investigación formativa, si bien menos conocida, desempeña un rol crucial al integrar la investigación en la experiencia de aprendizaje, lo cual fomenta habilidades de pensamiento crítico y resolución de problemas desde etapas tempranas. En esta travesía educativa e investigativa, diversos actores y componentes adquieren protagonismo. Los institutos de investigación se presentan como pilares fundamentales, ya que proporcionan el ambiente propicio para la colaboración interdisciplinaria y el florecimiento de ideas innovadoras. Los docentes, por su parte, ejercen un rol de guías y mentores, transmitiendo su experiencia y conocimientos a las nuevas generaciones de profesionales. Además, las actividades extracurriculares o extraprogramáticas en el área de la investigación surgen como terrenos fértiles para la expansión de horizontes intelectuales, que ofrecen espacios para la exploración creativa y la aplicación práctica de

Background: university social responsibility constitutes an academic management approach that promotes sustainable social development.Objective: to analyze knowledge management in the Department of Basic Biomedical Sciences, of the "Salvador Allende" Faculty of Medical Sciences, from 2018 to 2022, based on university social responsibility.Methods: a descriptive investigation was developed, with a qualitative approach, complementing the methodologies of the case study and the constructivist grounded theory. Theoretical methods were used: analysis-synthesis, induction-deduction, ascent from the abstract to the concrete and historical logic; and empirical ones: documentary review, content analysis and focal group.Results: the analysis carried out showed that knowledge management in the Department of Basic Biomedical Sciences, of the aforementioned faculty, has a strategic approach and has had a positive educational impact as a result of organizational learning, and it´s linked to academic management.Conclusions: the departmental knowledge management determined an update of the organizational culture, causing a favorable social impact, mediated by an educational intervention tributary of a relevant professional training., Fundamento: la responsabilidad social universitaria constituye un enfoque de gestión académica promotor del desarrollo social sostenible.Objetivo: analizar la gestión del conocimiento en el departamento de Ciencias Básicas Biomédicas, de la Facultad de Ciencias Médicas “Salvador Allende”, durante los años 2018 al 2022, sobre la base de la responsabilidad social universitaria.Métodos: se desarrolló una investigación descriptiva, con enfoque cualitativo, complementando las metodologías del estudio de caso y de la teoría fundamentada constructivista. Se utilizaron métodos teóricos: análisis–síntesis, inducción-deducción, ascenso de lo abstracto a lo concreto e histórico lógico; y empíricos: revisión documental, análisis de contenido y grupo focal.Resultados: el análisis realizado evidenció que la gestión del conocimiento en el departamento de Ciencias Básicas Biomédicas, de la referida facultad, ostenta un enfoque estratégico y ha tenido un impacto educativo positivo como resultado del aprendizaje organizacional, y está vinculado con la gestión académica.Conclusiones: la gestión del conocimiento departamental determinó una actualización de la cultura organizacional, causante de un impacto social favorable, mediado por una intervención educativa tributaria de una formación profesional pertinente.

This article describes the rationale, aims, and methodology of the Accelerating Medicines Partnership® Schizophrenia (AMP® SCZ). This is the largest international collaboration to date that will develop algorithms to predict trajectories and outcomes of individuals at clinical high risk (CHR) for psychosis and to advance the development and use of novel pharmacological interventions for CHR individuals. We present a description of the participating research networks and the data processing analysis and coordination center, their processes for data harmonization across 43 sites from 13 participating countries (recruitment across North America, Australia, Europe, Asia, and South America), data flow and quality assessment processes, data analyses, and the transfer of data to the National Institute of Mental Health (NIMH) Data Archive (NDA) for use by the research community. In an expected sample of approximately 2000 CHR individuals and 640 matched healthy controls, AMP SCZ will collect clinical, environmental, and cognitive data along with multimodal biomarkers, including neuroimaging, electrophysiology, fluid biospecimens, speech and facial expression samples, novel measures derived from digital health technologies including smartphone-based daily surveys, and passive sensing as well as actigraphy. The study will investigate a range of clinical outcomes over a 2-year period, including transition to psychosis, remission or persistence of CHR status, attenuated positive symptoms, persistent negative symptoms, mood and anxiety symptoms, and psychosocial functioning. The global reach of AMP SCZ and its harmonized innovative methods promise to catalyze the development of new treatments to address critical unmet clinical and public health needs in CHR individuals.

The use of the p-value in quantitative research, particularly its threshold of P < 0.05 for determining statistical significance, has long been a cornerstone of statistical analysis in research. However, this standard has been increasingly scrutinized for its potential to mislead findings, especially when the practical significance, the number of comparisons, or the suitability of statistical tests are not properly considered. In response to controversy around use of p-values, the American Statistical Association published a statement in 2016 that challenged the research community to abandon the term statistically significant. This stance has been echoed by leading scientific journals to urge a significant reduction or complete elimination in the reliance on p-values when reporting results. To provide guidance to researchers in health professions education, this paper provides a succinct overview of the ongoing debate regarding the use of p-values and the definition of p-values. It reflects on the controversy by highlighting the common pitfalls associated with p-value interpretation and usage, such as misinterpretation, overemphasis, and false dichotomization between significant and non-significant results. This paper also outlines specific recommendations for the effective use of p-values in statistical reporting including the importance of reporting effect sizes, confidence intervals, the null hypothesis, and conducting sensitivity analyses for appropriate interpretation. These considerations aim to guide researchers toward a more nuanced and informative use of p-values.

Background Diffuse midline glioma (DMG) is a devastating pediatric brain tumor unresponsive to hundreds of clinical trials. Approximately 80% of DMGs harbor H3K27M oncohistones, which reprogram the epigenome to increase the metabolic profile of the tumor cells. Methods We have previously shown preclinical efficacy of targeting both oxidative phosphorylation and glycolysis through treatment with ONC201, which activates the mitochondrial protease ClpP, and paxalisib, which inhibits PI3K/mTOR, respectively. Results ONC201 and paxalisib combination treatment aimed at inducing metabolic distress led to the design of the first DMG-specific platform trial PNOC022 (NCT05009992). Conclusions Here, we expand on the PNOC022 rationale and discuss various considerations, including liquid biome, microbiome, and genomic biomarkers, quality-of-life endpoints, and novel imaging modalities, such that we offer direction on future clinical trials in DMG.

