EClinicalMedicine最新文献

{"title":"Estimating cardiovascular effects of influenza vaccination in older adults: a target trial emulation using proximal causal inference.","authors":"Jinxin Guo, Tiansheng Wang, Zhike Liu, Weihong Zeng, Peng Shen, Yexiang Sun, Siyan Zhan, Yang Xu","doi":"10.1016/j.eclinm.2025.103449","DOIUrl":"10.1016/j.eclinm.2025.103449","url":null,"abstract":"<p><strong>Background: </strong>The substantial burden of cardiovascular diseases highlights the urgent need for cost-effective interventions to mitigate their impact. While existing evidence on the cardioprotective effect of the influenza vaccine comes primarily from populations with cardiovascular comorbidities, these studies remain susceptible to several sources of bias, including immortal time bias and unmeasured confounding. To attenuate these limitations, our study aimed to assess the effect of influenza vaccination on cardiovascular events in an older population in China, utilizing a target trial emulation framework in conjunction with a proximal causal inference (PCI) approach.</p><p><strong>Methods: </strong>This is a sequentially designed, propensity score (PS) matched, vaccine effectiveness study under a target trial emulation framework. We used data from the Yinzhou Regional Health Care Database and included permanent residents of Yinzhou, China, aged 65 years or older. We employed a sequential trial approach in which participants were categorized as influenza vaccinees or non-vaccinees based on their vaccination regimen during the one-week enrollment period of each sequential trial from 2020 to 2022. The outcomes of interest were major adverse cardiovascular events (MACE) and acute coronary syndromes (ACS) within one year of follow-up. To address measured and unmeasured confounding, PS matching was performed in conjunction with PCI using a two-stage Poisson regression to estimate incidence rate ratios (IRRs).</p><p><strong>Findings: </strong>A total of 8,181,638 older adults were included across the 50 emulated trials between 2020 and 2022. Of these, 170,011 received influenza vaccination, while 8,011,627 remained unvaccinated. Vaccinated participants were generally frailer (severely frail: 19.1% <i>vs.</i> 14.7%) and had a higher prevalence of hypertension (83.0% <i>vs.</i> 74.9%). After PS matching, all measured characteristics were well-balanced among 339,976 matched participants. In conjunction with the PCI approach, we found influenza vaccination was associated with a decrease in one-year risk of MACE (IRR: 0.86 [95% CI: 0.83-0.89]) and one-year risk of ACS (IRR: 0.87 [95% CI: 0.83-0.91]) compared to non-vaccination. Results were consistent across strata of enrollment year, age, sex, current smoking status, hypertension, hyperlipidemia, prior influenza vaccination status, and numerous sensitivity analyses.</p><p><strong>Interpretation: </strong>Influenza vaccination may reduce the risk of MACE and ACS among older adults. Aligned with the World Health Organization guidelines, our findings further support influenza vaccination as an effective public health strategy for potentially reducing cardiovascular disease burden.</p><p><strong>Funding: </strong>National Natural Science Foundation of China; Science and Technology Project of Science and Technology Bureau of Yinzhou District, Ningbo City; Zhejiang Provincial Centre for Disease","PeriodicalId":11393,"journal":{"name":"EClinicalMedicine","volume":"87 ","pages":"103449"},"PeriodicalIF":10.0,"publicationDate":"2025-08-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12398818/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144946938","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Antibiotic treatment of pediatric infections in primary healthcare setting: evaluation and comparison of 80 national treatment guidelines with the WHO AWaRe book recommendations.","authors":"Daniele Donà, Giulia Brigadoi, Anna Cantarutti, Giovanni Autore, Marco Masetti, Shrey Mathur, Mike Sharland, Susanna Esposito","doi":"10.1016/j.eclinm.2025.103437","DOIUrl":"10.1016/j.eclinm.2025.103437","url":null,"abstract":"<p><strong>Background: </strong>Antibiotic recommendations for pediatric infections in national standard treatment guidelines (STGs) vary widely, particularly for Access and Watch antibiotics. The WHO AWaRe book recommends Access antibiotics as first-line treatment for over 80% of common infections managed in primary healthcare. This study aims to evaluate the agreement between first and second-line antibiotics in national STGs with AWaRe book recommendations and the inclusion of these antibiotics in Essential Medicine Lists (EMLs).