BMJ Open最新文献

{"title":"CADOR (Capsaicin in neuropathic-like pain in digital osteoarthritis) study protocol: a multicentre randomised parallel-group trial.","authors":"Angelique Fan, Céline Lambert, Jeremie Sellam, Roland Chapurlat, Hubert Marotte, Bruno Pereira, Thierry Thomas, Anne Tournadre, Martin Soubrier, Sylvain Mathieu","doi":"10.1136/bmjopen-2024-093409","DOIUrl":"https://doi.org/10.1136/bmjopen-2024-093409","url":null,"abstract":"<p><strong>Introduction: </strong>It is common for finger pain in hand osteoarthritis (HOA) to display a neuropathic component. Non-steroidal anti-inflammatory drugs (NSAIDs) and conventional analgesics are not very effective in relieving this neuropathic-like pain. Capsaicin, a compound extracted from chilli peppers, is approved for the management of localised neuropathic pain. However, the effectiveness of an 8% capsaicin transdermal patch has never been tested in HOA patients with neuropathic-like pain in a randomised setting. In this study, we aimed to compare the 60-day (D60) efficacy of a transdermal application of capsaicin 8% versus a control (very low-dose capsaicin at 0.04%) on hand pain in patients with painful digital osteoarthritis with a neuropathic-like pain component.</p><p><strong>Methods and analysis: </strong>CADOR (CApsaicin in neuropathic-like pain in Digital Osteoarthritis: a Randomised trial) is a multicentre, randomised, controlled, double-blind, two parallel group, phase 3 clinical trial. Eligible patients have HOA according to the American College of Rheumatology criteria and at least Kellgren-Lawrence grade≥2, with neuropathic-like pain ('Douleur neuropathique en 4 questions' (DN4) score≥4/10 and pain intensity≥40/100 mm). At Day 0, 120 patients will be randomised (1:1) to receive a single 30 min topical application of either capsaicin 8% transdermal patch (experimental group) or capsaicin 0.04% transdermal patch (control group). The primary outcome is pain intensity in the fingers over the past 48 hours at Day 60, measured with a Visual Analogue Scale (VAS) ranging from 0 to 100 mm. Secondary outcomes are functional disability, quality of life, anxiety and depression, the patient's global impression of improvement, use of analgesics and NSAIDs and the safety of capsaicin. At visit D60, patients who still have finger pain≥40/100 mm on the VAS will receive, if they wish a single 30 min topical application of capsaicin 8% transdermal patch (not blinded). The efficacy of two transdermal applications of capsaicin (either two applications of 8% patch in the experimental arm or one application of 0.04% and one application of 8% in the control arm) will be assessed on Day 120.</p><p><strong>Ethics and dissemination: </strong>Ethics approval was obtained from the French Health Authorities (Comité de protection des Personnes SUD EST V and Agence Nationale de Sécurité du Médicament) on 23 May 2024 in accordance with European Regulation n°536/2014 of 16 April 2014 (ref: 2024-511159-16-00) before participant enrolment.</p><p><strong>Trial registration number: </strong>ClinicalTrials.gov: NCT06444919.</p>","PeriodicalId":9158,"journal":{"name":"BMJ Open","volume":"15 3","pages":"e093409"},"PeriodicalIF":2.4,"publicationDate":"2025-03-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143572070","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Detecting and responding to deterioration of a baby during labour: surveys of maternity professionals to inform co-design and implementation of a new standardised approach.","authors":"Jan W van der Scheer, Margaret Blott, Mary Dixon-Woods, Annabelle Olsson, Jordan Moxey, Sarah Kelly, Matthew Woodward, Giulia Maistrello, Wendy Randall, Sarah Blackwell, Chloe Hughes, Caroline Walker, Louise Dewick, Rachna Bahl, Tim J Draycott, Jenni Burt","doi":"10.1136/bmjopen-2024-084578","DOIUrl":"https://doi.org/10.1136/bmjopen-2024-084578","url":null,"abstract":"<p><strong>Objectives: </strong>Detecting and responding to deterioration of a baby during labour is likely to benefit from a standardised approach supported by principles of track-and-trigger systems. To inform co-design of a standardised approach and associated implementation strategies, we sought the views of UK-based maternity professionals.</p><p><strong>Design: </strong>Two successive cross-sectional surveys were hosted on an online collaboration platform (Thiscovery) between July 2021 and April 2022.</p><p><strong>Setting: </strong>UK.</p><p><strong>Participants: </strong>Across both surveys, 765 UK-based maternity professionals.