Lancet Regional Health-Europe最新文献

{"title":"Evaluating the effect of probiotics on severe necrotising enterocolitis in preterm infants born before 32 weeks gestation in England and Wales: a propensity-matched population study","authors":"Alice Aveline ,&nbsp;Lisa Szatkowski ,&nbsp;Janet Berrington ,&nbsp;Kate Costeloe ,&nbsp;Shalini Ojha ,&nbsp;Paul Fleming ,&nbsp;Cheryl Battersby","doi":"10.1016/j.lanepe.2025.101571","DOIUrl":"10.1016/j.lanepe.2025.101571","url":null,"abstract":"<div><h3>Background</h3><div>Necrotising enterocolitis (NEC) remains an important cause of morbidity and mortality in preterm infants. This study aimed to examine whether probiotics reduce the risk of severe NEC and other key neonatal morbidities including late onset sepsis and mortality.</div></div><div><h3>Methods</h3><div>Retrospective study using the United Kingdom National Neonatal Research Database. Infants &lt;32 weeks gestation in England and Wales (01/01/2016–31/12/2022) were included if alive on day four, without major congenital anomaly. A propensity score matched approach was applied matching for gestational age cohorts, birth year epochs and 17 further items. Comparators were infants who were exposed or not to probiotics within 14 days. Primary outcome was severe NEC (confirmed at laparotomy or postmortem or listed primary cause of death). Parent focus groups and former NICU patients supported this study but did not contribute to design or writing.</div></div><div><h3>Findings</h3><div>48,048 infants (45.2% (21,695/48,048) female), median gestational age 29.4 weeks (IQR 27.4–30.9) were included; 25.3% (12,161/48,048) were exposed to at least one of five available probiotics. 3.6% (1728/48,048) had severe NEC. Of 16,586 infants (8293 exposed and 8293 unexposed) in the propensity-matched analysis, incidence and odds ratios (OR) (95% CI) for exposed versus unexposed for severe NEC was 3.3% versus 4.2%, OR 0.80 (0.72–0.89); <em>other definitions of NEC yielded similar results.</em> Incidence for late onset sepsis (10.8% versus 11.5%, OR 0.94 (0.90–0.97)) and survival to discharge (96.6% versus 94.2%, OR 1.76 (1.65–1.88)). In infants &lt;28 weeks gestation, severe NEC (8.7% versus 9.8%, OR 0.88 (0.82–0.93) and for ≥28 weeks (1.0% versus 1.7%, OR 0.59 (0.47–0.73).</div></div><div><h3>Interpretation</h3><div>Probiotics were associated with a reduction in severe NEC including in those &lt;28 weeks gestation. We currently recommend neonatal units not already using probiotics, to consider the introduction of products meeting appropriate recommendations, in the context of their local morbidity rates.</div></div><div><h3>Funding</h3><div><span>NIHR Advanced Fellowship</span> (CB reference: NIHR300617), <span>Imperial College PhD studentship</span> (AA), <span>NIHR RfPB</span> grant (NIHR203590, SO), <span>Imperial NIHR Biomedical Research Centre</span>.</div></div>","PeriodicalId":53223,"journal":{"name":"Lancet Regional Health-Europe","volume":"62 ","pages":"Article 101571"},"PeriodicalIF":13.0,"publicationDate":"2025-12-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145841221","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Anterior cruciate ligament reconstruction combined with anterolateral ligament reconstruction using hamstring autograft versus anterior cruciate ligament reconstruction using bone–patellar tendon–bone autograft: a randomised controlled trial with 5-year follow-up","authors":"Bertrand Sonnery-Cottet ,&nbsp;Alessandro Carrozzo ,&nbsp;Hervé Poilvache ,&nbsp;Jean-Marie Fayard ,&nbsp;Benjamin Freychet ,&nbsp;Mathieu Thaunat ,&nbsp;Thais Dutra Vieira ,&nbsp;Adnan Saithna","doi":"10.1016/j.lanepe.2025.101561","DOIUrl":"10.1016/j.lanepe.2025.101561","url":null,"abstract":"<div><h3>Background</h3><div>Anterior Cruciate Ligament (ACL) rupture is common knee injury. Although ACL reconstruction (ACLR) is standard, graft failure rates remain high in young active patients. This study investigated whether combining ACLR with anterolateral ligament (ALL) reconstruction (ALLR) reduces grafts failure compared with ACLR.</div></div><div><h3>Methods</h3><div>In this prospective, single-centre, randomised controlled trial conducted at the Santy Orthopedic Center in Lyon, France, patients aged 18–35 years with symptomatic ACL rupture were randomly allocated (1:1) to ACL + ALL reconstruction using hamstring tendon autograft (ACLR + ALLR) or ACLR with bone-patellar tendon-bone autograft (ACLR). Randomisation was performed with a block size of four using telematic software by an independent study coordinator, with concealed allocation. Surgeons were informed of the assigned procedure on the morning of surgery. Outcome assessors were not blinded. The primary outcome was graft failure at 5 years, assessed clinically and by magnetic resonance imaging (MRI) by an independent sports medicine physicians not involved in the index surgery. Efficacy analyses were performed on the Full Analysis Set in accordance with the intention-to-treat principle, while safety analyses were conducted on the Safety Set. Trial registration: <span><span>ClinicalTrials.gov</span><svg><path></path></svg></span>, ID <span><span>NCT03740022</span><svg><path></path></svg></span>. The trial has been completed.