</p><p><strong>Methods: </strong>National STGs of 80 countries were systematically collected from databases and grey literature (up to May 2025). Antibiotic recommendations for the ten most common primary healthcare infections in children were compared with the WHO AWaRe book (2022), the WHO Essential Medicines List for children (EMLc) and national Essential Medicines Lists (nEMLs) where available.</p><p><strong>Findings: </strong>A median of eight STGs per country were collected, with higher numbers in LMICs due to guidelines for cholera and enteric fever. A total of 1124 first-line and 841 second-line antibiotic recommendations were identified. Over 70% of first-line recommended treatments were Access antibiotics, while Watch antibiotics accounted for more than 50% of second-line recommended treatments. First-line recommendations showed strong agreement with WHO guidance, whereas second-line treatments exhibited lower agreement and greater variability across regions. More than 80% of first-line antibiotics were included in the EMLc and nEMLs, although some high-income countries lacked nEMLs.</p><p><strong>Interpretation: </strong>First-line antibiotic recommendations in national pediatric STGs largely align with the WHO AWaRe book guidance focusing on Access antibiotic use. In contrast, second-line treatments vary considerably, commonly recommending Watch antibiotics. Strengthening the evidence base of national STGs and aligning second-line recommendations with the WHO AWaRe book could help meet the 79th UNGA High-Level Meeting on AMR target, which aims for 70% of all human antibiotic use to come from the Access group.</p><p><strong>Funding: </strong>PRIN 2022 \"A Cluster randomized clinical trial to change Antibiotic Prescribing behavior in Outpatient pediatric primary care setting in Italy (CAPO project)\", funded in the framework of the National Recovery and Resilience Plan (NRRP), Mission 4, Component 2, Investment 1.1, funded by the European Union-Next Generation EU, Project 2022A7LA2W, CUP C53D23006050006.</p>","PeriodicalId":11393,"journal":{"name":"EClinicalMedicine","volume":"87 ","pages":"103437"},"PeriodicalIF":10.0,"publicationDate":"2025-08-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12396473/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144946837","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The prevalence and risk of mortality associated with antimicrobial resistance within nosocomial settings-a global systematic review and meta-analysis of over 20,000 patients.","authors":"Nisha A George, Daniel Pan, Luisa Silva, Rebecca F Baggaley, Patricia Irizar, Pip Divall, Amani Al-Oraibi, Durdana P Khan, Christopher A Martin, Joshua Nazareth, Ian Collins, See Lek Chew, Laura B Nellums, Manish Pareek","doi":"10.1016/j.eclinm.2025.103384","DOIUrl":"10.1016/j.eclinm.2025.103384","url":null,"abstract":"<p><strong>Background: </strong>Bacterial antimicrobial resistance (AMR) is a leading cause of death globally. However, there has been no data synthesis on whether it influences mortality within hospital settings. We conducted a systematic review and meta-analysis to quantify the prevalence and risk of mortality associated in hospitalised patients with AMR, compared to patients with infections not classified as AMR.</p><p><strong>Methods: </strong>Databases (MEDLINE, EMBASE, and Cochrane library) were searched from inception up to 14th April 2025 for studies that reported the prevalence of AMR in patients who acquired infections in hospitals and mortality (PROSPERO CRD42023420609). We calculated pooled prevalence estimates of AMR as well as unadjusted and adjusted estimates of the effect of AMR on mortality using a random-effects model. Study quality was assessed using the Joanna Briggs Quality Appraisal Tool, risk of bias using DOI plots and LFK index and certainty of evidence of mortality using GRADE criteria.