Journal: BMJ Open

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BMJ

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2044-6055

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2015 - 201982020 - 202523
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Publications 1 - 10 of 31
  • Reference values and validation of the 1-minute sit-to-stand test in healthy 5-16-year-old youth: A cross-sectional study
    Item type: Journal Article
    Haile, Sarah R.; Fühner, Thea; Granacher, Urs; et al. (2021)
    BMJ Open
    Objectives It is essential to have simple, reliable and valid tests to measure children’s functional capacity in schools or medical practice. The 1-minute sit-to-stand (STS) test is a quick fitness test requiring little equipment or space that is increasingly used in both healthy populations and those with chronic disease. We aimed to provide age-specific and sex-specific reference values of STS test in healthy children and adolescents and to evaluate its short-term reliability and construct validity. Design setting and participants Cross-sectional convenience sample from six public schools and one science fair in central Europe. Overall, 587 healthy participants aged 5–16 years were recruited and divided into age groups of 3 years each. Outcomes 1-minute STS. To evaluate short-term reliability, some children performed the STS test twice. To evaluate construct validity, some children also performed a standing long jump (SLJ) and a maximal incremental exercise test. Results Data from 547 youth aged 5–16 years were finally included in the analyses. The median number of repetitions in 1 min in males (females) ranged from 55 [95% CI: 38 to 72] (53 [95% CI: 35 to 76]) in 14–16-year olds to 59 [95% CI: 41 to 77] (60 [95% CI: 38 to 77]) in 8–10-year olds. Children who repeated STS showed a learning effect of on average 4.8 repetitions more than the first test (95% limits of agreement: −6.7 to 16.4). Moderate correlations were observed between the STS and the SLJ (r=0.48) tests and the maximal exercise test (r=0.43). Conclusions The reported STS reference values can be used to interpret STS test performance in children and adolescents. The STS appears to have good test–retest reliability, but a learning effect of about 10%. The association of STS with other measures of physical fitness should be further explored in a larger study and technical standards for its conduct are needed.
  • Modelling years of life lost due to acute type A aortic dissection in the German healthcare setting: a predictive study
    Item type: Journal Article
    Schiele, Philipp; König, Adriana N.; Meyer, Alexander; et al. (2024)
    BMJ Open
    OBJECTIVES: This study aimed to develop a patient-centred approach to the burden of acute type A aortic dissection (ATAAD) through modelling. The main objective was to identify potential improvements in managing this life-threatening cardiovascular condition and to provide evidence-based recommendations to optimise outcomes. DESIGN: We developed a predictive model along patient pathways to estimate the burden of ATAAD through the years of life lost (YLLs) metric. The model was created based on a systematic review of the literature and was parameterised using demographic data from the German healthcare environment. The model was designed to allow interactive simulation of different scenarios resulting from changes in key impact factors. SETTING: The study was conducted using data from the German healthcare environment and results from the literature review. PARTICIPANTS: The study included a comprehensive modelling of ATAAD cases in Germany but did not directly involve participants. INTERVENTIONS: There were no specific interventions applied in this study based on the modelling design. PRIMARY AND SECONDARY OUTCOME MEASURES: The single outcome measure was the estimation of YLL due to ATAAD in Germany. RESULTS: Our model estimated 102 791 YLL per year for ATAAD in Germany, with 62 432 and 40 359 YLL for men and women, respectively. Modelling an improved care setting yielded 93 191 YLL or 9.3% less YLL compared with the current standard while a worst-case scenario resulted in 113 023 or 10.0% more YLL. The model is accessible at https://acuteaorticdissection.com/ to estimate custom scenarios. CONCLUSIONS: Our study provides an evidence-based approach to estimating the burden of ATAAD and identifying potential improvements in the management of pathways. This approach can be used by healthcare decision-makers to inform policy changes aimed at optimising patient outcomes. By considering patient-centred approaches in any healthcare environment, the model has the potential to improve efficient care for patients suffering from ATAAD.