INTRODUCTION: Person-centred care (PCC) is provision of care that is respectful of and responsive to individual patient preferences, needs and values, and ensures that patient values guide all clinical decisions. While there is a large body of evidence on the benefits of PCC in high-income countries, little research exists on PCC in Ghana and Sub-Saharan Africa at large. Most studies on PCC have focused on maternity care as part of the global movement of respectful maternity care. The few studies on patient experiences and health system responsiveness beyond maternal health also highlight gaps in patient experience and satisfaction as well as discrimination in health facilities, which leads to the most vulnerable having the poorest experiences. The protocol for this scoping review aims to systematically map the extent of literature focused on PCC in Ghana by identifying patient expectations and preferences, barriers and facilitators, and interventions. METHODS AND ANALYSIS: The protocol will be guided by the Arksey and OMalley methodological framework and recommendations by Levac et al. A comprehensive search strategy will be used to search for published articles in PubMed, EMBASE, Web of Science and the African Journals Online from their inception to August 2022. Grey literature and reference lists of included studies will also be searched. Two independent reviewers will perform the literature search, eligibility assessments and study selection. Any disagreements will be resolved through discussion with a third reviewer. A Preferred Reporting Items for Systematic Reviews and Meta-Analyses flow diagram for the scoping reviews will be used to outline the study selection process. Extracted data from the included articles will be synthesised and reported under key concepts derived from the outcomes of the scoping review. ETHICS AND DISSEMINATION: This scoping review does not require ethical approval. The findings will be disseminated through publications and conference

Medical Respite Programs (MRPs) characterize a care model that has been developed to address the health care and social needs of persons experiencing homelessness by providing post-acute hospital care in a safe environment. Although this model has been shown to reduce hospitalizations, improve health outcomes and increase access to health services, prior studies of MRP programs and outcomes have been limited to individual sites and may not generalize to the population of individuals receiving MRP care. This study protocol describes a mixed method design to collect organizational, provider, and patient-level data from a sample of MRPs.

IMPORTANCE: The prevalence, pathophysiology, and long-term outcomes of COVID-19 (post-acute sequelae of SARS-CoV-2 [PASC] or Long COVID) in children and young adults remain unknown. Studies must address the urgent need to define PASC, its mechanisms, and potential treatment targets in children and young adults. OBSERVATIONS: We describe the protocol for the Pediatric Observational Cohort Study of the NIHs REsearching COVID to Enhance Recovery (RECOVER) Initiative. RECOVER-Pediatrics is an observational meta-cohort study of caregiver-child pairs (birth through 17 years) and young adults (18 through 25 years), recruited from more than 100 sites across the US. This report focuses on two of four cohorts that comprise RECOVER-Pediatrics: 1) a de novo RECOVER prospective cohort of children and young adults with and without previous or current infection; and 2) an extant cohort derived from the Adolescent Brain Cognitive Development (ABCD) study (n = 10,000). The de novo cohort incorporates three tiers of data collection: 1) remote baseline assessments (Tier 1, n = 6000); 2) longitudinal follow-up for up to 4 years (Tier 2, n = 6000); and 3) a subset of participants, primarily the most severely affected by PASC, who will undergo deep phenotyping to explore PASC pathophysiology (Tier 3, n = 600). Youth enrolled in the ABCD study participate in Tier 1. The pediatric protocol was developed as a collaborative partnership of investigators, patients, researchers, clinicians, community partners, and federal partners, intentionally promoting inclusivity and diversity. The protocol is adaptive to facilitate responses to emerging science. CONCLUSIONS AND RELEVANCE: RECOVER-Pediatrics seeks to characterize the clinical course, underlying mechanisms, and long-term effects of PASC from birth through 25 years old. RECOVER-Pediatrics is designed to elucidate the epidemiology, four-year clinical course, and sociodemographic correlates of pediatric PASC. The data and biosamples will allow

PURPOSE: Treatment guidelines for gender-affirming hormone therapy with estrogen (GAHT-E) recommend specific dosing regimens based on limited data. Well-controlled efficacy trials are essential to tailoring treatment to patient goals as the guidelines recommend. The goal of this study was to take a foundational step toward designing community-centered effectiveness trials for gender-diverse individuals seeking GAHT-E. METHODS: Our team developed a cross-sectional survey based on broad clinical experience and consultation with our community advisory board. The survey included 60 items covering demographics, transition history, goals and priorities for treatment, indicators of treatment success, sexual function goals, and future research priorities. The survey was distributed during the summer of 2021, primarily through social networks designed for gender-expansive individuals seeking treatment with estrogen. RESULTS: A total of 1270 individuals completed the survey. Overall treatment goals most frequently rated extremely important or very important were the following: (1) improved satisfaction with life (81%), (2) appearing more feminine (80%), (3) appearing less masculine (77%), (4) improved mental health (76%), and (5) being seen as your true gender by others (75%). The three body characteristics most frequently rated highest priority or high priority among changes were the following: (1) facial hair (85%), (2) breast shape or size (84%), and (3) body shape (80%). The highest-rated research priority was comparing feminization with different routes of estrogen administration. CONCLUSION: The goals and experiences of individuals seeking GAHT-E are diverse. Future clinical trials of GAHT-E should be grounded in the needs and priorities of community stakeholders.

The biomedical research community addresses reproducibility challenges in animal studies through standardized nomenclature, improved experimental design, transparent reporting, data sharing, and centralized repositories. The ARRIVE guidelines outline documentation standards for laboratory animals in experiments, but genetic information is often incomplete. To remedy this, we propose the Laboratory Animal Genetic Reporting (LAG-R) framework. LAG-R aims to document animals genetic makeup in scientific publications, providing essential details for replication and appropriate model use. While verifying complete genetic compositions may be impractical, better reporting and validation efforts enhance reliability of research. LAG-R standardization will bolster reproducibility, peer review, and overall scientific rigor.

Qualitative research seeks to provide context, nuance, and depth of understanding in regard to systems, behaviors, and/or lived experiences. As such, it plays a key role in many areas of medical education. Composed of myriad methods and methodologies, each of which may be valuable for some areas of inquiry but less so for others, qualitative research can be challenging to design, conduct, and report. This challenge can be conceptualized as ensuring that the study design, conduct, and reporting are fit for purpose, following directly from a well-formulated research question. In this Perspective, we share seven important and practical recommendations to enhance the design and conduct of high-quality qualitative research in medical education: 1) craft a strong research question, 2) link the study design to this question, 3) assemble a team with diverse expertise, 4) prioritize information power when selecting recruitment and sampling strategies, 5) collect data carefully, 6) rigorously analyze data, and 7) disseminate results that tell a complete story.