</p><p><strong>Findings: </strong>We identified 34 studies (20,658 patients with resistant organisms) from 18 countries-namely the USA, China, the UK, Canada, Israel, Japan, Malaysia, Korea, Brazil, and Singapore. Of these, 33 were observational studies whilst two studies (one observational study and one purely modelling study) mechanistically modelled risk of mortality in relation to transmission. No studies were conducted in the African subcontinent, the Middle-East, Russia, and India. The prevalence of AMR was high in patients in hospital (pooled prevalence: 36.5%, 95% CI: 29%-44%, <i>I</i> ^<i>2</i> = 99%) and associated with higher mortality (unadjusted pooled risk ratio [RR]: 1.64, 95% CI: 1.37-1.97, <i>I</i> ^<i>2</i> = 96.22%, τ² = 0.20; adjusted pooled RR: 1.58, 95% CI: 1.33-1.87, <i>I</i> ^<i>2</i> = 85.9%, τ² = 0.13) compared to non-AMR organisms.Sensitivity analyses showed particularly elevated risks for in-hospital mortality and for AMR-associated bacteraemia. Study quality was generally rated to be high, but there was evidence of publication bias in estimates of both prevalence and mortality. Overall certainty of evidence of mortality was graded to be low.</p><p><strong>Interpretation: </strong>AMR is highly prevalent within hospital settings globally and associated with increased in-hospital mortality. Crucially, no data was identified from the India subcontinent, African subcontinent, the Middle East, and Russia, and only two studies used mechanistic modelling to explore how transmission of AMR affects mortality. Further research is required, particularly in underrepresented regions to inform interventions aimed at reducing both AMR transmission and its related mortality within hospital settings.</p><p><strong>Funding: </strong>Pacific Life.</p>","PeriodicalId":11393,"journal":{"name":"EClinicalMedicine","volume":"87 ","pages":"103384"},"PeriodicalIF":10.0,"publicationDate":"2025-08-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12496217/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145238279","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Complexity of interstitial lung disease disproportionality signal analysis in pharmacovigilance databases.","authors":"Romane Freppel, Alex Hlavaty, Imen Bari, Charles Khouri, Aurélie Grandvuillemin","doi":"10.1016/j.eclinm.2025.103450","DOIUrl":"10.1016/j.eclinm.2025.103450","url":null,"abstract":"","PeriodicalId":11393,"journal":{"name":"EClinicalMedicine","volume":"87 ","pages":"103450"},"PeriodicalIF":10.0,"publicationDate":"2025-08-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12396481/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144946897","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Two-year outcomes after selective early treatment of patent ductus arteriosus with ibuprofen in preterm babies: follow-up of Baby-OSCAR-a randomised controlled trial.","authors":"Samir Gupta, Heather O'Connor, Edmund Juszczak, Nimish V Subhedar, Ursula Bowler, Charlotte Clarke, David Field, Elizabeth Hutchison, Wilf Kelsall, Justine Pepperell, Tracy Roberts, Sunil Sinha, Kayleigh Stanbury, Jonathan Wyllie, Pollyanna Hardy, Samantha Johnson","doi":"10.1016/j.eclinm.2025.103424","DOIUrl":"10.1016/j.eclinm.2025.103424","url":null,"abstract":"<p><strong>Background: </strong>Children born extremely preterm are at increased risk of developmental problems and respiratory morbidity due to patent ductus arteriosus (PDA). The objective of this study was to evaluate whether early treatment of a PDA ≥1.5 mm with ibuprofen improved neurodevelopmental and respiratory outcomes at 24 months of age, corrected for prematurity.</p><p><strong>Methods: </strong>Baby-OSCAR was a UK multi-center placebo-controlled masked randomized clinical trial in infants born 23⁺⁰-28⁺⁶ weeks' gestation. The main long-term outcome was survival without moderate or severe neurodevelopmental impairment at 24 months' corrected age, assessed using parent report primarily or classified by blinded end-point review committee where parent-reported data were not available. Other secondary outcomes included survival without respiratory morbidity and duration of oxygen supplementation. (ISRCTN Registry number ISRCTN84264977).