  • Minimal disease activity and remission in patients with psoriatic arthritis with elevated body mass index: an observational cohort study in the Swiss Clinical Quality Management cohort
    Item type: Journal Article
    Vallejo-Yagüe, Enriqueta; Burkard, Theresa; Micheroli, Raphael; et al. (2022)
    BMJ Open
    Objective: To assess the impact of elevated body mass index (BMI) in the achievement of minimal disease activity (MDA) and several definitions of remission in patients with psoriatic arthritis (PsA) in Switzerland. Secondarily, to assess the overlapping across the study outcomes. Methods: This observational cohort study in the Swiss Clinical Quality Management in Rheumatic Diseases (SCQM) registry included patients with PsA starting their first biologic or targeted synthetic disease-modifying antirheumatic drug (b/tsDMARD) from 1997 to 30 June 2018. Exposure was BMI category at b/tsDMARD start: overweight, obese, and normal weight (reference). Logistic regression was used to assess the achievement of MDA and remission at ≤12 months, as well as treatment persistence at 1 year, in overweight patients and patients with obesity compared with the normal weight group. Remission was defined by Disease Activity for Psoriatic Arthritis (DAPSA), clinical DAPSA (cDAPSA) and 28-joint Disease Activity Score (DAS28). Additionally, overlapping across study outcomes was investigated. Results: The study included 306 (39.5%) normal weight patients, 285 (36.8%) overweight patients and 183 (23.6%) patients with obesity. Compared with the normal weight group, patients with obesity had lower odds of achieving MDA at ≤ 12 months (adjusted OR (ORadj) 0.45, 95% CI 0.24 to 0.82). This was consistent with the observed reduced odds of achieving DAPSA-remission (ORadj 0.42, 95% CI 0.21 to 0.85), cDAPSA-remission (ORadj 0.51, 95% CI 0.27 to 0.96) and DAS28-remission (ORadj 0.51, 95% CI 0.32 to 0.81) in patients with obesity versus normal weight patients. Among the 125 patients achieving MDA, the majority (81.8% normal weight, 80.0% overweight, 78.9% obese) achieved cDAPSA-remission. No differences were observed in the odds to achieving treatment persistence between the BMI strata. Conclusions: Obesity halved the likelihood of achieving MDA and remission in patients with PsA with b/tsDMARDs compared with those with normal weight, while it did not impact treatment persistence. High overlapping of patients achieving the outcomes MDA and cDAPSA-remission was observed across every BMI group.
  • Comparison of carbon dioxide control during pressure controlled versus pressure-regulated volume controlled ventilation in children (CoCO2): protocol for a pilot digital randomised controlled trial in a quaternary paediatric intensive care unit
    Item type: Journal Article
    Mozun, Rebeca; Chopard, Daphné; Zapf, Florian; et al. (2025)
    BMJ Open
    Introduction Digital trials are a promising strategy to increase the evidence base for common interventions and may convey considerable efficiency benefits in trial conduct. Although paediatric intensive care units (PICUs) are rich in routine electronic data, highly pragmatic digital trials in this field remain scarce. There are unmet evidence needs for optimal mechanical ventilation modes in paediatric intensive care. We aim to test the feasibility of a digital PICU trial comparing two modes of invasive mechanical ventilation using carbon dioxide (CO2) control as the outcome measure. Methods and analysis Single-centre, open-labelled, randomised controlled pilot trial with two parallel treatment arms comparing pressure control versus pressure-regulated volume control. Patients are eligible if aged <18 years, weighing >2 kg, have an arterial line and require >60 min of mechanical ventilation during PICU hospitalisation at the University Children’s Hospital Zurich. Exclusion criteria include cardiac shunt lesions, pulmonary hypertension under treatment and intracranial hypertension. CO2 is measured using three methods: end-tidal (continuous), transcutaneous (continuous) and blood gas analyses (intermittent). Baseline, intervention and outcome data are collected electronically from the patients’ routine electronic health records. The primary feasibility outcome is adherence to the assigned ventilation mode, while the primary physiological outcome is the proportion of time spent within the target range of CO2 (end-tidal, normocarbia defined as CO2 ≥ 4.5 and ≤ 6 kPa). Both primary outcomes are captured digitally every minute from randomisation until censoring (at 48 hours after randomisation, extubation, discharge or death, whichever comes first). Analysis will occur on an intention-to-treat basis. We aim to enrol 60 patients in total. Recruitment started in January 2024 and continued for 9 months. Ethics and dissemination This study received ethical approval from the Cantonal Ethics Commission of Zurich (identification number: 2022–00829). Study results will be disseminated through publication in a peer-reviewed journal and other media like podcasts. Trial registration number NCT05843123.
