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Respiratory Research Jul 2021Idiopathic pulmonary fibrosis (IPF) is a progressive debilitating lung disease with considerable morbidity. Heterogeneity in epidemiologic studies means the full impact... (Review)
Review
BACKGROUND
Idiopathic pulmonary fibrosis (IPF) is a progressive debilitating lung disease with considerable morbidity. Heterogeneity in epidemiologic studies means the full impact of the disease is unclear.
METHODS
A targeted literature search for population-based, observational studies reporting incidence and/or prevalence of IPF from January 2009 to April 2020 was conducted. Identified studies were aggregated by country. For countries with multiple publications, a weighted average was determined. Incidence and prevalence data were adjusted for between-study differences where possible. The final model included adjusted estimates of incidence and prevalence per 10,000 of the population with 95% confidence intervals. As prevalence estimates vary depending on the definitions used, estimates were based on a specific case definition of IPF.
RESULTS
Overall, 22 studies covering 12 countries met the inclusion criteria, with 15 reporting incidence and 18 reporting prevalence estimates. The adjusted incidence estimates (per 10,000 of the population) ranged from 0.35 to 1.30 in Asia-Pacific countries, 0.09 to 0.49 in Europe, and 0.75 to 0.93 in North America. Unadjusted and adjusted incidence estimates were consistent. The adjusted prevalence estimates ranged from 0.57 to 4.51 in Asia-Pacific countries, 0.33 to 2.51 in Europe, and 2.40 to 2.98 in North America. South Korea had the highest incidence and prevalence estimates. When prevalence estimates were compared to country-specific rare disease thresholds, IPF met the definition of a rare disease in all countries except South Korea. There were notable geographic gaps for IPF epidemiologic data.
CONCLUSIONS
Due to differences in study methodologies, there is worldwide variability in the reported incidence and prevalence of IPF. Based on the countries included in our analysis, we estimated the adjusted incidence and prevalence of IPF to be in the range of 0.09-1.30 and 0.33-4.51 per 10,000 persons, respectively. According to these prevalence estimates, IPF remains a rare disease. For consistency, future epidemiologic studies of IPF should take age, sex, smoking status, and the specificity of case definitions into consideration.
Topics: Global Health; Humans; Idiopathic Pulmonary Fibrosis; Incidence; Observational Studies as Topic; Population Surveillance; Prevalence
PubMed: 34233665
DOI: 10.1186/s12931-021-01791-z -
BMJ Quality & Safety Jul 2020Double checking medication administration in hospitals is often standard practice, particularly for high-risk drugs, yet its effectiveness in reducing medication... (Review)
Review
BACKGROUND
Double checking medication administration in hospitals is often standard practice, particularly for high-risk drugs, yet its effectiveness in reducing medication administration errors (MAEs) and improving patient outcomes remains unclear. We conducted a systematic review of studies evaluating evidence of the effectiveness of double checking to reduce MAEs.
METHODS
Five databases (PubMed, Embase, CINAHL, Ovid@Journals, OpenGrey) were searched for studies evaluating the use and effectiveness of double checking on reducing medication administration errors in a hospital setting. Included studies were required to report any of three outcome measures: an effect estimate such as a risk ratio or risk difference representing the association between double checking and MAEs, or between double checking and patient harm; or a rate representing adherence to the hospital's double checking policy.
RESULTS
Thirteen studies were identified, including 10 studies using an observational study design, two randomised controlled trials and one randomised trial in a simulated setting. Studies included both paediatric and adult inpatient populations and varied considerably in quality. Among three good quality studies, only one showed a significant association between double checking and a reduction in MAEs, another showed no association, and the third study reported only adherence rates. No studies investigated changes in medication-related harm associated with double checking. Reported double checking adherence rates ranged from 52% to 97% of administrations. Only three studies reported if and how independent and primed double checking were differentiated.