</p><p><strong>Findings: </strong>From July 2015 through December 2020, 653 infants underwent randomization. At 24 months' corrected age, outcome data were available for 537 children: 263 in the ibuprofen group and 274 in the placebo group. Survival without moderate to severe neurodevelopmental impairment in the ibuprofen and placebo groups was 131/248 (53.0%) and 134/259 (51.9%) respectively; adjusted risk ratio 1.01 (95% confidence interval [CI] 0.86-1.18); p = 0.901. Survival without respiratory morbidity was 66/220 (30%) and 74/225 (32.9%) respectively; adjusted risk ratio 0.89 (95% CI 0.68-1.18). Median duration of oxygen supplementation from randomization was 76.0 and 78.0 days, respectively; adjusted median difference -1.5 (-13.8 to 10.9).</p><p><strong>Interpretation: </strong>We found no evidence of an improvement in neurodevelopmental and respiratory outcomes at 24 months' corrected age, after selective early treatment of a PDA ≥1.5 mm with ibuprofen in children born extremely preterm.</p><p><strong>Funding: </strong>This study was funded by the National Institute for Health Research (NIHR) Health Technology Assessment Programme (11/92/15).</p>","PeriodicalId":11393,"journal":{"name":"EClinicalMedicine","volume":"87 ","pages":"103424"},"PeriodicalIF":10.0,"publicationDate":"2025-08-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12396396/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144946548","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Age-specific protective effects of statins against cirrhosis in patients with chronic liver enzyme elevation associated with metabolic dysfunction: a retrospective cohort study of electronic health records.","authors":"Ayako Suzuki, Georgia Sofia Karachaliou, Amy M Perkins, Chad Dorn, Ruth M Reeves, Timothy Arnold, Mustafa R Bashir, Jimmy T Efird, Manal F Abdelmalek, Anna Mae Diehl","doi":"10.1016/j.eclinm.2025.103431","DOIUrl":"10.1016/j.eclinm.2025.103431","url":null,"abstract":"<p><strong>Background: </strong>The hepatoprotective effects of statins in chronic liver diseases are well-documented; however, variation by age, sex, and formulation remains unclear. The optimal regimen for cirrhosis prevention has yet to be defined. We aimed to address this knowledge gap.</p><p><strong>Methods: </strong>In this retrospective cohort study using Veterans Affairs (VA) electronic health records, we defined a cohort of patients with chronic liver enzyme elevation primarily associated with metabolic dysfunction, between Jan 1, 2007, and Dec 31, 2009. Patients were followed-up for incident cirrhosis for a 10-year period until 2019. Time to cirrhosis was analysed using Cox proportional hazards models, with and without adjustment for demographics, relevant comorbidities, and co-medications. Hazard ratio (HR) estimates were used to compare event rates between groups. We also assessed potential effect modification by age and sex and evaluated differences across statin formulations.</p><p><strong>Findings: </strong>In adjusted models, baseline statin use was significantly associated with a reduced risk of cirrhosis over 10 years (HR 0.74 [95% confidence interval 0.70-0.78], p < 0.0001). Cumulative statin dose, standardised by low-density lipoprotein (LDL)-lowering intensity as simvastatin-equivalent units, and duration were dose-dependently associated with cirrhosis risk reduction (p < 0.05). The protective effect of statin use, at baseline and during follow-up, demonstrated a significant effect modification with age (p ≤ 0.01), with greater protection in older individuals. No sex disparities were observed. Borderline to significant protection against cirrhosis was achieved with a daily dose of ≥6961 mg simvastatin-equivalent for at least 245 days per year among individuals aged ≥54 years. Those younger than 54 years required a higher dose (>15,561 mg annually) to achieve comparative protection. No significant differences in effectiveness were observed among different formulations.</p><p><strong>Interpretation: </strong>Statins reduce cirrhosis risk in a dose- and age-dependent manner. A daily dose equivalent to ≥20 mg simvastatin (or >40 mg for <54 years) appears effective, regardless of formulation. Independent validation in a cohort with a higher proportion of women is warranted.