</p><p><strong>Primary and secondary outcome measures: </strong>Count and percentage of participants selecting closed-ended response options, and categorisation and counting of free-text responses.</p><p><strong>Results: </strong>More than 90% of participants supported the principle of a standardised approach that systematically considers a range of intrapartum risk factors alongside fetal heart rate features. Over 80% of participants agreed on the importance of a proposed set of evidence-based risk factors underpinning such an approach, but many (over 75%) also indicated a need to clarify the clinical definitions of the proposed risk factors. A need for clarity was also suggested by participants' widely varying views on thresholds for actions of the proposed risk factors, particularly for meconium-stained liquor, rise in baseline fetal heart rate and changes in fetal heart rate variability. Most participants (>75%) considered a range of resources to support good practice as very useful when implementing the approach, such as when and how to escalate in different situations (82%), how to create a supportive culture (79%) and effective communication and decision-making with those in labour and their partners (75%).</p><p><strong>Conclusions: </strong>We found strong professional support for the principle of a standardised approach to detection and response to intrapartum fetal deterioration and high agreement on the clinical importance of a set of evidence-based risk factors. Further work is needed to address: (1) clarity of clinical definitions of some risk factors, (2) building evidence and agreement on thresholds for action and (3) deimplementation strategies for existing local practices.</p>","PeriodicalId":9158,"journal":{"name":"BMJ Open","volume":"15 3","pages":"e084578"},"PeriodicalIF":2.4,"publicationDate":"2025-03-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143572082","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Screening to identify people with type 2 diabetes at risk of liver cancer in primary care: a randomised controlled trial protocol.","authors":"Ryan M Buchanan, Tina Reinson, Josh Bilson, Hazel Woodland, Chinonso Nwoguh, Keith Cooper, Scott Harris, Karen Malone, Christopher D Byrne","doi":"10.1136/bmjopen-2024-088043","DOIUrl":"https://doi.org/10.1136/bmjopen-2024-088043","url":null,"abstract":"<p><strong>Introduction: </strong>Hepatocellular carcinoma (HCC) is expected to become the third most common cause of cancer death worldwide by 2030. The increase in HCC is in large part due to the rising prevalence of risk factors such as type 2 diabetes mellitus (T2DM). Up to 1 in 20 people living with T2DM have liver cirrhosis, and they have a 1% to 2% incidence of HCC per year. Patients with cirrhosis enter surveillance for HCC to identify early-stage, curable tumours. A diagnosis of T2DM does not mandate testing to identify patients with cirrhosis, with testing restricted to those with additional risks. There has never been a trial and nested cost-effectiveness evaluation comparing screening all patients with T2DM for cirrhosis against usual care.</p><p><strong>Methods and analysis: </strong>The study will use a multi-centre, unblinded individual randomised controlled trial design. The aim will be to determine the effectiveness and cost-effectiveness of screening all adults with T2DM to identify those at high risk of HCC. The recruitment strategy has been supported by patient and public involvement (PPI). Participants will be identified via an automated search of primary care records and invited to participate via text. 320 participants will be randomised for screening. The screening will include measurement of bio-markers for liver fibrosis (ELF and Fib-4) and vibration-controlled transient elastography. Another 320 participants will be randomised to standard care. Demographic and medical history data will be collected at baseline from all participants. Outcome data will be collected remotely from healthcare records. The primary outcome is the proportion of participants in each arm who are referred to HCC surveillance following testing for liver disease within 12 months of randomisation. The results will be used to calculate the incremental cost-effectiveness ratio of screening via a Markov model.</p><p><strong>Ethics and dissemination: </strong>The results of this study will be presented directly to National Health Service England. Additional dissemination via conference proceedings and publication will be supported by our PPI team. Ethical approval was granted by the West of Scotland Research Ethics Service on 2 August 2023, REC reference 23/WS/0102.