CONCLUSION
There is insufficient evidence that double versus single checking of medication administration is associated with lower rates of MAEs or reduced harm. Most comparative studies fail to define or investigate the level of adherence to independent double checking, further limiting conclusions regarding effectiveness in error prevention. Higher-quality studies are needed to determine if, and in what context (eg, drug type, setting), double checking produces sufficient benefits in patient safety to warrant the considerable resources required. CRD42018103436.
Topics: Databases, Factual; Humans; Medication Errors; Observational Studies as Topic; Pharmaceutical Preparations; Randomized Controlled Trials as Topic
PubMed: 31391315
DOI: 10.1136/bmjqs-2019-009552 -
Journal of the International Society of... 2022Tendinopathy is a painful condition that is prevalent in athletes as well as the general human population, and whose management is challenging. (Review)
Review
BACKGROUND
Tendinopathy is a painful condition that is prevalent in athletes as well as the general human population, and whose management is challenging.
OBJECTIVE
This systematic review aimed to evaluate the impact of nutrition on the prevention and treatment of tendinopathy.
METHODS
Searches were conducted in PubMed, EMBASE, Web of Science, and SPORTDiscus without restriction to year of publication. Studies examining the impact of exposure to nutrient intake in an adult human population on 1) prevalence/incidence of tendinopathy, 2) clinical outcomes of tendinopathy, 3) structural changes in the tendon by imaging modalities. Experimental and observational study designs written in English, Dutch, or German were eligible.
RESULTS
Nineteen studies met the inclusion criteria. The effects of the habitual diet were investigated in one study. Four studies examined the effects of exposure to alcohol. Alcohol consumption can be a potential risk factor associated with Achilles tendinopathy and rotator cuff tears, although findings were inconsistent. The use of dietary supplements was examined in fourteen studies. Among these, collagen-derived peptides were most often part of the supplements evaluated. Combining training and dietary supplements seems to induce better clinical and functional outcomes in tendinopathy.
CONCLUSION
This review demonstrates the paucity of high-quality studies and a wide variety among studies regarding nutrients, tendon location, study population, and reported outcome measures. Individual studies showed promising clinical implications for the use of dietary supplements, particularly those containing collagen-derived peptides. However, giving any definitive dietary recommendations on the prevention and treatment of tendinopathy remains elusive.
Topics: Achilles Tendon; Adult; Diet; Dietary Supplements; Humans; Nutritional Status; Observational Studies as Topic; Tendinopathy
PubMed: 35937777
DOI: 10.1080/15502783.2022.2104130 -
Nature Medicine Nov 2021The particularly interdisciplinary nature of human microbiome research makes the organization and reporting of results spanning epidemiology, biology, bioinformatics,... (Review)
Review
The particularly interdisciplinary nature of human microbiome research makes the organization and reporting of results spanning epidemiology, biology, bioinformatics, translational medicine and statistics a challenge. Commonly used reporting guidelines for observational or genetic epidemiology studies lack key features specific to microbiome studies. Therefore, a multidisciplinary group of microbiome epidemiology researchers adapted guidelines for observational and genetic studies to culture-independent human microbiome studies, and also developed new reporting elements for laboratory, bioinformatics and statistical analyses tailored to microbiome studies. The resulting tool, called 'Strengthening The Organization and Reporting of Microbiome Studies' (STORMS), is composed of a 17-item checklist organized into six sections that correspond to the typical sections of a scientific publication, presented as an editable table for inclusion in supplementary materials. The STORMS checklist provides guidance for concise and complete reporting of microbiome studies that will facilitate manuscript preparation, peer review, and reader comprehension of publications and comparative analysis of published results.
Topics: Computational Biology; Dysbiosis; Humans; Microbiota; Observational Studies as Topic; Research Design; Translational Science, Biomedical
PubMed: 34789871
DOI: 10.1038/s41591-021-01552-x -
Journal of Traditional Chinese Medicine... Aug 2020To analyze clinical studies on correlations between Traditional Chinese Medicine (TCM) body constitution types and diseases published in the past 10 years, and to... (Review)
Review
OBJECTIVE
To analyze clinical studies on correlations between Traditional Chinese Medicine (TCM) body constitution types and diseases published in the past 10 years, and to provide an evidence base to support the use of such correlations for health maintenance and disease prevention.