</p><p><strong>Funding: </strong>The Department of Defence, Translational Team Science Award.</p>","PeriodicalId":11393,"journal":{"name":"EClinicalMedicine","volume":"87 ","pages":"103431"},"PeriodicalIF":10.0,"publicationDate":"2025-08-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12396468/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144946869","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Immunogenicity and safety of self-amplifying mRNA COVID-19 vaccine (ARCT-2303), with or without co-administration of seasonal inactivated influenza vaccine in adults: a phase 3, randomised, controlled, observer-blind, multicentre study.","authors":"Michelle L Giles, Charissa Tabora, Carmen Baccarini, Leonela Barrientos, Javier Cespedes Vargas, May Emmeline Montellano, Paul Nguyen, Sachin Deshmukh, Munro Neville, Matthew Hohenboken, Josephine van Boxmeer, Hongfan Jin, Roberto Bugarini, Xuexuan Liu, Judd L Walson, Carole Verhoeven, Igor Smolenov","doi":"10.1016/j.eclinm.2025.103428","DOIUrl":"10.1016/j.eclinm.2025.103428","url":null,"abstract":"<p><strong>Background: </strong>A recently licenced self-amplifying mRNA (sa-mRNA) COVID-19 vaccine induces a robust, broad, and long-lasting immune response, extending the arsenal of efficacious COVID-19 countermeasures. We ran a clinical study to assess the benefits of vaccine strain update and the feasibility of co-administration with influenza vaccines.</p><p><strong>Methods: </strong>Between March 27, 2024 and April 10, 2025, we performed a randomised, observer-blind, placebo-controlled, phase 3 study with 1499 adult participants to compare immune responses of sa-mRNA vaccine, encoding spike glycoprotein of the XBB.1.5 subvariant (ARCT-2303), with vaccine encoding the ancestral strain (ARCT-154), as measured by geometric mean titres of neutralising antibodies and SARS-CoV-2 neutralising antibody seroconversion rates against the Omicron XBB.1.5, and to assess the immunological non-inferiority of co-administered ARCT-2303 and influenza vaccines compared with separately administered vaccines, as measured by neutralising antibodies against Omicron XBB.1.5.6 and haemagglutinin inhibition against influenza vaccine strains. Reactogenicity (adverse events on Days 1-7) and safety (adverse events on Days 1-181) were also assessed. The trial was registered on ClinicalTrials.gov (identifier NCT06279871).</p><p><strong>Findings: </strong>The geometric mean ratio (ARCT-2303/ARCT-154) of neutralising antibodies against Omicron XBB.1.5.6 on Day 29 was 2.7 (95% confidence interval (CI): 2.3-3.2), and the seroconversion rate difference was 28.4% (21.8-34.9); both met the prespecified superiority criteria. Concomitant administration of ARCT-2303 had no impact on the immune response to the quadrivalent influenza vaccine antigens, whether the non-adjuvanted vaccine given to 18‒64 year-old adults or the adjuvanted vaccine given to adults 65 years and older. The non-inferiority of the immune response against Omicron XBB.1.5.6 was also demonstrated when ARCT-2303 was co-administered or administered separately.</p><p><strong>Interpretation: </strong>We conclude that ARCT-2303 induces a robust immune response against the vaccine variant of SARS-CoV-2 and can be co-administered with licenced influenza vaccines in adults with no impact on the safety or immunogenicity of either vaccine.</p><p><strong>Funding: </strong>The study is funded by CSL under a collaboration agreement with Arcturus Therapeutics.