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BMJ Open Jun 2021Stroke is the leading neurological cause of adult long-term disability in Europe. Even though functional consequences directly related to neurological impairment are...
INTRODUCTION
Stroke is the leading neurological cause of adult long-term disability in Europe. Even though functional consequences directly related to neurological impairment are well studied, post-stroke trajectories of functional health according to the International Classification of Functioning, Disability and Health are poorly understood. Particularly, no study investigated the relationship between post-stroke trajectories of activities of daily living (ADL) and self-rated health (SRH). However, such knowledge is of major importance to identify patients at risk of unfavourable courses. This prospective observational study aims to investigate trajectories of ADL and SRH, and their modifying factors in the course of the first year after stroke.
METHODS AND ANALYSIS
The study will consecutively enrol 300 patients admitted to a tertiary care hospital with acute ischaemic stroke or transient ischaemic attack (TIA; Age, Blood Pressure, Clinical Features, Duration of symptoms, Diabetes score ≥3). Patient inclusion is planned from May 2021 to September 2022. All participants will complete an interview assessing ADL, SRH, mental health, views on ageing and resilience-related concepts. Participants will be interviewed face-to-face 1-5 days post-stroke/TIA in the hospital; and will be followed up after 6 weeks, 3 months, 6 months and 12 months via telephone. The 12-month follow-up will also include a neurological assessment. Primary endpoints are ADL operationalised by modified Rankin Scale scores and SRH. Secondary outcomes are further measures of ADL, functional health, physical activity, falls and fatigue. Views on ageing, social support, resilience-related concepts, affect, frailty, illness perceptions and loneliness will be examined as modifying factors. Analyses will investigate the bidirectional relationship between SRH and ADL using bivariate latent change score models.
ETHICS AND DISSEMINATION
The study has been approved by the institutional review board of the University Medicine Greifswald (Ref. BB 237/20). The results will be disseminated through scientific publications, conferences and media. Moreover, study results and potential implications will be discussed with patient representatives.
TRIAL REGISTRATION NUMBER
NCT04704635.
Topics: Activities of Daily Living; Adult; Brain Ischemia; Europe; Humans; Ischemic Attack, Transient; Observational Studies as Topic; Stroke
PubMed: 34187831
DOI: 10.1136/bmjopen-2021-049944 -
Assessment Aug 2016Observational measurement plays an integral role in a variety of scientific endeavors within biology, psychology, sociology, education, medicine, and marketing. The...
Observational measurement plays an integral role in a variety of scientific endeavors within biology, psychology, sociology, education, medicine, and marketing. The current article provides an interdisciplinary primer on observational measurement; in particular, it highlights recent advances in observational methodology and the challenges that accompany such growth. First, we detail the various types of instrument that can be used to standardize measurements across observers. Second, we argue for the importance of validity in observational measurement and provide several approaches to validation based on contemporary validity theory. Third, we outline the challenges currently faced by observational researchers pertaining to measurement drift, observer reactivity, reliability analysis, and time/expense. Fourth, we describe recent advances in computer-assisted measurement, fully automated measurement, and statistical data analysis. Finally, we identify several key directions for future observational research to explore.
Topics: Automation; Computers; Forecasting; Humans; Observational Studies as Topic; Observer Variation; Reproducibility of Results; Statistics as Topic
PubMed: 26933139
DOI: 10.1177/1073191116635807 -
Genes May 2023Congenital disorders of glycosylation (CDGs) are a wide group of genetic diseases characterised by a severe clinical spectrum, consisting of developmental delays,... (Review)
Review
Congenital disorders of glycosylation (CDGs) are a wide group of genetic diseases characterised by a severe clinical spectrum, consisting of developmental delays, dysmorphisms, and neurological deficits. Mutations in the gene lead to a disorder called hyperphosphatasia with impaired intellectual development syndrome 1 (HPMRS1), distinct from other CDGs in terms of hyperphosphatemia related to abnormal ALP activity and brachytelephalangy. This article discusses the phenotype of six Polish patients with HPMRS1 with a special focus on behavioural and imaging features, which were not addressed in 26 previously reported cases. The medical records of six patients aged 6 to 22 years were collected and analysed. In all cases, the same homozygotic mutation (c.1022C>A; p.Ala341Glu) was found, although the patients presented a diverse spectrum of neurological and developmental disorders, concerning in most cases the muscular tonus and general developmental delay. The most prevalent dysmorphic features included hypertelorism, high palate, and finger anomalies, whereas other characteristics present in all previously described cases, such as a short, broad nose and brachytelephalangy, were less frequently observed. Similarly to previous reports, the magnetic resonance (MR) and computed tomography (CT) head scans returned varied results, including physiological and pathological brain images in equal measure, the latter of which consisted of cortical atrophy, delayed myelination, hydrocephalus, and hypoplastic corpus callosum. Each patient exhibited symptoms characteristic of autism spectrum disorders, especially in terms of attention deficits, as well as controlling and expressing emotions. The most common type of sensory processing disorder was over-responsivity. Despite the low prevalence of HPMRS1, the patients reported in the literature presented a rather uniform phenotype, which does not correspond with the one found in each individual of the studied group. Behavioural disorders and sensory impairment require additional care and awareness considering the global developmental delay often observed in these patients.