</p><p><strong>Trial registration number: </strong>ISRCTN17017677.</p>","PeriodicalId":9158,"journal":{"name":"BMJ Open","volume":"15 3","pages":"e088043"},"PeriodicalIF":2.4,"publicationDate":"2025-03-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143572131","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Addressing the challenges and constraints of social protection policies for Peruvian women domestic workers: the ANITA project study protocol.","authors":"Janeth Tenorio-Mucha, Sebastián García, Jill Portocarrero, Maria Lazo-Porras, Karina Romero Rivero, David Vera Tudela, María Kathia Cárdenas, Silvana Pérez-León, Nathaly Aya Pastrana, Maria Sofia Cuba-Fuentes, Ayu Pinky Hapsari, Christopher Meaney, Andrew David Pinto, Viviana Cruzado, Archna Gupta","doi":"10.1136/bmjopen-2024-088921","DOIUrl":"https://doi.org/10.1136/bmjopen-2024-088921","url":null,"abstract":"<p><strong>Introduction: </strong>Domestic workers (DWs) are vulnerable to precarious or informal working conditions with limited access to social protection policies such as employer-paid health insurance or retirement pensions. This study aims to examine the working conditions, health status and access to healthcare for women DWs in Peru and propose recommendations to improve their access to social protection policies.</p><p><strong>Methods and analysis: </strong>The project uses a participatory action research approach by engaging three committees: a DW co-researcher committee, an advisory committee and a steering committee. The first two include former or current DWs, while the third includes policymakers and academics. We use a sequential mixed-methods design organised in four phases: (1) secondary data analysis (n=4216): using two Peruvian national surveys to characterise working conditions, health status and access to healthcare; (2) face-to-face survey (n=448): with DWs in three cities, using respondent-driven sampling to further characterise working and health conditions and to identify factors that influence knowledge of and access to social protection policies; (3) qualitative interviews (n=30-46): with DWs, leaders of DW organisations, employers and policymakers to gather different perspectives on the facilitators and barriers to access to social protection policies; and (4) deliberative dialogues (n=14-26): with DW, leaders of DW organisations, employers, policymakers and academics to identify key barriers to the implementation of social protection policies and to develop recommendations for overcoming these barriers.</p><p><strong>Ethics and dissemination: </strong>Phase 1 and Phase 2 received ethical clearance from Universidad Peruana Cayetano Heredia in Peru and Unity Health Toronto in Canada. Phase 3 and Phase 4 received ethical clearance from PRISMA Charitable Association in Peru and Unity Health Toronto in Canada. To mobilise knowledge, in collaboration with the committees, we will co-generate policy briefs and audiovisual materials to disseminate the results from this project to different audiences and sectors.</p>","PeriodicalId":9158,"journal":{"name":"BMJ Open","volume":"15 3","pages":"e088921"},"PeriodicalIF":2.4,"publicationDate":"2025-03-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143572032","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Assessing the impact of COmorbidities and Sociodemographic factors on Multiorgan Injury following COVID-19: rationale and protocol design of COSMIC, a UK multicentre observational study of COVID-negative controls.","authors":"Simran Shergill, Mohamed Elshibly, Sandeep S Hothi, Kelly S Parke, Rachel J England, Joanne V Wormleighton, George J Hudson, Elizabeth M Tunnicliffe, James Wild, Stephen M Smith, Sue Francis, Mark Toshner, Naveed Sattar, Kamlesh Khunti, Christopher E Brightling, Charalambos Antoniades, Colin Berry, John P Greenwood, Alastair Moss, Stefan Neubauer, Gerry P McCann, Betty Raman, Jayanth Ranjit Arnold","doi":"10.1136/bmjopen-2024-089508","DOIUrl":"https://doi.org/10.1136/bmjopen-2024-089508","url":null,"abstract":"<p><strong>Introduction: </strong>SARS-CoV-2 disease (COVID-19) has had an enormous health and economic impact globally. Although primarily a respiratory illness, multi-organ involvement is common in COVID-19, with evidence of vascular-mediated damage in the heart, liver, kidneys and brain in a substantial proportion of patients following moderate-to-severe infection. The pathophysiology and long-term clinical implications of multi-organ injury remain to be fully elucidated. Age, gender, ethnicity, frailty and deprivation are key determinants of infection severity, and both morbidity and mortality appear higher in patients with underlying comorbidities such as ischaemic heart disease, hypertension and diabetes. Our aim is to gain mechanistic insights into the pathophysiology of multiorgan dysfunction in people with COVID-19 and maximise the impact of national COVID-19 studies with a comparison group of COVID-negative controls.