ObjectivesUnhealthy diets and inadequate exercise are associated with chronic health conditions and excess mortality. Older African Americans do not meet dietary and exercise guidelines, and this may have worsened during the COVID-19 pandemic due to individual and environmental factors, including food insecurity. Studies evaluating these dynamics are essential for developing interventions. This narrative details a study protocol and data collection experiences during the pandemic.MethodsParticipants > 55 years African American old completed detailed food frequency, exercise, and food access questionnaires between October 2020 and July 2021. Observations of the study administrators (authors of this manuscript) for the duration of the study are presented. Details on the study design and reflections on the opportunities, challenges, and lessons learned are summarized. Future manuscripts will report data analysis of study findings.ResultsA total of 123 older African American adults participated in the study, and 118 (70% female) completed all three questionnaires. More than 50% of the participants had at least two primary chronic conditions. About 85% were fully vaccinated against COVID-19. Applying community-based participatory approaches, leveraging partnerships, and exercising flexibility approaches were pivotal to successfully implementing the study protocol.ConclusionsDespite challenges related to the COVID-19 pandemic, detailed data on older African American adults' diet and exercise habits were obtained. Our study design and experiences will benefit future researchers. More importantly, results from our study will inform interventions and policies aimed at minimizing consequences associated with poor diet and exercise habits during the pandemic among this vulnerable population.

BACKGROUND: Primary sclerosing cholangitis (PSC) is a progressive immune-mediated liver disease, for which no medical therapy has been shown to slow disease progression. However, the horizon for new therapies is encouraging, with several innovative clinical trials in progress. Despite these advancements, there is considerable heterogeneity in the outcomes studied, with lack of consensus as to what outcomes to measure, when to measure and how to measure. Furthermore, there has been a paradigm shift in PSC treatment targets over recent years, moving from biochemistry-based endpoints to histological assessment of liver fibrosis, imaging-based biomarkers and patient-reported outcome measures. The abundance of new interventional trials and evolving endpoints pose opportunities for all stakeholders involved in evaluating novel therapies. To this effect, there is a need to harmonise measures used in clinical trials through the development of a core outcome set (COS). METHODS AND ANALYSIS: Synthesis of a PSC-specific COS will be conducted in four stages. Initially, a systematic literature review will be performed to identify outcomes previously used in PSC trials, followed by semistructured qualitative interviews conducted with key stakeholders. The latter may include patients, clinicians, researchers, pharmaceutical industry representatives and healthcare payers and regulatory agencies, to identify additional outcomes of importance. Using the outcomes generated from the literature review and stakeholder interviews, an international two-round Delphi survey will be conducted to prioritise outcomes for inclusion in the COS. Finally, a consensus meeting will be convened to ratify the COS and disseminate findings for application in future PSC trials. ETHICS AND DISSEMINATION: Ethical approval has been granted by the East Midlands-Leicester Central Research Ethics Committee (Ref: 24/EM/0126) for this study. The COS from this study will be widely disseminated including publicatio

Cluster randomized trials (CRTs) often enroll large numbers of participants; yet due to resource constraints, only a subset of participants may be selected for outcome assessment, and those sampled may not be representative of all cluster members. Missing data also present a challenge: if sampled individuals with measured outcomes are dissimilar from those with missing outcomes, unadjusted estimates of arm-specific endpoints and the intervention effect may be biased. Further, CRTs often enroll and randomize few clusters, limiting statistical power and raising concerns about finite sample performance. Motivated by SEARCH-TB, a CRT aimed at reducing incident tuberculosis infection, we demonstrate interlocking methods to handle these challenges. First, we extend Two-Stage targeted minimum loss-based estimation to account for three sources of missingness: (i) subsampling; (ii) measurement of baseline status among those sampled; and (iii) measurement of final status among those in the incidence cohort (persons known to be at risk at baseline). Second, we critically evaluate the assumptions under which subunits of the cluster can be considered the conditionally independent unit, improving precision and statistical power but also causing the CRT to behave like an observational study. Our application to SEARCH-TB highlights the real-world impact of different assumptions on measurement and dependence; estimates relying on unrealistic assumptions suggested the intervention increased the incidence of TB infection by 18% (risk ratio [RR]=1.18, 95% confidence interval [CI]: 0.85-1.63), while estimates accounting for the sampling scheme, missingness, and within community dependence found the intervention decreased the incident TB by 27% (RR=0.73, 95% CI: 0.57-0.92).

Difference-in-differences (DiD) is a powerful, quasi-experimental research design widely used in longitudinal policy evaluations with health outcomes. However, DiD designs face several challenges to ensuring reliable causal inference, such as when policy settings are more complex. Recent economics literature has revealed that DiD estimators may exhibit bias when heterogeneous treatment effects, a common consequence of staggered policy implementation, are present. To deepen our understanding of these advancements in epidemiology, in this methodologic primer, we start by presenting an overview of DiD methods. We then summarize fundamental problems associated with DiD designs with heterogeneous treatment effects and provide guidance on recently proposed heterogeneity-robust DiD estimators, which are increasingly being implemented by epidemiologists. We also extend the discussion to violations of the parallel trends assumption, which has received less attention. Last, we present results from a simulation study that compares the performance of several DiD estimators under different scenarios to enhance understanding and application of these methods.

This research, done as part of the mechanical engineering program at Linnaeus University in partnership with Rottne Industri AB, aims to improve safety in forest industri. It focuses on designing a user-friendly emergency exit system for harvester cabins. Driven by strict safety rules and societal expectations, the goal is to craft an exit that allows quick evacuation during emergencies and fits well with forest machinery safety standards.