Topics: Humans; Poland; Abnormalities, Multiple; Phenotype; Neuroimaging; Observational Studies as Topic
PubMed: 37372388
DOI: 10.3390/genes14061208 -
BMJ Open Apr 2022Due to a global shortage of healthcare workers, there is a lack of basic healthcare for 4 billion people worldwide, particularly affecting low-income and middle-income...
Study protocol for a pilot prospective, observational study investigating the condition suggestion and urgency advice accuracy of a symptom assessment app in sub-Saharan Africa: the AFYA-'Health' Study.
INTRODUCTION
Due to a global shortage of healthcare workers, there is a lack of basic healthcare for 4 billion people worldwide, particularly affecting low-income and middle-income countries. The utilisation of AI-based healthcare tools such as symptom assessment applications (SAAs) has the potential to reduce the burden on healthcare systems. The purpose of the AFYA Study (AI-based Assessment oF health sYmptoms in TAnzania) is to evaluate the accuracy of the condition suggestions and urgency advice provided by a user on a Swahili language Ada SAA.
METHODS AND ANALYSIS
This study is designed as an observational prospective clinical study. The setting is a waiting room of a Tanzanian district hospital. It will include patients entering the outpatient clinic with various conditions and age groups, including children and adolescents. Patients will be asked to use the SAA before proceeding to usual care. After usual care, they will have a consultation with a study-provided physician. Patients and healthcare practitioners will be blinded to the SAA's results. An expert panel will compare the Ada SAA's condition suggestions and urgency advice to usual care and study provided differential diagnoses and triage. The primary outcome measures are the accuracy and comprehensiveness of the Ada SAA evaluated against the gold standard differential diagnoses.
ETHICS AND DISSEMINATION
Ethical approval was received by the ethics committee (EC) of Muhimbili University of Health and Allied Sciences with an approval number MUHAS-REC-09-2019-044 and the National Institute for Medical Research, NIMR/HQ/R.8c/Vol. I/922. All amendments to the protocol are reported and adapted on the basis of the requirements of the EC. The results from this study will be submitted to peer-reviewed journals, local and international stakeholders, and will be communicated in editorials/articles by Ada Health.
TRIAL REGISTRATION NUMBER
NCT04958577.
Topics: Adolescent; Child; Humans; Mobile Applications; Observational Studies as Topic; Pilot Projects; Prospective Studies; Symptom Assessment; Tanzania
PubMed: 35410928
DOI: 10.1136/bmjopen-2021-055915 -
Advances in Nutrition (Bethesda, Md.) Oct 2022Gastroparesis (Gp) is a delay in gastric emptying in the absence of a mechanical obstruction and has the capacity to cause symptoms that significantly impact a patient's...