</p><p><strong>Methods and analysis: </strong>COmorbidities and Sociodemographic factors on Multiorgan Injury following COVID-19 (COSMIC) is a prospective, multicentre UK study which will recruit 200 subjects without clinical evidence of prior COVID-19 and perform extensive phenotyping with multiorgan imaging, biobank serum storage, functional assessment and patient reported outcome measures, providing a robust control population to facilitate current work and serve as an invaluable bioresource for future observational studies.</p><p><strong>Ethics and dissemination: </strong>Approved by the National Research Ethics Service Committee East Midlands (REC reference 19/EM/0295). Results will be disseminated via peer-reviewed journals and scientific meetings.</p><p><strong>Trial registration number: </strong>COSMIC is registered as an extension of C-MORE (Capturing Multi-ORgan Effects of COVID-19) on ClinicalTrials.gov (NCT04510025).</p>","PeriodicalId":9158,"journal":{"name":"BMJ Open","volume":"15 3","pages":"e089508"},"PeriodicalIF":2.4,"publicationDate":"2025-03-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143572054","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Perceptions of occupational physicians in supporting transgender and gender-diverse people (returning) at work - a focus group study: <i>The uncharted territory of gender-diverse occupational healthcare</i>.","authors":"Joy Van de Cauter, Joz Motmans, Dominique Van de Velde, Lisa Bernaers, Lutgart Braeckman","doi":"10.1136/bmjopen-2023-083604","DOIUrl":"https://doi.org/10.1136/bmjopen-2023-083604","url":null,"abstract":"<p><strong>Objectives: </strong>This study aimed to explore the role of occupational physicians (OPs) in supporting transgender and gender-diverse (TGD) workers during gender transition and return to work (RTW) following gender-affirming (medical) interventions.</p><p><strong>Design: </strong>We conducted a qualitative study (ONZ-2023-0026) using focus groups.</p><p><strong>Setting: </strong>This study involved OPs in Belgium.</p><p><strong>Participants: </strong>Two semistructured focus group interviews were held with 19 OPs working in occupational health services in Belgium in May and November 2023. Purposeful sampling was used, which included OPs with at least 2 years of seniority and experience with TGD people or inclusive company culture. Participants were predominantly white and cisgender, with varying levels of seniority and sectoral coverage.</p><p><strong>Methods: </strong>Qualitative data was thematically analysed using Braun and Clarke to find patterned meaning.</p><p><strong>Results: </strong>The analysis created four themes: (1) 'What is the right professional attitude?: You never get a second chance to make a good first impression' discusses the aspects of professional attitude alongside ethical considerations; (2) 'Controlled open-mindedness' entails values and views of gender in light of medicine, the individual worker, organisations and society; (3) 'The balance game: \"Fingerspitzengefühl\" (intuitive flair or instinct) without treating' and (4) 'Being gender-bombarded: the need for OP-tailored training and best practices'. These themes highlighted the limited knowledge and experience of OPs regarding gender-affirming care (GAC) and their need for additional training. Participants struggled to find the best gender-inclusive professional approach to support TGD workers as well as employers and require 'best practices'. Implementing overarching legislative frameworks can help OPs and companies create an inclusive work environment considering the differences in occupational sectors and company culture. RTW policies should focus on abilities without medicalisation and stigmatisation and involve multiple stakeholders.