</div></div><div><h3>Findings</h3><div>Between November 11, 2016, and January 20, 2020, 593 patients were randomized (297 assigned to ACLR + ALLR and 296 to ACLR). The mean age was 25.0 years (SD 4.5); 447 (75%) participants were male and 146 (25%) female. Of these 593 patients, 556 (94%) completed a mean 5-year follow-up. Graft failure occurred in 12/283 (4.2%) with ACLR + ALLR versus 28/273 (10.3%) with ACLR (p = 0.006; adjusted odds ratio 2.54 [95% CI 1.27; 5.36]—p = 0.008). The number needed to treat was 17 overall, and 9 in patients younger than 25 years.</div></div><div><h3>Interpretation</h3><div>In our study of young, active adults with ACL rupture, who are considered high-risk for graft failure, combining ACL reconstruction with anterolateral ligament reconstruction (ACLR + ALLR) significantly decreased graft failure compared with ACLR. These results suggest that ACLR + ALLR might be beneficial for young or highly active individuals and provide a basis for future research to refine patient selection, evaluate long-term outcomes beyond five years, and explore benefits in other subgroups of patients with ACL injuries.</div></div><div><h3>Funding</h3><div><span>GCS Ramsay Santé pour l'Enseignement et la Recherche</span> funds the scientific activity at the Santy center.</div></div>","PeriodicalId":53223,"journal":{"name":"Lancet Regional Health-Europe","volume":"62 ","pages":"Article 101561"},"PeriodicalIF":13.0,"publicationDate":"2025-12-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145841222","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Doubling the diagnostic rate of at-risk metabolic dysfunction-associated steatohepatitis—leave no one behind","authors":"Elamin Abdelgadir ,&nbsp;Fatheya Alawadi ,&nbsp;Fauzia Rashid ,&nbsp;Alaaeldin Bashir ,&nbsp;Jörn M. Schattenberg","doi":"10.1016/j.lanepe.2025.101572","DOIUrl":"10.1016/j.lanepe.2025.101572","url":null,"abstract":"","PeriodicalId":53223,"journal":{"name":"Lancet Regional Health-Europe","volume":"60 ","pages":"Article 101572"},"PeriodicalIF":13.0,"publicationDate":"2025-12-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145790204","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Socioeconomic disparities in long-term mortality after infective endocarditis in Denmark: a nationwide cohort study","authors":"Søren K. Martiny ,&nbsp;Morten Schmidt ,&nbsp;Jonas A. Povlsen ,&nbsp;Kirstine K. Søgaard ,&nbsp;Hans E. Bøtker ,&nbsp;Henrik T. Sørensen","doi":"10.1016/j.lanepe.2025.101568","DOIUrl":"10.1016/j.lanepe.2025.101568","url":null,"abstract":"<div><h3>Background</h3><div>Socioeconomic position (SEP) influences several determinants of infective endocarditis (IE) progression. Whether IE mortality differs by SEP remains unclear. We examined 5-year mortality after IE by individual-level SEP.</div></div><div><h3>Methods</h3><div>Using nationwide Danish registries, we identified patients with first-time IE (2010–2022). SEP was assessed by educational and affluence level, categorised as low, medium, and high. The Kaplan–Meier estimator provided 30-day, 1-, 3-, and 5-year mortality risks, risk differences, and risk ratios. Time-varying hazard ratios were derived from flexible parametric models. We estimated attendance at outpatient and dental follow-up visits (indicating adherence to follow-up) using the Aalen-Johansen estimator and modelled hospitalisation rate during follow-up (indicating comorbid disease burden) using a joint frailty model, treating death as a competing event in all models.</div></div><div><h3>Findings</h3><div>The 5-year mortality by educational level was 63.1% (95% CI: 60.8%–65.2%) for low, 53.1% (95% CI: 51.3%–54.9%) for medium, and 45.8% (95% CI: 42.5%–48.8%) for high education. The excess mortality was particularly pronounced within the first 2 years. Within one year after discharge, 65.2% (95% CI: 62.9%–67.4%) of low, 73.6% (95% CI: 72.0%–75.2%) of medium, and 74.5% (95% CI: 71.8%–77.2%) of high-education patients had an outpatient contact. The corresponding five year dental visit proportions were 44.0% (95% CI: 41.6%–46.5%), 64.0% (95% CI: 62.2%–65.9%), and 74.0% (95% CI: 71.2%–76.9%), respectively. The hazard ratio for hospitalisations was 1.35 (95% CI: 1.25–1.49) when comparing low <em>vs.</em> high education and 1.19 (95% CI: 1.10–1.27) for low <em>vs.