Gastroparesis (Gp) is a delay in gastric emptying in the absence of a mechanical obstruction and has the capacity to cause symptoms that significantly impact a patient's quality of life. Dietary interventions are the first-line treatment in Gp, but the efficacy of different diets is unclear. This systematic review seeks to determine the effectiveness of dietary interventions on clinical outcomes in Gp. A literature search of MEDLINE Ovid from 1 March 2008 to 1 October 2021 was conducted to identify randomized controlled trials, cohort studies, and cross-sectional studies that reported dietary interventions in Gp. From the initial search, 2789 studies resulted. These were assessed by 2 independent reviewers and selected based on the primary outcomes of interest: changes in symptom-specific patient-reported outcomes and changes in gastric emptying time. A third reviewer resolved any discrepancies. Six adult studies (185 subjects) met the inclusion criteria, whereas no pediatric study did. Five of the included studies were randomized controlled trials and one was an observational study. The systematic review suggested low-fat diets, small-particle diets, diets with isoflavones, and foods considered bland, starchy, sweet, and salty did not exacerbate Gp symptoms. Small-particle diets and diets with isoflavones were found to improve gastric emptying time in patients. Additionally, small-particle diets were shown to reduce anxiety in comparison to large-particle diets. Of the randomized controlled trials, 80% were low risk of bias and 20% were fair risk of bias. The observational study was considered fair quality. The data presented in this review suggest specific dietary interventions could potentially improve Gp symptoms and gastric emptying in adult patients, particularly low-fat and small-particle diets. For pediatric Gp, data are lacking. The limited data available highlights a critical gap in the literature.
Topics: Adult; Cross-Sectional Studies; Diet; Gastroparesis; Humans; Isoflavones; Observational Studies as Topic; Quality of Life
PubMed: 35425953
DOI: 10.1093/advances/nmac037 -
Journal of Internal Medicine Jun 2014Self-controlled observational study designs, such as the case-crossover design and the self-controlled case series, are reviewed, and their respective rationale,... (Review)
Review
Self-controlled observational study designs, such as the case-crossover design and the self-controlled case series, are reviewed, and their respective rationale, strengths and limitations are compared. Although no single design is generally superior to the others, they share the trait of being robust towards confounders that are stable over time. The self-controlled designs can be particularly useful when using secondary healthcare data for pharmacoepidemiological research and might be useful in screening for adverse drug effects. The main limitations of self-controlled designs are that they are amenable only to transient effects; some may be inefficient with long-term exposure; and they may be sensitive towards trends in exposure.
Topics: Bias; Case-Control Studies; Causality; Confounding Factors, Epidemiologic; Epidemiologic Research Design; Humans; Observational Studies as Topic; Pharmacoepidemiology; Statistics as Topic
PubMed: 24635348
DOI: 10.1111/joim.12186 -
Veterinary Pathology Sep 2018Observational studies are the basis for much of our knowledge of veterinary pathology and are highly relevant to the daily practice of pathology. However,...
Observational studies are the basis for much of our knowledge of veterinary pathology and are highly relevant to the daily practice of pathology. However, recommendations for conducting pathology-based observational studies are not readily available. In part 1 of this series, we offer advice on planning and conducting an observational study with examples from the veterinary pathology literature. Investigators should recognize the importance of creativity, insight, and innovation in devising studies that solve problems and fill important gaps in knowledge. Studies should focus on specific and testable hypotheses, questions, or objectives. The methodology is developed to support these goals. We consider the merits and limitations of different types of analytic and descriptive studies, as well as of prospective vs retrospective enrollment. Investigators should define clear inclusion and exclusion criteria and select adequate numbers of study subjects, including careful selection of the most appropriate controls. Studies of causality must consider the temporal relationships between variables and the advantages of measuring incident cases rather than prevalent cases. Investigators must consider unique aspects of studies based on archived laboratory case material and take particular care to consider and mitigate the potential for selection bias and information bias. We close by discussing approaches to adding value and impact to observational studies. Part 2 of the series focuses on methodology and validation of methods.
Topics: Animals; Observational Studies as Topic; Pathology, Veterinary; Research Design
PubMed: 30071806
DOI: 10.1177/0300985818785705 -
BMC Medical Research Methodology Dec 2019All clinical research benefits from transparency and validity. Transparency and validity of studies may increase by prospective registration of protocols and by...
BACKGROUND
All clinical research benefits from transparency and validity. Transparency and validity of studies may increase by prospective registration of protocols and by publication of statistical analysis plans (SAPs) before data have been accessed to discern data-driven analyses from pre-planned analyses.
MAIN MESSAGE
Like clinical trials, recommendations for SAPs for observational studies increase the transparency and validity of findings. We appraised the applicability of recently developed guidelines for the content of SAPs for clinical trials to SAPs for observational studies. Of the 32 items recommended for a SAP for a clinical trial, 30 items (94%) were identically applicable to a SAP for our observational study. Power estimations and adjustments for multiplicity are equally important in observational studies and clinical trials as both types of studies usually address multiple hypotheses. Only two clinical trial items (6%) regarding issues of randomisation and definition of adherence to the intervention did not seem applicable to observational studies. We suggest to include one new item specifically applicable to observational studies to be addressed in a SAP, describing how adjustment for possible confounders will be handled in the analyses.