</p><p><strong>Conclusions: </strong>Occupational medicine can be crucial in improving the health and well-being of TGD workers. However, with the ageing workforce, gender diversity poses new 'hidden' challenges for sick leave management, RTW and sensitive health surveillance. Multidisciplinary training with stakeholders and GAC professionals can enhance occupational practice and equip future OPs with the necessary competencies and confidence.</p>","PeriodicalId":9158,"journal":{"name":"BMJ Open","volume":"15 3","pages":"e083604"},"PeriodicalIF":2.4,"publicationDate":"2025-03-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143572129","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Prevalence and risk factors of persistent cough in patients diagnosed with renal cell carcinoma: a systematic review and meta-analysis.","authors":"Wendy Smith, Joseph Santiapillai, Marilena Loizidou, Stuart Mazzone, Maxine G B Tran, Hirak K Patra, Muhammad Imran Omar, Faiz Mumtaz","doi":"10.1136/bmjopen-2024-088963","DOIUrl":"https://doi.org/10.1136/bmjopen-2024-088963","url":null,"abstract":"<p><strong>Objectives: </strong>Cough occurring in patients with renal cell carcinoma (RCC) was first described in 1935 and is a frequently discussed symptom on patient forums. We aimed to systematically review the available evidence to explore the prevalence and risk factors for persistent cough in patients diagnosed with RCC to establish whether cough could be a presenting symptom of RCC.</p><p><strong>Design: </strong>This epidemiological systematic review used the Preferred Reporting Items for Systematic Reviews and Meta-Analyses statement 2020.</p><p><strong>Data sources: </strong>Medline, Embase, Science Citation Index, The Cochrane Library, ClinicalTrials.gov and the WHO trials register were searched without language restrictions until 1 June 2023.</p><p><strong>Eligibility criteria for selecting studies: </strong>We included articles of all study designs reporting cough in patients (18 years or older) with RCC attributed to the disease itself or to treatment.</p><p><strong>Data extraction and synthesis: </strong>Data from included articles was extracted using a preprepared and piloted form, and quality assessment was conducted independently by two authors. The risk of bias was assessed in studies other than case reports or case series using the critical appraisal instrument for studies reporting prevalence data. Narrative techniques were used for data analysis and, where appropriate, meta-analysis using a fixed-effects model was performed.</p><p><strong>Results: </strong>Of 509 studies screened, 105 full-text articles were assessed, with 46 papers subsequently excluded, resulting in 59 analysed in depth. There were 105 patients with RCC reported as having a cough due to the disease itself within 30 case reports and 8 case series. When present, most coughs were described as persistent and dry in nature. The cause of cough was attributed to various aetiologies including pulmonary and endobronchial metastasis and paraneoplastic syndromes. Studies reporting patients with RCC developing a cough because of systemic treatment were heterogeneous. Two studies with 238 patients on temsirolimus and 230 on interferon-α (IFN-α) were suitable for meta-analysis using a fixed-effects model. Patients on temsirolimus were more likely to develop a cough than those on IFN-α (OR 1.95 with a 95% CI of 1.05 to 3.63, overall effect Z=2.12 (p=0.03), I²=0%).</p><p><strong>Conclusion: </strong>Cough can occur in patients with RCC, as part of the disease pathogenesis, as an adverse effect of systemic treatment or due to unrelated causes such as pre-existing conditions (eg, asthma). Further research is required to determine the true prevalence and cause and to assess whether cough could be a presenting symptom for RCC.</p><p><strong>Prospero registration number: </strong>CRD42022302962.</p>","PeriodicalId":9158,"journal":{"name":"BMJ Open","volume":"15 3","pages":"e088963"},"PeriodicalIF":2.4,"publicationDate":"2025-03-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143572130","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"What are the symptom trajectories of self-regulatory fatigue among family caregivers of stroke survivors? A protocol of mixed-methods study in Chinese rehabilitation settings.","authors":"Chao-Yue Xu, Ping Zou, Xi Chen, Shu-Lin Li, Jia-Chun You, Zhi-Qing He, Yan-Jin Huang","doi":"10.1136/bmjopen-2024-092724","DOIUrl":"https://doi.org/10.1136/bmjopen-2024-092724","url":null,"abstract":"<p><strong>Introduction: </strong>Stroke presents a considerable burden not only to patients but also to their families and society at large. In many instances, stroke patients opt for home rehabilitation, relying on family caregivers for daily assistance. This dynamic significantly influences the physiological, psychological and social well-being of these caregivers. Despite its importance, the phenomenon of self-regulatory fatigue (SRF) among family caregivers has received insufficient attention in the literature. Therefore, the objective of this study is to investigate the levels of SRF, the characteristics of associated symptoms and the trajectories of symptom change experienced by family caregivers of stroke patients.