</p>","PeriodicalId":11393,"journal":{"name":"EClinicalMedicine","volume":"87 ","pages":"103428"},"PeriodicalIF":10.0,"publicationDate":"2025-08-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12396474/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144946912","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Development of a core patient-centred outcome set for adults living with obesity: a modified delphi-based international consensus.","authors":"Teferi Mekonnen, Leanne Staniford, Spencer Connell, Zofia Das-Gupta, Umanga DeSilva, Yasmine Saoud, Isabel Miller, Mohapradeep Mohan, Simon Nienhuijs, Megha Poddar, Vaishali Chetan Deshmukh, Marli Conradie-Smit, Soo Huat Teoh, Sanjeev Sockalingam, Emilia Huvinen, Paolo Sbraccia, Ronald Liem, Michael Vallis, Kenneth Clare, Tracy Zvenyach, Karen Coulman, Marianela Aguirre Ackermann, Verónica Vázquez-Velázquez, Yudith Preiss Contreras, Josep Vidal, Agbo Urudinachi, María Fernanda Tejeda-Muñoz, Ian Patton, T Alafia Samuels, Morgan Emile Gabriel Salmon Leguede, Georgia Rigas, Ximena Ramos Salas, Michael Crotty, Bruno Halpern, Daniel Bessesen, Laura Pizzi, Omniyat Mohammed Al Hajeri, Arya M Sharma","doi":"10.1016/j.eclinm.2025.103422","DOIUrl":"10.1016/j.eclinm.2025.103422","url":null,"abstract":"<p><strong>Background: </strong>Obesity is a chronic disease linked to over 200 health conditions, reduced quality of life, and increased mortality. Despite the availability of multimodal treatments, there is a lack of standardised, patient-centred outcome measures to effectively assess and improve clinical care. This project aimed to define a core set of standardised outcome measures for adults with obesity, incorporating both patient-reported and clinician-reported outcomes. Additional objectives included determining the optimal measurement frequency and identifying case-mix variables essential for risk adjustment.</p><p><strong>Methods: </strong>The International Consortium for Health Outcomes Measurement (ICHOM) established the Obesity Working Group (OWG), composed of 29 international experts and individuals living with obesity. Members were selected based on their expertise in obesity care and research and represented 21 countries. The development process, conducted from May 2023 to July 2024, began with a kick-off meeting to define scope, followed by eight virtual meetings. A comprehensive literature review informed the identification of relevant clinical outcomes, patient-reported outcomes, treatment-related complications, and case-mix variables. A three-round Delphi process was used to reach consensus on key outcomes and follow-up intervals. Outcomes were included in the final set if at least 80% of OWG members rated them between 7 and 9 on a 9-point scale. Validation was conducted through surveys with 95 patients and 106 healthcare professionals from fields such as dietetics, obesity medicine, and general practice.</p><p><strong>Findings: </strong>The OWG developed a standardised set of 20 outcome measures, comprising 17 core measures and 3 population-specific measures. These span domains including physical health, psychosocial well-being, health behaviours, body functioning, and adverse events. Specific measures were also developed for bariatric surgery and female reproductive health (both pregnant and non-pregnant individuals). Key case-mix factors and appropriate follow-up periods were defined to support global consistency in reporting.</p><p><strong>Interpretation: </strong>Adoption of this standardised outcome set enables clinicians, patients, and stakeholders to identify care gaps, monitor outcomes, and evaluate the quality of obesity care against global benchmarks. Implementation can support the creation of a robust international data resource to guide research, inform clinical practice, and shape policy and guidelines for adult obesity treatment. Limitations include limited geographical diversity in validation surveys, the exclusion of internalised weight bias due to a lack of validated measurement tools, and limited applicability to individuals with obesity and multiple comorbidities.</p><p><strong>Funding: </strong>Novo Nordisk, Eli Lilly, and Boehringer Ingelheim International GmbH.