BACKGROUND: The hospital's physical environment can impact health and well-being. Patients spend most of their time in their hospital rooms. However, little experimental evidence supports specific physical design variables in these rooms, particularly for people poststroke. The study aimed to explore the influence of patient room design variables modeled in virtual reality using a controlled experimental design. METHODS: Adults within 3 years of stroke who had spent >2 nights in hospital for stroke and were able to consent were included (Melbourne, Australia). Using a factorial design, we immersed participants in 16 different virtual hospital patient rooms in both daytime and nighttime conditions, systematically varying design attributes: patient room occupancy, social connectivity, room size (spaciousness), noise (nighttime), greenery outlook (daytime). While immersed, participants rated their affect (Pick-A-Mood Scale) and preference. Mixed-effect regression analyses were used to explore participant responses to design variables in both daytime and nighttime conditions. Feasibility and safety were monitored throughout. Australian New Zealand Clinical Trials Registry, Trial ID: ACTRN12620000375954. RESULTS: Forty-four adults (median age, 67 [interquartile range, 57.3-73.8] years, 61.4% male, and a third with stroke in the prior 3-6 months) completed the study in 2019-2020. We recorded and analyzed 701 observations of affective responses (Pick-A-Mood Scale) in the daytime (686 at night) and 698 observations of preference responses in the daytime (685 nighttime) while continuously immersed in the virtual reality scenarios. Although single rooms were most preferred overall (daytime and nighttime), the relationship between affective responses differed in response to different combinations of nighttime noise, social connectivity, and greenery outlook (daytime). The virtual reality scenario intervention was feasible and safe for stroke participants. CONCLUSIONS: Immed

BACKGROUND: The hospital's physical environment can impact health and well-being. Patients spend most of their time in their hospital rooms. However, little experimental evidence supports specific physical design variables in these rooms, particularly for people poststroke. The study aimed to explore the influence of patient room design variables modeled in virtual reality using a controlled experimental design. METHODS: Adults within 3 years of stroke who had spent >2 nights in hospital for stroke and were able to consent were included (Melbourne, Australia). Using a factorial design, we immersed participants in 16 different virtual hospital patient rooms in both daytime and nighttime conditions, systematically varying design attributes: patient room occupancy, social connectivity, room size (spaciousness), noise (nighttime), greenery outlook (daytime). While immersed, participants rated their affect (Pick-A-Mood Scale) and preference. Mixed-effect regression analyses were used to explore participant responses to design variables in both daytime and nighttime conditions. Feasibility and safety were monitored throughout. Australian New Zealand Clinical Trials Registry, Trial ID: ACTRN12620000375954. RESULTS: Forty-four adults (median age, 67 [interquartile range, 57.3-73.8] years, 61.4% male, and a third with stroke in the prior 3-6 months) completed the study in 2019-2020. We recorded and analyzed 701 observations of affective responses (Pick-A-Mood Scale) in the daytime (686 at night) and 698 observations of preference responses in the daytime (685 nighttime) while continuously immersed in the virtual reality scenarios. Although single rooms were most preferred overall (daytime and nighttime), the relationship between affective responses differed in response to different combinations of nighttime noise, social connectivity, and greenery outlook (daytime). The virtual reality scenario intervention was feasible and safe for stroke participants. CONCLUSIONS: Immed

This research, done as part of the mechanical engineering program at Linnaeus University in partnership with Rottne Industri AB, aims to improve safety in forest industri. It focuses on designing a user-friendly emergency exit system for harvester cabins. Driven by strict safety rules and societal expectations, the goal is to craft an exit that allows quick evacuation during emergencies and fits well with forest machinery safety standards.

Background: Because poor health in youth risk affecting their entry in adulthood, improved methods for their early identification are needed. Health and welfare technology is widely accepted by youth populations, presenting a potential method for identifying their health problems. However, healthcare technology must be evidence-based. Specifically, feasibility studies contribute valuable information prior to more complex effects-based research. The current study assessed the process, resource, management, and scientific feasibility of the Youth Health Report System prototype, developed within a youth health clinic context in advance of an intervention study. Methods: This mixed-methods feasibility study was conducted in a clinical setting. The process, resource, management, and scientific feasibility of the Youth Health Report System were investigated, as recommended in the literature. Participants were youth aged 16–23 years old, attending a youth health clinic, and healthcare professionals from three clinics. The youth participants used their smart phones to respond to Youth Health Report System health questions and healthcare professionals used their computer to access the results and for registration system entries. Qualitative data were collected from interviews with healthcare professionals, which were described with thematic analysis. Youth participants’ quantitative Youth Health Report System data were analyzed for descriptive statistics. Results: Feasibility analysis of qualitative data from interviews with 11 healthcare professionals resulted in three themes: We expected it could be hard; Information and routines helped but time was an issue; and The electronic case report form was valuable in the health assessment. Qualitative data were collected from the Youth Health Report System. A total of 54 youth participants completed the evaluation questionnaire, and healthcare professionals retrieved information from, and made post-appointment system entries. Qua, Article; Export Date: 20 March 2024; Cited By: 0; Correspondence Address: P.V. Lostelius; Centre for Innovation, Research and Education, Region Västmanland, Västmanland Hospital Västerås, Västerås, Sweden; email: petra.lostelius@regionvastmanland.se

Background: Heart failure is a global problem affecting millions of people worldwide. Current care of heart failure patients follows standard protocols and often overlooks the patients’ specific needs, which leads to low compliance in the rehabilitation phase. Telerehabilitation, where the patients communicate with health care professionals about their rehabilitation program and monitor their vital signs, aims to increase the degree of compliance as well as enhancing their quality of life. Objective: The aim of this study is to investigate whether application of the Future Patient Telerehabilitation Program II can improve the health-related quality of life for patients with heart failure. Methods: A randomized controlled trial will be conducted. A total of 70 patients will be enrolled, 35 in the intervention group, 35 in the control group. The intervention group will follow an add-on to traditional care, while the control group will follow the conventional Danish cardiac rehabilitation program, which is based on periodic visits to the clinic. The patients will be followed for a period of six months. The intervention group will have access to an online HeartPortal and will use various home-based devices for self-monitoring. The primary outcome to be investigated is health-related quality of life as measured by the EuroQol-5 Dimension. Secondary outcomes are the number of visits to the outpatient clinic, number of readmissions and number of tele-communications contacts (phone and video) with health care professionals. The primary and secondary outcomes will be assessed using questionnaires and through the data generated by digital technologies for self-monitoring. Results: Enrolment began in August 2020. The results will be published in peer-reviewed journals. Results from the Future Patient II Telerehabilitation program are expected to be published in 2024. Discussion: This study is a further development of the Future Patient Telerehabilitation I study, and it is