</p><p><strong>Methods and analysis: </strong>This research employs a mixed-methods approach, combining a cross-sectional study with a prospective longitudinal quantitative and qualitative design. The Chinese version of the SRF Scale and the Chinese version of Patient-Reported Outcomes Measurement Information System profile-29 are used to assess SRF, psychological and physiological symptoms, and related functional outcomes among family caregivers of stroke patients. Latent class growth analysis will be employed to model the heterogeneous developmental trajectories of SRF-related symptoms among family caregivers of stroke patients. Reflexive thematic analysis will be employed to analyse, organise and summarise qualitative data, to identify the experiences and management needs related to SRF among family caregivers during home care. Through this comprehensive mixed-methods approach, the study aims to: investigate the levels of SRF experienced by family caregivers of stroke patients, identify patterns and trajectories of related symptoms. The integration of cross-sectional and longitudinal data allows for a thorough examination of both immediate and long-term aspects of caregiver experiences, providing valuable insights into the complex dynamics of SRF in this population.</p><p><strong>Ethics and dissemination: </strong>The study protocol was approved by the Medical Ethics Committee of the University of South China (approval number 2023-NHHL-051). Data collection was authorised by the ethics committees of the First Affiliated Hospital, Second Affiliated Hospital and Nanhua Affiliated Hospital of the University of South China. The results of this study will be disseminated through publication in pertinent peer-reviewed journals, presentation at local and international conferences, and communication with all relevant stakeholders.</p><p><strong>Trial registration number: </strong>ChiCTR2400082717.</p>","PeriodicalId":9158,"journal":{"name":"BMJ Open","volume":"15 3","pages":"e092724"},"PeriodicalIF":2.4,"publicationDate":"2025-03-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143572144","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cost-effectiveness of atezolizumab plus bevacizumab versus sorafenib as first-line therapy in unresectable hepatocellular carcinoma in the US and Chinese setting: a modelling comparison study.","authors":"Lulu Liu, Lei Wang, Yiling Ding, Qilin Zhang, Yamin Shu","doi":"10.1136/bmjopen-2024-094804","DOIUrl":"https://doi.org/10.1136/bmjopen-2024-094804","url":null,"abstract":"<p><strong>Objective: </strong>Atezolizumab plus bevacizumab demonstrates a significant improvement in overall survival and progression-free survival compared with sorafenib in patients with unresectable hepatocellular carcinoma (HCC). The combined usage of these two medications could result in substantial consumption of resources, primarily due to their exceptionally high costs. The current study aims to evaluate the cost-effectiveness of atezolizumab plus bevacizumab as a first-line treatment for advanced HCC from the perspective of payers in developed and developing countries.</p><p><strong>Design: </strong>A partitioned survival model was constructed to evaluate the cost-effectiveness of atezolizumab plus bevacizumab versus sorafenib as a first-line treatment for advanced HCC. The efficacy and safety data incorporated within the model were derived from the IMbrave150 trial. Costs and utilities were extracted from published sources.</p><p><strong>Interventions: </strong>Atezolizumab plus bevacizumab versus sorafenib.</p><p><strong>Outcome measures: </strong>Estimates were calculated for costs, life-years, quality-adjusted life-years (QALYs), incremental cost-effectiveness ratio (ICER) for both treatment strategies. One-way sensitivity, probabilistic sensitivity, expected value of perfect information (EVPI), subgroup and scenario analyses were conducted.