METHODS
We searched five databases for the period April 2009 to December 2019: China National Knowledge Infrastructure Database, Wanfang Database, China Science and Technology Journal Database, PubMed and Embase. Three types of observational studies on correlation between constitution types and diseases were included: cross-sectional, case-control and cohort studies. Descriptive statistical methods were employed for data analysis.
RESULTS
A total of 1639 clinical studies were identified: 1452 (88.59%) cross-sectional studies, 115 (7.02%) case-control studies and 72 (4.39%) cohort studies covering 30 regions of China and five other countries (Malaysia, South Korea, Singapore, Thailand and France). The collection of studies comprised 19 disease categories and 333 different diseases. The 10 most commonly studied diseases were hypertension, diabetes, stroke, coronary atherosclerotic heart disease (CAHD), sleep disorders, neoplasm of the breast, dysmenorrhea, fatty liver disease, chronic viral hepatitis B and dyslipidemia. We found high distributions for each biased constitution type in different patient populations as follows: Qi-deficiency constitution in stroke, diabetes, chronic obstructive pulmonary disease, acquired immunodeficiency syndrome and hypertension; Yang-deficiency constitution in female infertility, osteoporosis, irritable bowel syndrome, gonarthrosis and dysmenorrhea; Yin-deficiency constitution in hypertension, diabetes, constipation, female climacteric states and osteoporosis; phlegm- dampness constitution in hypertension, stroke, fatty liver disease, diabetes and metabolic syndrome; damp-heat constitution in acne, chronic gastritis, chronic viral hepatitis B, human papillomavirus infection and hyperuricemia; blood-stasis constitution in CAHD, endometriosis and stroke; Qi-stagnation constitution in hyperplasia and neoplasms of the breast, insomnia, depression and thyroid nodules; and inherited-special constitution in asthma and allergic rhinitis.
CONCLUSION
Eight biased TCM constitutions were closely related to specific diseases, and could be used to guide individualized prevention and treatment. More rigorously designed studies are recommended to further verify the constitution-disease relationship.
Topics: Drug Therapy; Drugs, Chinese Herbal; Humans; Medicine, Chinese Traditional; Observational Studies as Topic; Treatment Outcome
PubMed: 32744037
DOI: 10.19852/j.cnki.jtcm.2020.04.019 -
Advances in Nutrition (Bethesda, Md.) Oct 2021Numerous observational studies have investigated the role of the Dietary Inflammatory Index (DII®) in chronic disease risk. The aims of this umbrella review and... (Review)
Review
Numerous observational studies have investigated the role of the Dietary Inflammatory Index (DII®) in chronic disease risk. The aims of this umbrella review and integrated meta-analyses were to systematically synthesize the observational evidence reporting on the associations between the DII and health outcomes based on meta-analyses, and to assess the quality and strength of the evidence for each associated outcome. This umbrella review with integrated meta-analyses investigated the association between the DII and a range of health outcomes based on meta-analyses of observational data. A credibility assessment was conducted for each outcome using the following criteria: statistical heterogeneity, 95% prediction intervals, evidence for small-study effect and/or excess significance bias, as well as effect sizes and P values using calculated random effects meta-analyses. In total, 15 meta-analyses reporting on 38 chronic disease-related outcomes were included, incorporating a total population of 4,360,111 subjects. Outcomes (n = 38) were examined through various study designs including case-control (n = 8), cross-sectional (n = 5), prospective (n = 5), and combination (n = 20) study designs. Adherence to a pro-inflammatory dietary pattern had a significant positive association with 27 (71%) of the included health outcomes (P value < 0.05). Using the credibility assessment, Class I (Convincing) evidence was identified for myocardial infarction only, Class II (Highly suggestive) evidence was identified for increased risk of all-cause mortality, overall risk of incident cancer, and risk of incident site-specific cancers (colorectal, pancreatic, respiratory, and oral cancers) with increasing (more pro-inflammatory) DII score. Most outcomes (n = 31) presented Class III (Suggestive) or lower evidence (Weak or No association). Pro-inflammatory dietary patterns were nominally associated with an increased risk of many chronic disease outcomes. However, the strength of evidence for most outcomes was limited. Further prospective studies are required to improve the precision of the effect size.