Background: Studies show considerable variability in the definitions and operationalization of child maltreatment (CM), which limits research, policy formation, surveillance, and cross-country and cross-sector comparisons. Objective: To review the recent literature (2011–2021) to understand current issues and challenges in defining CM, to assist in the planning, testing and implementing of CM conceptualizations. Methods: We searched eight international databases. Articles were included if the substantive content was related to issues, challenges, and debates in defining CM, and the article was an original study, review, commentary, report, or guideline. The review followed methodological guidance for the conduct of scoping reviews and was reported in accordance with the PRISMA-ScR checklist. Four experts in CM conducted a thematic analysis to summarize findings. Methodological rigor of the included studies was not formally assessed. Results: We identified 7372 potentially relevant articles; 55 full-text studies were assessed for eligibility, 25 satisfied the inclusion criteria. We identified three themes: 1) strategies to define CM, including the integration of child and victim perspectives; 2) difficulties in defining specific CM types; and 3) real-world implications for research, prevention and policy. Conclusions: Despite longstanding concerns, challenges regarding the definitions of CM persist. A small minority of studies have tested and implemented CM definitions and operationalizations in practice. The findings will inform international multi-sectoral processes to develop uniform definitions of CM, for example by highlighting the need to acknowledge challenges in defining some CM types and emphasizing the importance of considering the perspectives of children and CM survivors.

Introduction: Childhood sexual abuse (CSA) is a global public health problem with potentially severe health and mental health consequences. Healthcare professionals (HCPs) should be familiar with risk factors and potential indicators of CSA, and able to provide appropriate medical management. The WHO issued global guidelines for the clinical care of children with CSA, based on rigorous review of the evidence base. The current systematic review identifies existing CSA guidelines issued by government agencies and academic societies in the European Region and assesses their quality and clarity to illuminate strengths and identify opportunities for improvement. Methods and analysis: This 10-database systematic review will be conducted according to the Centre for Reviews and Dissemination guidelines and will be reported according to The Preferred Reporting Items for Systematic Reviews and Meta-Analyses statement. Guidance for HCPs regarding CSA, written by a national governmental agency or academic society of HCPs within 34 COST Action 19106 Network Countries (CANC) and published in peer-reviewed or grey literature between January 2012 and November 2022, is eligible for inclusion. Two independent researchers will search the international literature, screen, review and extract data. Included guidelines will be assessed for completeness and clarity, compared with the WHO 2017/2019 guidelines on CSA, and evaluated for consistency between the CANC guidelines. The Appraisal of Guidelines for Research and Evaluation II tool and Grading of Recommendations Assessment, Development and Evaluation methodology will be used to evaluate CANC guidelines. Descriptive statistics will summarise content similarities and differences between the WHO guidelines and national guidelines; data will be summarised using counts, frequencies, proportions and per cent agreement between country-specific guidelines and the WHO 2017/2019 guidelines. Ethics and dissemination: There are no individuals or

The COVID-19 pandemic has highlighted a debate about whether marginalized communities suffered the disproportionate brunt of the pandemic's mortality. Empirical studies addressing this question typically suffer from statistical uncertainties and potential biases associated with uneven and incomplete reporting. We use geo-coded micro-level data for the entire population of Sweden to analyze how local neighborhood characteristics affect the likelihood of dying with COVID-19 at individual level, given the individual's overall risk of death. We control for several individual and regional characteristics to compare the results in specific communities to overall death patterns in Sweden during 2020. When accounting for the probability to die of any cause, we find that individuals residing in socioeconomically disadvantaged neighborhoods were not more likely to die with COVID-19 than individuals residing elsewhere. Importantly, we do find that individuals show a generally higher probability of death in these neighborhoods. Nevertheless, ethnicity is an important explanatory factor for COVID-19 deaths for foreign-born individuals, especially from East Africa, who are more likely to pass away regardless of residential neighborhood.

This second Part of the paper (i) develops a detailed procedural model for handling complicated research problematics, (ii) transfers the procedural framework into a scenario of processing steps, (iii) demonstrates the deployment of the procedural scenario in a sample case, and (iv) addresses some important issues of research model development for supradisciplinary research. First, the framework called Holistic Systematic Combinatorial Scoping (HSCS) is introduced that comprises 21 subsequent activity steps. To illustrate the practical application of the HSCS framework, a demonstrative case study of preventing frequently occurring accidents at an uncontrolled intersection is presented. This case-study explains how the HSCS framework can facilitate (i) semantic capturing and rendering of complicated research problematics, (ii) scoping the overall research problematics and constructing definitive research problematics, and (iii) deriving a manageable research model of definitive research problematics. Further research intends to test the methodology by transdisciplinary research collectives and other CPSs related problematics., System Engineering, Cyber-Physical Systems

BACKGROUND: Recently revised WHO guidelines on malaria chemoprevention have opened the door to more tailored implementation. Countries face choices on whether to replace old drugs, target additional age groups, and adapt delivery schedules according to local drug resistance levels and malaria transmission patterns. Regular routine assessment of protective efficacy of chemoprevention is key. Here, we apply a novel modelling approach to aid the design and analysis of chemoprevention trials and generate measures of protection that can be applied across a range of transmission settings.METHODS AND FINDINGS: We developed a model of genotype-specific drug protection, which accounts for underlying risk of infection and circulating genotypes. Using a Bayesian framework, we fitted the model to multiple simulated scenarios to explore variations in study design, setting, and participant characteristics. We find that a placebo or control group with no drug protection is valuable but not always feasible. An alternative approach is a single-arm trial with an extended follow-up (>42 days), which allows measurement of the underlying infection risk after drug protection wanes, as long as transmission is relatively constant. We show that the currently recommended 28-day follow-up in a single-arm trial results in low precision of estimated 30-day chemoprevention efficacy and low power in determining genotype differences of 12 days in the duration of protection (power = 1.4%). Extending follow-up to 42 days increased precision and power (71.5%) in settings with constant transmission over this time period. However, in settings of unstable transmission, protective efficacy in a single-arm trial was overestimated by 24.3% if recruitment occurred during increasing transmission and underestimated by 15.8% when recruitment occurred during declining transmission. Protective efficacy was estimated with greater precision in high transmission settings, and power to detect difference