</p><p><strong>Results: </strong>The combination therapy of atezolizumab and bevacizumab results in an additional 0.72 life-years/0.57 QALYs in the USA and 0.64 life-years/0.47 QALYs in China compared with standard sorafenib treatment, although with a significant increase in costs, yielding an average ICER of US$253 247.07/QALY in the USA and US$181 552.71/QALY in China. The probability sensitivity analysis indicated that atezolizumab plus bevacizumab demonstrated a 13.60% likelihood of cost-effectiveness in the USA, whereas this likelihood is negligible (0%) in China. The expected value of uncertainty, as quantified by the EVPI, was estimated at approximately US$3658.41/patient in the USA and US$0/patient in China. The ICER was most sensitive to the cost of subsequent treatment in the USA, and most sensitive to the cost of atezolizumab in China. In scenario analyses, the atezolizumab plus bevacizumab treatment becomes favourable when the cost of atezolizumab decreases to 67.85% and 18.45% of its original price in the USA and China, respectively.</p><p><strong>Conclusions: </strong>The atezolizumab plus bevacizumab is unlikely to be cost-effective compared with sorafenib for patients with unresectable HCC in the context of the USA and China. The implementation of significant reductions in drug prices may render the treatment economically viable.</p>","PeriodicalId":9158,"journal":{"name":"BMJ Open","volume":"15 3","pages":"e094804"},"PeriodicalIF":2.4,"publicationDate":"2025-03-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143572080","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Risk prediction models for dental caries in children and adolescents: a systematic review and meta-analysis.","authors":"Xijia Wang, Peng Zhang, Huifei Lu, Dandan Luo, Dunhui Yang, Kang Li, Shuqi Qiu, Haotao Zeng, Xianhai Zeng","doi":"10.1136/bmjopen-2024-088253","DOIUrl":"10.1136/bmjopen-2024-088253","url":null,"abstract":"<p><strong>Objective: </strong>This study aimed to systematically evaluate published predictive models for dental caries in children and adolescents.</p><p><strong>Design: </strong>A systematic review and meta-analysis of observational studies.</p><p><strong>Data sources: </strong>Comprehensive searches were conducted in PubMed, Web of Science, Cochrane Library, Cumulative Index to Nursing and Allied Health Literature, Embase, China National Knowledge Infrastructure, Wanfang Database, China Science and Technology Journal Database (VIP) and SinoMed for relevant studies published up to 18 January 2024. The search focused on caries prediction models in children and adolescents.</p><p><strong>Eligibility criteria: </strong>Eligible studies included observational research (cohort, case-control and cross-sectional designs) that developed risk prediction models for dental caries in children and adolescents aged ≤18 years. Each model was required to include a minimum of two predictors. Studies were excluded if they were not available in English or Chinese, primarily focused on oral microbiome modelling, or lacked essential details regarding study design, model construction or statistical analyses.</p><p><strong>Results: </strong>A total of 11 studies were included in the review. All models demonstrated a high risk of bias, primarily due to inappropriate statistical methods and unclear applicability resulting from insufficiently detailed presentations of the models. Logistic regression, random forests and support vector machines were the most commonly employed methods. Frequently used predictors included fluoride toothpaste use and brushing frequency. Reported area under the curve (AUC) values ranged from 0.57 to 0.91. A combined predictive model incorporating six caries predictors achieved an AUC of 0.79 (95% CI: 0.73 to 0.84).</p><p><strong>Conclusions: </strong>Simplified predictive models for childhood caries showed moderate discriminatory performance but exhibited a high risk of bias, as assessed using the Prediction Model Risk of Bias Assessment Tool (PROBAST). Future research should adhere to PROBAST guidelines to minimise bias risk, focus on enhancing model quality, employ rigorous study designs and prioritise external validation to ensure reliable and generalisable clinical predictions.</p><p><strong>Prospero registration number: </strong>CRD42024523284.</p>","PeriodicalId":9158,"journal":{"name":"BMJ Open","volume":"15 3","pages":"e088253"},"PeriodicalIF":2.4,"publicationDate":"2025-03-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143566052","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}