Topics: Diet; Humans; Meta-Analysis as Topic; Neoplasms; Observational Studies as Topic
PubMed: 33873204
DOI: 10.1093/advances/nmab037 -
JAMA Apr 2023Nonrandomized studies using insurance claims databases can be analyzed to produce real-world evidence on the effectiveness of medical products. Given the lack of...
IMPORTANCE
Nonrandomized studies using insurance claims databases can be analyzed to produce real-world evidence on the effectiveness of medical products. Given the lack of baseline randomization and measurement issues, concerns exist about whether such studies produce unbiased treatment effect estimates.
OBJECTIVE
To emulate the design of 30 completed and 2 ongoing randomized clinical trials (RCTs) of medications with database studies using observational analogues of the RCT design parameters (population, intervention, comparator, outcome, time [PICOT]) and to quantify agreement in RCT-database study pairs.
DESIGN, SETTING, AND PARTICIPANTS
New-user cohort studies with propensity score matching using 3 US claims databases (Optum Clinformatics, MarketScan, and Medicare). Inclusion-exclusion criteria for each database study were prespecified to emulate the corresponding RCT. RCTs were explicitly selected based on feasibility, including power, key confounders, and end points more likely to be emulated with real-world data. All 32 protocols were registered on ClinicalTrials.gov before conducting analyses. Emulations were conducted from 2017 through 2022.
EXPOSURES
Therapies for multiple clinical conditions were included.
MAIN OUTCOMES AND MEASURES
Database study emulations focused on the primary outcome of the corresponding RCT. Findings of database studies were compared with RCTs using predefined metrics, including Pearson correlation coefficients and binary metrics based on statistical significance agreement, estimate agreement, and standardized difference.
RESULTS
In these highly selected RCTs, the overall observed agreement between the RCT and the database emulation results was a Pearson correlation of 0.82 (95% CI, 0.64-0.91), with 75% meeting statistical significance, 66% estimate agreement, and 75% standardized difference agreement. In a post hoc analysis limited to 16 RCTs with closer emulation of trial design and measurements, concordance was higher (Pearson r, 0.93; 95% CI, 0.79-0.97; 94% meeting statistical significance, 88% estimate agreement, 88% standardized difference agreement). Weaker concordance occurred among 16 RCTs for which close emulation of certain design elements that define the research question (PICOT) with data from insurance claims was not possible (Pearson r, 0.53; 95% CI, 0.00-0.83; 56% meeting statistical significance, 50% estimate agreement, 69% standardized difference agreement).
CONCLUSIONS AND RELEVANCE
Real-world evidence studies can reach similar conclusions as RCTs when design and measurements can be closely emulated, but this may be difficult to achieve. Concordance in results varied depending on the agreement metric. Emulation differences, chance, and residual confounding can contribute to divergence in results and are difficult to disentangle.
Topics: Humans; Randomized Controlled Trials as Topic; Research Design; Observational Studies as Topic
PubMed: 37097356
DOI: 10.1001/jama.2023.4221 -
BMJ (Clinical Research Ed.) Oct 2021Mendelian randomisation (MR) studies allow a better understanding of the causal effects of modifiable exposures on health outcomes, but the published evidence is often...
Mendelian randomisation (MR) studies allow a better understanding of the causal effects of modifiable exposures on health outcomes, but the published evidence is often hampered by inadequate reporting. Reporting guidelines help authors effectively communicate all critical information about what was done and what was found. STROBE-MR (strengthening the reporting of observational studies in epidemiology using mendelian randomisation) assists authors in reporting their MR research clearly and transparently. Adopting STROBE-MR should help readers, reviewers, and journal editors evaluate the quality of published MR studies. This article explains the 20 items of the STROBE-MR checklist, along with their meaning and rationale, using terms defined in a glossary. Examples of transparent reporting are used for each item to illustrate best practices.