</p>","PeriodicalId":11393,"journal":{"name":"EClinicalMedicine","volume":"87 ","pages":"103422"},"PeriodicalIF":10.0,"publicationDate":"2025-08-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12396397/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144946930","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Response to \"complexity of interstitial lung disease disproportionality signal analysis in pharmacovigilance databases\".","authors":"Zijun Zhu, Yongxin Li, Jiuda Zhao","doi":"10.1016/j.eclinm.2025.103451","DOIUrl":"10.1016/j.eclinm.2025.103451","url":null,"abstract":"","PeriodicalId":11393,"journal":{"name":"EClinicalMedicine","volume":"87 ","pages":"103451"},"PeriodicalIF":10.0,"publicationDate":"2025-08-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12394965/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144946960","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Development and validation of machine learning models to predict esophagogastric variceal rebleeding risk in HBV-related cirrhosis after endoscopic treatment: a prospective multicenter study.","authors":"Linlin Zheng, Nannan Shi, Peizhao Li, HongFei Ge, Chuantao Tu, Ying Qu, Yin Wang, Yuanyuan Lin, Shiyao Chen, Dalong Sun, Chengzhao Weng, Shengdi Wu, Wei Jiang","doi":"10.1016/j.eclinm.2025.103436","DOIUrl":"10.1016/j.eclinm.2025.103436","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;Rebleeding after initial endoscopic therapy is associated with high mortality in patients with hepatitis B virus (HBV)-related liver cirrhosis complicated by esophagogastric variceal bleeding (EGVB), imposing a substantial public health burden. Spontaneous portosystemic shunts (SPSS), a compensatory mechanism for portal hypertension, are closely associated with disease progression. This study aimed to develop and validate machine learning (ML) models incorporating clinical and imaging features to predict the risk and frequency of rebleeding following initial endoscopic treatment.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;This multicenter prospective study enrolled patients with HBV-related cirrhosis and EGVB treated at Zhongshan Hospital, Fudan University (the development cohort). External validation was completed in five tertiary centers in China. The trial was registered at ClinicalTrials.gov, NCT03277651. Data were collected between January 2017 and January 2022. Five classic ML algorithms, Hierarchical Gradient Boosting (HGB), Multilayer Perceptron (MLP), Random Forest (RF), Support Vector Machine (SVM), and Extreme Gradient Boosting with classification trees (XGB), were utilized to predict rebleeding. Model performance was evaluated using the area under the receiver operating characteristic curve (AUC), accuracy, sensitivity, specificity, and F1 score. Time-dependent ML was further applied, with predictive performance compared between conventional and time-dependent models using the concordance index (C-index). The optimal model was interpreted via Shapley Additive Explanations (SHAP) and externally validated. Additionally, key predictors were integrated into a Support Vector Regression (SVR) model to estimate rebleeding frequency.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Findings: &lt;/strong&gt;Among 295 patients in the development cohort and 190 in the external cohort, rebleeding occurred in 77 and 68 patients with SPSS, respectively. The XGB model demonstrated the best discrimination (AUCs: 0.814 internal, 0.776 external), significantly outperforming the other models (&lt;i&gt;P&lt;/i&gt; = 0.014, 0.008). Compared with the Model for End-stage Liver Disease (MELD) and Child-Pugh scores (AUCs: 0.557 and 0.590), the XGB model significantly improved rebleeding prediction (&lt;i&gt;P &lt;&lt;/i&gt; 0.0001). SHAP analysis identified hemoglobin, portal vein thrombosis, superior mesenteric vein diameter, platelet count, minimum shunt diameter, and splenic vein diameter as the top predictors. The SVR model achieved robust performance in estimating rebleeding frequency, with mean squared error (MSE) and R&lt;sup&gt;2&lt;/sup&gt; values of 0.030 and 0.914 in the training set, 0.073 and 0.777 in the internal validation set, and 0.143 and 0.708 in the external validation set.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Interpretation: &lt;/strong&gt;The ML-based model offers a noninvasive, accurate tool for individualized risk stratification and follow-up planning in patients with HBV-related cirrhosis and SPSS after initial endoscopi","PeriodicalId":11393,"journal":{"name":"EClinicalMedicine","volume":"87 ","pages":"103436"},"PeriodicalIF":10.0,"publicationDate":"2025-08-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12395071/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144946882","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}