BACKGROUND: Recently revised WHO guidelines on malaria chemoprevention have opened the door to more tailored implementation. Countries face choices on whether to replace old drugs, target additional age groups, and adapt delivery schedules according to local drug resistance levels and malaria transmission patterns. Regular routine assessment of protective efficacy of chemoprevention is key. Here, we apply a novel modelling approach to aid the design and analysis of chemoprevention trials and generate measures of protection that can be applied across a range of transmission settings.METHODS AND FINDINGS: We developed a model of genotype-specific drug protection, which accounts for underlying risk of infection and circulating genotypes. Using a Bayesian framework, we fitted the model to multiple simulated scenarios to explore variations in study design, setting, and participant characteristics. We find that a placebo or control group with no drug protection is valuable but not always feasible. An alternative approach is a single-arm trial with an extended follow-up (>42 days), which allows measurement of the underlying infection risk after drug protection wanes, as long as transmission is relatively constant. We show that the currently recommended 28-day follow-up in a single-arm trial results in low precision of estimated 30-day chemoprevention efficacy and low power in determining genotype differences of 12 days in the duration of protection (power = 1.4%). Extending follow-up to 42 days increased precision and power (71.5%) in settings with constant transmission over this time period. However, in settings of unstable transmission, protective efficacy in a single-arm trial was overestimated by 24.3% if recruitment occurred during increasing transmission and underestimated by 15.8% when recruitment occurred during declining transmission. Protective efficacy was estimated with greater precision in high transmission settings, and power to detect difference

166 p., ill., APPENDIX REPORT 1 -- TABLE OF CONTENTS 1 -- APPENDIX 1. GREY LITERATURE REVIEWS 3 -- APPENDIX 1.1. SCREENED WEBSITES 3 -- APPENDIX 1.2. OVERVIEW LITERATURE 11 -- APPENDIX 2. RAPID REVIEW 27 -- APPENDIX 2.1. SEARCH STRING 28 -- APPENDIX 2.2. EXCLUDED ARTICLES BASED ON FULL-TEXT SCREENING 29 -- APPENDIX 2.3. DATA EXTRACTION FORM 31 -- APPENDIX 2.4. SUMMARY TABLE – INCLUDED STUDIES 35 -- Appendix 2.4.1. Literature based studies 35 -- Appendix 2.4.2. Quantitative studies 39 -- Appendix 2.4.3. Qualitative studies 56 -- Appendix 2.4.4. Mixed-method studies 65 -- APPENDIX 2.5. OVERVIEW OF TOPICS STUDIED PER TYPE OF METHOD 71 -- APPENDIX 2.6. SPECIFIC SCALES/ VALIDATED INSTRUMENTS FOR RARE DISEASE 78 -- APPENDIX 2.7. PROPOSED NEW INDICATORS AND RESPECTIVE SUGGESTIONS FOR PATIENT QUESTIONNAIRE 84 -- APPENDIX 3. STAKEHOLDER WORKSHOPS 86 -- APPENDIX 3.1. APPENDIX 3.1. SURVEY 86 -- Appendix 3.1.1. Appendix 3.1.1. Workshop 1 86 -- Appendix 3.1.2. Workshop 2 132 -- APPENDIX 3.2. WORKSHOP GUIDE 141 -- Appendix 3.2.1. Workshop 1 141 -- Appendix 3.2.2. Workshop 2 145 -- APPENDIX 3.3. CODE-TREE 149 -- APPENDIX 3.3.1 Stakeholder consultation 1 149 -- Appendix 3.2.3. Stakeholder consultation 2 150 -- REFERENCES 151

144 p., ill., LIST OF FIGURES 4 -- LIST OF TABLES 5 -- LIST OF ABBREVIATIONS 6 -- GLOSSARY 10 --  SCIENTIFIC REPORT 11 -- 1 INTRODUCTION .11 -- 1.1 MOVING FROM A SUPPLY-DRIVEN TO A NEEDS-DRIVEN HEALTHCARE SYSTEM .11 -- 1.2 THE NEED APPROACH .13 -- 1.3 THE NEED APPROACH AS APPLICABLE TO RARE DISEASES 14 -- 1.3.1 Rare diseases and challenges in research on rare diseases 14 -- 1.3.2 Policies to incentivise research and development in rare diseases 15 -- 1.3.3 Remaining challenges related to diagnosis and care of patients with rare diseases 15 -- 2 RESEARCH QUESTIONS, AIM AND OBJECTIVES 17 -- 2.1 RESEARCH QUESTIONS .17 -- 2.2 AIM AND OBJECTIVES 17 -- 3 METHODOLOGICAL APPROACH 18 -- 3.1 GREY LITERATURE REVIEW .19 -- 3.1.1 Search strategy and screening .19 -- 3.1.2 Data extraction 19 -- 3.1.3 Data synthesis and analysis 19 -- 3.2 RAPID REVIEW 20 -- 3.2.1 Search strategy design .20 -- 3.2.2 Screening and selection 21 -- 3.2.3 Data extraction 21 -- 3.2.4 Data synthesis and analysis 21 -- 3.3 STAKEHOLDER CONSULTATIONS 21 -- 3.3.1 Sampling strategy of the stakeholder consultations 21 -- 3.3.2 Data retrieval 22 -- 3.3.3 Data analysis 22 -- 4 DEFINITIONS OF RARE DISEASES 24 -- 4.1 METHODS 24 -- 4.2 RESULTS 26 -- 4.3 DISCUSSION AND CONCLUSION 30 -- 4.3.1 Implications of the diversity of definitions among stakeholders 30 -- 4.3.2 Difficulties of the different elements of rare disease definitions 30 -- 4.3.3 Limitations 30 -- 4.3.4 Conclusion 31 -- 5 METHODOLOGICAL CHALLENGES .31 -- 5.1 METHODS 31 -- 5.1.1 Rapid Literature Review 31 -- 5.1.2 Stakeholder workshops .34 -- 5.2 RESULTS 36 -- 5.2.1 Methods to identify unmet needs in rare diseases .37 -- 5.2.2 Challenges related to rare disease research .43 -- 5.3 DISCUSSION AND CONCLUSION 56 -- 6 THE APPLICABILITY OF THE NEED ASSESSMENT FRAMEWORK IN RARE DISEASES .57 -- 6.1 METHODS 57 -- 6.2 RESULTS 57 -- 6.2.1 Patient needs .59 -- 6.2.2 Societal needs 90 -- 6.2.3 Future needs 106 -- 6.2.4 Transversal dimension: Equity 106 -- 6.3 DISCUSSION