CONCLUSION
With only few amendments, the guidelines for SAP of a clinical trial can be applied to a SAP for an observational study. We suggest SAPs should be equally required for observational studies and clinical trials to increase their transparency and validity.
Topics: Data Interpretation, Statistical; Humans; Observational Studies as Topic; Reproducibility of Results; Research Design
PubMed: 31818263
DOI: 10.1186/s12874-019-0879-5 -
American Journal of Epidemiology May 2019Much of the intellectual tradition of modern epidemiology stems from efforts to understand and combat chronic diseases persisting through the 20th century epidemiologic... (Review)
Review
Much of the intellectual tradition of modern epidemiology stems from efforts to understand and combat chronic diseases persisting through the 20th century epidemiologic transition of countries such as the United States and United Kingdom. After decades of relative obscurity, infectious disease epidemiology has undergone an intellectual rebirth in recent years amid increasing recognition of the threat posed by both new and familiar pathogens. Here, we review the emerging coalescence of infectious disease epidemiology around a core set of study designs and statistical methods bearing little resemblance to the chronic disease epidemiology toolkit. We offer our outlook on challenges and opportunities facing the field, including the integration of novel molecular and digital information sources into disease surveillance, the assimilation of such data into models of pathogen spread, and the increasing contribution of models to public health practice. We next consider emerging paradigms in causal inference for infectious diseases, ranging from approaches to evaluating vaccines and antimicrobial therapies to the task of ascribing clinical syndromes to etiologic microorganisms, an age-old problem transformed by our increasing ability to characterize human-associated microbiota. These areas represent an increasingly important component of epidemiology training programs for future generations of researchers and practitioners.
Topics: Anti-Infective Agents; Causality; Communicable Disease Control; Communicable Diseases; Data Interpretation, Statistical; Disaster Planning; Epidemiologic Methods; Humans; Observational Studies as Topic; Population Surveillance; Public Health Practice; United Kingdom; United States; Vaccines
PubMed: 30877295
DOI: 10.1093/aje/kwy264 -
BMJ Open Apr 2023Patients who continue to smoke cigarettes after a cancer diagnosis can experience poorer treatment tolerance and outcomes than those who quit immediately. Identifying...
INTRODUCTION
Patients who continue to smoke cigarettes after a cancer diagnosis can experience poorer treatment tolerance and outcomes than those who quit immediately. Identifying risk factors specific to patients with cancer who smoke, as well as their smoking behaviours (eg, frequency of use, types of tobacco products), dependency level and quit intentions, is necessary to better inform patients and encourage quitting smoking after a cancer diagnosis. This study aims to examine the occurrence of smoking in patients with cancer treated at specialised oncology departments and outpatient clinics based within the metropolitan region of Hamburg, Germany, and presents an analysis of their smoking patterns. This understanding is the first step in developing an adequate smoking cessation intervention and shall contribute to a sustainable improvement in the treatment results, long-term survival and quality of life of patients with cancer.
METHODS AND ANALYSIS
A questionnaire will be administered to patients with cancer (N=865) aged 18 years and above in the catchment area of Hamburg, Germany. Data acquisition includes sociodemographic, medical and psychosocial data as well as information on current smoking patterns. To identify the associations between smoking patterns and sociodemographic characteristics, disease-related variables, and psychological risk factors, descriptive statistics and multiple logistic as well as multinomial regressions will be performed.
ETHICS AND DISSEMINATION
This study was registered at Open Science Framework (https://doi.org/10.17605/OSF.IO/PGBY8). It was approved by the ethics committee of the local psychological Ethic committee at the centre of psychosocial medicine Hamburg, Germany (LPEK) (tracking number: LPEK-0212). The study will be carried out in accordance with the Code of Ethics of the Declaration of Helsinki. The results will be published in peer-reviewed scientific journals.
Topics: Humans; Intention; Smoking Cessation; Quality of Life; Neoplasms; Smoking; Observational Studies as Topic
PubMed: 37055212
DOI: 10.1136/bmjopen-2022-069570