Topics: Epidemiologic Research Design; Guidelines as Topic; Humans; Mendelian Randomization Analysis; Observational Studies as Topic
PubMed: 34702754
DOI: 10.1136/bmj.n2233 -
Atencion Primaria May 2022To evaluate the experiencie with a health education program in Primary Care in patients with chronic shoulder pain of musculoskeletal origin, on pain and disability and... (Observational Study)
Observational Study
OBJECTIVE
To evaluate the experiencie with a health education program in Primary Care in patients with chronic shoulder pain of musculoskeletal origin, on pain and disability and establish the protocol in primary care.
DESIGN
Quasi-experimental longitudinal descriptive observational study.
LOCATION
Arroyo de la Vega Health Center, Alcobendas, Madrid.
PARTICIPANTS
Patients referred by their Primary Care Physician to the Primary Care Physiotherapy Unit for shoulder pain of musculoskeletal origin.
INTERVENTION
7 group sessions of health education and therapeutic exercise.
MAIN MEASUREMENTS
Pain intensity was assessed through the Visual Analogue Scale (VAS), the disability of the upper limb with the Disabilities of the Arm, Shoulder and Hand (DASH) questionnaire and the level of disability and shoulder pain with the Shoulder Pain and Disability Index (SPADI) questionnaire.
RESULTS
Statistically significant differences were found in the reduction of pain and disability (P<.01), in addition, drug use and recurrences were reduced.
CONCLUSIONS
The shoulder physiotherapy protocol with health education was effective in reducing pain and disability in patients with chronic shoulder pain of musculoskeletal origin in Primary Care.
Topics: Chronic Pain; Health Education; Humans; Musculoskeletal Pain; Observational Studies as Topic; Physical Therapy Modalities; Primary Health Care; Shoulder; Shoulder Pain; Upper Extremity
PubMed: 35461039
DOI: 10.1016/j.aprim.2022.102284 -
Pharmacoepidemiology and Drug Safety Oct 2020There is a need to develop hybrid trial methodology combining the best parts of traditional randomized controlled trials (RCTs) and observational study designs to... (Review)
Review
PURPOSE
There is a need to develop hybrid trial methodology combining the best parts of traditional randomized controlled trials (RCTs) and observational study designs to produce real-world evidence (RWE) that provides adequate scientific evidence for regulatory decision-making.
METHODS
This review explores how hybrid study designs that include features of RCTs and studies with real-world data (RWD) can combine the advantages of both to generate RWE that is fit for regulatory purposes.
RESULTS
Some hybrid designs include randomization and use pragmatic outcomes; other designs use single-arm trial data supplemented with external comparators derived from RWD or leverage novel data collection approaches to capture long-term outcomes in a real-world setting. Some of these approaches have already been successfully used in regulatory decisions, raising the possibility that studies using RWD could increasingly be used to augment or replace traditional RCTs for the demonstration of drug effectiveness in certain contexts. These changes come against a background of long reliance on RCTs for regulatory decision-making, which are labor-intensive, costly, and produce data that can have limited applicability in real-world clinical practice.
CONCLUSIONS
While RWE from observational studies is well accepted for satisfying postapproval safety monitoring requirements, it has not commonly been used to demonstrate drug effectiveness for regulatory purposes. However, this position is changing as regulatory opinions, guidance frameworks, and RWD methodologies are evolving, with growing recognition of the value of using RWE that is acceptable for regulatory decision-making.
Topics: Decision Making; Drug Approval; Humans; Observational Studies as Topic; Pragmatic Clinical Trials as Topic; Randomized Controlled Trials as Topic; Research Design
PubMed: 31823482
DOI: 10.1002/pds.4932