Introduction Stroke is a time-critical condition and one of the leading causes of mortality and disability worldwide. To decrease mortality and improve patient outcome by improving access to optimal treatment, there is an emerging need to improve the accuracy of the methods used to identify and characterise stroke in prehospital settings and emergency departments (EDs). This might be accomplished by developing computerised decision support systems (CDSSs) that are based on artificial intelligence (AI) and potential new data sources such as vital signs, biomarkers and image and video analysis. This scoping review aims to summarise literature on existing methods for early characterisation of stroke by using AI. Methods and analysis The review will be performed with respect to the Arksey and O'Malley's model. Peer-reviewed articles about AI-based CDSSs for the characterisation of stroke or new potential data sources for stroke CDSSs, published between January 1995 and April 2023 and written in English, will be included. Studies reporting methods that depend on mobile CT scanning or with no focus on prehospital or ED care will be excluded. Screening will be done in two steps: title and abstract screening followed by full-text screening. Two reviewers will perform the screening process independently, and a third reviewer will be involved in case of disagreement. Final decision will be made based on majority vote. Results will be reported using a descriptive summary and thematic analysis. Ethics and dissemination The methodology used in the protocol is based on information publicly available and does not need ethical approval. The results from the review will be submitted for publication in a peer-reviewed journal. The findings will be shared at relevant national and international conferences and meetings in the field of digital health and neurology. © 2023 BMJ Publishing Group. All rights reserved.

Background: Qualitative longitudinal research (QLR) is an emerging methodology used in health research. The method literature states that the change in a phenomenon through time should be the focus of any QLR study, but in empirical studies, the analysis of changes through time is often poorly described, and the emphasis on time/change in the findings varies greatly. This inconsistency might depend on limitations in the existing method literature in terms of describing how QLR studies can present findings. The aim of this study was to develop and describe a typology of alternative approaches for integrating time and/or change in QLR findings. Methods: In this method study, we used an adapted scoping review design. Articles were identified using EBSCOhost. In total, methods and results sections from 299 QLR articles in the field of health research were analyzed with inspiration from content analysis. Results: We constructed a typology of three types and seven subtypes. The types were based on the underlying structural principles of how time/change was presented: Type A) Findings have a low utilization of longitudinal data, Type B) Findings are structured according to chronological time, and Type C) Findings focus on changes through time. These types differed in 1) the way the main focus was on time, change or neither; 2) the level of interpretation in the findings; and 3) how theoretical understandings of time/change were articulated in the articles. Each type encompassed two or three subtypes that represented distinct approaches to the aim and results presentation of QLR findings. Conclusions: This method study is the first to describe a coherent and comprehensive typology of alternative approaches for integrating time/change into QLR findings in health research. By providing examples of various subtypes that can be used for results presentations, it can help researchers make informed decisions suitable to their research intent.

Introduction Stroke is a time-critical condition and one of the leading causes of mortality and disability worldwide. To decrease mortality and improve patient outcome by improving access to optimal treatment, there is an emerging need to improve the accuracy of the methods used to identify and characterise stroke in prehospital settings and emergency departments (EDs). This might be accomplished by developing computerised decision support systems (CDSSs) that are based on artificial intelligence (AI) and potential new data sources such as vital signs, biomarkers and image and video analysis. This scoping review aims to summarise literature on existing methods for early characterisation of stroke by using AI. Methods and analysis The review will be performed with respect to the Arksey and O'Malley's model. Peer-reviewed articles about AI-based CDSSs for the characterisation of stroke or new potential data sources for stroke CDSSs, published between January 1995 and April 2023 and written in English, will be included. Studies reporting methods that depend on mobile CT scanning or with no focus on prehospital or ED care will be excluded. Screening will be done in two steps: title and abstract screening followed by full-text screening. Two reviewers will perform the screening process independently, and a third reviewer will be involved in case of disagreement. Final decision will be made based on majority vote. Results will be reported using a descriptive summary and thematic analysis. Ethics and dissemination The methodology used in the protocol is based on information publicly available and does not need ethical approval. The results from the review will be submitted for publication in a peer-reviewed journal. The findings will be shared at relevant national and international conferences and meetings in the field of digital health and neurology. © 2023 BMJ Publishing Group. All rights reserved.

Background: Qualitative longitudinal research (QLR) is an emerging methodology used in health research. The method literature states that the change in a phenomenon through time should be the focus of any QLR study, but in empirical studies, the analysis of changes through time is often poorly described, and the emphasis on time/change in the findings varies greatly. This inconsistency might depend on limitations in the existing method literature in terms of describing how QLR studies can present findings. The aim of this study was to develop and describe a typology of alternative approaches for integrating time and/or change in QLR findings. Methods: In this method study, we used an adapted scoping review design. Articles were identified using EBSCOhost. In total, methods and results sections from 299 QLR articles in the field of health research were analyzed with inspiration from content analysis. Results: We constructed a typology of three types and seven subtypes. The types were based on the underlying structural principles of how time/change was presented: Type A) Findings have a low utilization of longitudinal data, Type B) Findings are structured according to chronological time, and Type C) Findings focus on changes through time. These types differed in 1) the way the main focus was on time, change or neither; 2) the level of interpretation in the findings; and 3) how theoretical understandings of time/change were articulated in the articles. Each type encompassed two or three subtypes that represented distinct approaches to the aim and results presentation of QLR findings. Conclusions: This method study is the first to describe a coherent and comprehensive typology of alternative approaches for integrating time/change into QLR findings in health research. By providing examples of various subtypes that can be used for results presentations, it can help researchers make informed decisions suitable to their research intent.