  • Transmission of ESBL-producing Enterobacteriaceae and their mobile genetic elements-identification of sources by whole genome sequencing: study protocol for an observational study in Switzerland
    Item type: Journal Article
    Stadler, Tanja; Meinel, Dominik; Aguilar-Bultet, Lisandra; et al. (2018)
    BMJ Open
    Introduction Extended-spectrum beta-lactamases (ESBL)-producing Enterobacteriaceae were first described in relation with hospital-acquired infections. In the 2000s, the epidemiology of ESBL-producing organisms changed as especially ESBL-producing Escherichia coli was increasingly described as an important cause of community-acquired infections, supporting the hypothesis that in more recent years ESBL-producing Enterobacteriaceae have probably been imported into hospitals rather than vice versa. Transmission of ESBL-producing Enterobacteriaceae is complicated by ESBL genes being encoded on self-transmissible plasmids, which can be exchanged among the same and different bacterial species. The aim of this research project is to quantify hospital-wide transmission of ESBL-producing Enterobacteriaceae on both the level of bacterial species and the mobile genetic elements and to determine if hospital-acquired infections caused by ESBL producers are related to strains and mobile genetic elements predominantly circulating in the community or in the healthcare setting. This distinction is critical in prevention since the former emphasises the urgent need to establish or reinforce antibiotic stewardship programmes, and the latter would call for more rigorous infection control. Methods and analysis This protocol presents an observational study that will be performed at the University Hospital Basel and in the city of Basel, Switzerland. ESBL-producing Enterobacteriaceae will be collected from any specimens obtained by routine clinical practice or by active screening in both inpatient and outpatient settings, as well as from wastewater samples and foodstuffs, both collected monthly over a 12-month period for analyses by whole genome sequencing. Bacterial chromosomal, plasmid and ESBL-gene sequences will be compared within the cohort to determine genetic relatedness and migration between humans and their environment. Ethics and dissemination This study has been approved by the local ethics committee (Ethikkommission Nordwest-und Zentralschweiz) as a quality control project (Project-ID 2017–00100). The results of this study will be published in peer-reviewed medical journals, communicated to participants, the general public and all relevant stakeholders.
  • Systematic review of the effects of iodised salt and iodine supplements on prenatal and postnatal growth: study protocol
    Item type: Journal Article
    Farebrother, Jessica; Naude, Celeste E.; Nicol, Liesl; et al. (2015)
    BMJ Open
    Introduction Iodine is an essential micronutrient and component of the thyroid hormones. Sufficient ingestion of iodine is necessary for normal growth and development. If iodine requirements are not met, growth can be impaired. Salt iodisation and supplementation with iodine can prevent iodine deficiency disorders and stunted growth. No systematic review has yet collated the evidence linking iodine to growth. With an increased emphasis on stunting within the WHO Global Nutrition Targets for 2025, we propose a systematic review to address this question. Methods and analysis We will undertake a systematic review, and if appropriate, meta-analyses, evaluating the effects of iodised salt or iodine supplements on prenatal and postnatal somatic growth, until age 18. We will search a number of databases, including MEDLINE, EMBASE, Web of Science, CINAHL, PsychINFO, the Cochrane Library, including the CENTRAL register of Controlled Trials and also the WHO library and ICTRP (International Clinical Trials Registry Platform), which includes the Clinicaltrials.gov repository. We will also search Wanfang Data and the China Knowledge Resource Integrated Database. Included studies must have compared exposure to iodised salt, iodine supplements or iodised oil, to placebo, non-iodised salt or no intervention. Primary outcomes will be continuous and categorical markers of prenatal and postnatal somatic growth. Secondary outcomes will cover further measures of growth, including growth rates and indirect markers of growth such as insulin-like growth factor-1 (IGF-1). Ethics and dissemination The systematic review will be published in a peer-reviewed journal, and will be sent directly to the WHO, United Nations Children's Fund, International Council for the Control of Iodine Deficiency Disorders and other stakeholders. The results generated from this systematic review will provide evidence to support future programme recommendations regarding iodine fortification or supplementation and child growth.