Background: research training is essential in the teaching-learning process. Its purpose is to generate scientists who respond to the social needs of the 21st century.Objective: to characterize the research training of professionals in the medical sciences and related specialties incorporated into projects on the subject of diabetes and pregnancy in the research line "Studies in experimental bio-models of chronic non-transmissible diseases" and in a close relationship among teaching-university-society.Methods: a cross-sectional descriptive study of the research training process of undergraduate and postgraduate students linked to projects on diabetes and pregnancy was carried out, from the Biomedical Research Unit of Villa Clara University of Medical Sciences, from 2012 to 2022. Methods of the theoretical and empirical level were used. The information resulting from the documentary analysis was qualitatively processed and presented as a descriptive explanation.Results: through the incorporation of 16 undergraduate and graduate students to nine research projects, a working team was established with an adequate research pyramid. The scientific results generated two diploma papers, 10 medical specialty theses, two master's degrees, and two doctorates that were published in 14 peer-reviewed journals. Competences and values were developed during the investigative training process.Conclusions: the investigative training of the research team on diabetes and pregnancy was characterized by the integration of its members into projects, the obtaining and dissemination of scientific results, and the formation of skills and values that have allowed the consolidation of the teaching-research-society relationship., Fundamento: la formación investigativa es fundamental en el proceso enseñanza aprendizaje. Tiene como finalidad generar científicos que respondan a las necesidades sociales del siglo XXI.Objetivo: caracterizar la formación investigativa en profesionales de las ciencias médicas y especialidades afines incorporados a proyectos sobre la temática diabetes y embarazo en la línea de investigación “Estudios en biomodelos experimentales de enfermedades crónicas no transmisibles” y en estrecho vínculo docencia-universidad-sociedad.Métodos: se realizó un estudio descriptivo transversal del proceso de formación investigativa de los estudiantes de pregrado y posgrado vinculados a proyectos sobre diabetes y embarazo, desde la Unidad de Investigaciones Biomédicas de la Universidad de Ciencias Médicas de Villa Clara, en el periodo comprendido entre los años 2012 y 2022. Se emplearon métodos del nivel teórico y empírico. La información resultante del análisis documental se procesó cualitativamente y se presentó como explicación descriptiva.Resultados: a través de la incorporación de 16 estudiantes de pregrado y posgrado a nueve proyectos de investigación, se estableció un equipo de trabajo con una adecuada pirámide investigativa. Los resultados científicos generaron dos trabajos de diploma, 10 tesis de especialidades médicas, dos maestrías y dos doctorados que fueron difundidos en 14 revistas arbitradas. Se desarrollaron competencias y valores durante el proceso de formación investigativa.Conclusiones: la formación investigativa del equipo de investigación sobre diabetes y embarazo se caracterizó por la integración de sus miembros a proyectos, obtención y divulgación de resultados científicos, y formación de competencias y valores que han permitido consolidar la relación docencia-investigación-sociedad.

ObjectivesTo compare hospitals that did and did not participate in clinical trials evaluating potential inpatient COVID-19 therapeutics.MethodsWe conducted a cross-sectional study of hospitals participating in trials that were registered on clinicaltrials.gov between April and August 2020. Using the 2019 RAND Hospital Dataset and 2019 American Community Survey, we used logistic regression modeling to compare hospital-level traits including demographic features between trial and non-trial hospitals.ResultsWe included 488 hospitals that were participating in 298 interventional trials and 4232 non-participating hospitals. After controlling for demographic and other hospital traits, we found that teaching status (OR 2.11, 95% CI 1.52-2.95), higher patient acuity (OR 7.48, 4.39, 13.1), and location in the Northeast (OR 1.83, 95% CI 1.18, 2.85) and in wealthier counties (OR: 1.32, 95% CI 1.16-1.51) were associated with increased odds of trial participation, while being in counties with more White residents was associated with reduced odds (OR 0.98, 95% CI 0.98-0.99).ConclusionsHospitals participating and not participating in COVID-19 inpatient treatment clinical trials differed in many ways, resulting in important implications for the generalizability of trial data.

The widespread availability of high-dimensional biological data has made the simultaneous screening of many biological characteristics a central problem in computational and high-dimensional biology. As the dimensionality of datasets continues to grow, so too does the complexity of identifying biomarkers linked to exposure patterns. The statistical analysis of such data often relies upon parametric modeling assumptions motivated by convenience, inviting opportunities for model misspecification. While estimation frameworks incorporating flexible, data adaptive regression strategies can mitigate this, their standard variance estimators are often unstable in high-dimensional settings, resulting in inflated Type-I error even after standard multiple testing corrections. We adapt a shrinkage approach compatible with parametric modeling strategies to semiparametric variance estimators of a family of efficient, asymptotically linear estimators of causal effects, defined by counterfactual exposure contrasts. Augmenting the inferential stability of these estimators in high-dimensional settings yields a data adaptive approach for robustly uncovering stable causal associations, even when sample sizes are limited. Our generalized variance estimator is evaluated against appropriate alternatives in numerical experiments, and an open source R/Bioconductor package, biotmle, is introduced. The proposal is demonstrated in an analysis of high-dimensional DNA methylation data from an observational study on the epigenetic effects of tobacco smoking.

PurposeSymptom heterogeneity in interstitial cystitis/bladder pain syndrome and chronic prostatitis/chronic pelvic pain syndrome, collectively termed urological chronic pelvic pain syndrome, has resulted in difficulty in defining appropriate clinical trial endpoints. We determine clinically important differences for 2 primary symptom measures, pelvic pain severity and urinary symptom severity, and evaluate subgroup differences.Materials and methodsThe Multidisciplinary Approach to the Study of Chronic Pelvic Pain Symptom Patterns Study enrolled individuals with urological chronic pelvic pain syndrome. We defined clinically important differences by associating changes in pelvic pain severity and urinary symptom severity over 3 to 6 months with marked improvement on a global response assessment using regression and receiver operating characteristic curves. We evaluated clinically important differences for absolute and percent change and examined differences in clinically important differences by sex-diagnosis, presence of Hunner lesions, pain type, pain widespreadness, and baseline symptom severity.ResultsAn absolute change of -4 was clinically important in pelvic pain severity among all patients, but clinically important difference estimates differed by pain type, presence of Hunner lesions, and baseline severity. Pelvic pain severity clinically important difference estimates for percent change were more consistent across subgroups and ranged from 30% to 57%. The absolute change urinary symptom severity clinically important difference was -3 for female participants and -2 for male participants with chronic prostatitis/chronic pelvic pain syndrome only. Patients with greater baseline severity required larger decreases in symptoms to feel improved. Estimated clinically important differences had lower accuracy among participants with low baseline symptoms.ConclusionsA reduction of 30%-50% in pelvic pain severity is a clinically meaningful endpoint for future therapeutic

Psychological researchers often use standard linear regression to identify relevant predictors of an outcome of interest, but challenges emerge with incomplete data and growing numbers of candidate predictors. Regularization methods like the LASSO can reduce the risk of overfitting, increase model interpretability, and improve prediction in future samples; however, handling missing data when using regularization-based variable selection methods is complicated. Using listwise deletion or an ad hoc imputation strategy to deal with missing data when using regularization methods can lead to loss of precision, substantial bias, and a reduction in predictive ability. In this tutorial, we describe three approaches for fitting a LASSO when using multiple imputation to handle missing data and illustrate how to implement these approaches in practice with an applied example. We discuss implications of each approach and describe additional research that would help solidify recommendations for best practices. (PsycInfo Database Record (c) 2023 APA, all rights reserved).