  • Digital health technologies for metabolic disorders in older adults: a scoping review protocol
    Item type: Review Article
    Huynh, Panitda; Fleisch, Elgar; Brändle, Michael; et al. (2024)
    BMJ Open
    Introduction Metabolic disorders (type 2 diabetes, insulin resistance, hyperglycaemia, obesity, hyperlipidaemia, hypertension, non-alcoholic fatty liver disease and metabolic syndrome) are leading causes of mortality and disability worldwide. These disorders disproportionately affect older adults relative to those younger. Digital health technologies (DHTs), such as patient monitoring, digital diagnostics and digital therapeutics, emerge as promising tools for health promotion in day-to-day life. However, their role in targeting metabolic disorders, particularly among a key demographic of older adults, is not yet fully understood. Thus, this study aims to scope the use of DHTs in managing metabolic health disorders among older adults. To our knowledge, this is the first review to categorise DHTs for older adults with metabolic disorders, contributing to the growing literature in the intersection of metabolic disorders and DHTs. Methods and analysis We will conduct a scoping review following the recommended framework by Arksey and O’Malley. Our search will focus on three primary concepts: metabolic disorders, DHTs and older adults. We plan to search five online databases (Cochrane, ScienceDirect, PubMed, Scopus and Web of Science) to identify original research articles published between January 2014 and January 2024. Two independent reviewers will screen articles based on predefined eligibility criteria, with a third reviewer resolving conflicts. Data will be extracted using a standardised form, and results will be synthesised qualitatively and quantitatively. The database searches, data collection and analysis are planned and have not been conducted. Ethics and dissemination No ethics approval is required for this protocol and scoping review. Data will be used only from published studies with appropriate ethics approval. Results will be disseminated in a peer-reviewed publication.
  • Application of orphan drug designation to cancer treatments (2008–2017): a comprehensive and comparative analysis of the USA and EU
    Item type: Journal Article
    Vokinger, Kerstin Noëlle; Kesselheim, Aaron S. (2019)
    BMJ Open
    Objective To determine differences in the characteristics of cancer drugs designated as orphan drugs by the Food and Drug Administration (FDA) and European Medicines Agency (EMA). Design and setting Identification of all cancer drugs (initial or supplementary indication) with orphan status approved by the FDA between 2008–2017 based on publicly accessible reports. The European public assessment reports (EPAR) was searched to determine whether these FDA-approved drugs were also approved by the EMA. Main outcome measures Extraction of active ingredient, trade name, approval date and approved indication from two FDA data sources (Orphan Drug Product Designation Database, Drugs@FDA) and comparison with the same data from EPAR. Results The FDA approved 135 cancer drugs with orphan indications that met our inclusion criteria, of which 101 (75%) were also approved by the EMA. 80/101 (79%) were first approved in the USA. Only 41/101 (41%) also received orphan designation by the EMA. 33/101 (33%) were approved for biomarker-based indications in the USA, however, only nine approved cancer drug indications by the EMA were biomarker-derived drugs. 78% (47/60) of approved cancer drugs that were only approved in the USA with orphan status were indicated for solid tumours, 22% (13/60) had indications for non-solid tumours. By contrast, out of those approved cancer drugs that received orphan designation by both agencies, 20% (8/41) were indicated for solid, and 80% (33/41) for non-solid tumours. Conclusions Orphan designation was intended to encourage drug development for rare conditions. This study shows that the FDA approves more cancer drugs with such designations compared with the EMA, especially for subgroups of more prevalent cancers. One reason for the difference could be that the European Union requires demonstration of significant benefit for drugs that target the same indication as a drug already on the market to earn the orphan designation.
  • NapBiome trial: Targeting gut microbiota to improve sleep rhythm and developmental and behavioural outcomes in early childhood in a birth cohort in Switzerland - a study protocol
    Item type: Journal Article
    Zimmermann, Petra; Kurth, Salome; Giannoukos, Stamatios; et al. (2025)
    BMJ Open
    Introduction The gut–brain axis plays a crucial role in the regulation and development of psychological and physical processes. The first year of life is a critical period for the development of the gut microbiome, which parallels important milestones in establishing sleep rhythm and brain development. Growing evidence suggests that the gut microbiome influences sleep, cognition and early neurodevelopment. For term-born and preterm-born infants, difficulties in sleep regulation may have consequences on health. Identifying effective interventions on the gut–brain axis in early life is likely to have long-term implications for the health and development of at-risk infants. Methods and analyses In this multicentre, four-group, double-blinded, placebo (PLC)-controlled randomised trial with a factorial design, 120 preterm-born and 260 term-born infants will be included. The study will investigate whether the administration of daily synbiotics or PLC for a duration of 3 months improves sleep patterns and neurodevelopmental outcomes up to 2 years of age. The trial will also: (1) determine the association between gut microbiota, sleep patterns and health outcomes in children up to 2 years of age; and (2) leverage the interactions between gut microbiota, brain and sleep to develop new intervention strategies for at-risk infants. Ethics and dissemination The NapBiome trial has received ethical approval by the Committee of Northwestern and Central Switzerland and Canton Vaud, Switzerland (#2024–01681). Outcomes will be disseminated through publication and will be presented at scientific conferences. Metagenomic data will be shared through the European Nucleotide Archive. Trial registration number The US National Institutes of Health NCT06396689.
Publications 1 - 10 of 31