</em> medium. Estimates and time-dependent patterns were similar for affluence level.</div></div><div><h3>Interpretation</h3><div>Patients with low SEP had higher IE mortality, particularly within the first 2 years. Reduced adherence to follow-up care and comorbid diseases may contribute to this. Determining how the excess mortality is directly linked to the IE episode warrants further investigation.</div></div><div><h3>Funding</h3><div><span>Independent Research Fund</span> Denmark (grant no. 3101-00102B) and Center for Population Medicine, <span>Department of Clinical Epidemiology</span>, <span>Aarhus University</span>.</div></div>","PeriodicalId":53223,"journal":{"name":"Lancet Regional Health-Europe","volume":"62 ","pages":"Article 101568"},"PeriodicalIF":13.0,"publicationDate":"2025-12-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145798557","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Effectiveness of an environmental nutrition and physical activity intervention in early childhood education and care settings (NAPSACC UK): a multicentre cluster randomised controlled trial.","authors":"Ruth Kipping, Sharon Anne Simpson, Kim Hannam, Peter S Blair, Russell Jago, Corby K Martin, Zoi Toumpakari, Laura Johnson, James Garbutt, Rachel Maishman, James White, Rebecca Langford, William Hollingworth, Madeleine Cochrane, Laurence Moore, Chris Metcalfe, Anne Martin, Stephanie Chambers, Thomas Reid, Megan Pardoe, Alexandra Dobell, Marie Murphy, Susan Stratton, Jemima Cooper, Jodi Taylor, Miranda Pallan","doi":"10.1016/j.lanepe.2025.101550","DOIUrl":"10.1016/j.lanepe.2025.101550","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;Early childhood education and care (ECEC) provision is widespread. NAPSACC UK is an intervention in ECECs designed to improve nutrition and physical activity policies, practice and provision through ECEC staff workshops, self-assessment and assistance over one year. It was adapted for the UK from the USA and we tested whether it reduced energy consumption and increased physical activity.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;Repeated cross-sectional, multicentre, two-arm, single-blind, parallel-group, cluster-randomised controlled trial including ECEC providers in the UK. The randomisation was conducted by a statistician who was blinded to ECEC provider identity, with allocation within each local authority area and by ECEC Index of Multiple Deprivation scores to minimise differences between arms. Participants were not blind to allocation. Co-primary outcomes after 12-months were child average total energy consumed per eating occasion in the ECEC (lunch or snack) and child accelerometer-assessed total physical activity on ECEC days. Secondary outcomes were moderate-to-vigorous physical activity, sedentary time, energy served and consumed at lunch and snacks, diet quality, and Body Mass Index z-score. The senior statistician and majority of co-investigators were blinded. Analysis was intention-to-treat. Trial registration is ISRCTN33134697 and is completed.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Findings: &lt;/strong&gt;Between 14 March 2022 and 25 March 2024 we enrolled 52 ECEC providers (25 intervention; 27 control) and 835 2-5 year-olds (401 intervention, 434 control). The co-primary outcomes were assessed 12 months after baseline with data provided by 382 children for nutrition and 244 children for physical activity. There was no evidence of a difference in the co-primary outcomes compared to control of average kcal per eating occasion in ECEC (adjusted geometric mean ratio 0.86 (95% CI 0.72-1.03; p = 0.09)) or total physical activity (adjusted mean difference (aMD) -2.13 min (95% CI -10.96 to 6.70; p = 0.64)). There was evidence of lower lunch energy served (aMD -69.1 kcal per occasion (95% CI -116 to -22.2; p = 0.004)) and consumed (aMD -67.7 kcal per occasion (95% CI -118.6 to -18.7, p = 0.009)) with the intervention. There was no evidence of differences in other secondary outcomes. No adverse events were reported.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Interpretation: &lt;/strong&gt;NAPSACC UK did not improve average kcal per eating occasion in ECEC or physical activity. Lower lunch energy servings and consumption closer to recommendations were observed as secondary outcomes. The lower fidelity to the intervention than intended and staffing pressures give insight into interpretation of the null result. Therefore, we recommend that policy-level and statutory changes, which require low agency by individual ECEC settings are research and policy priorities for nutrition and physical activity in ECEC.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Funding: &lt;/strong&gt;National Institute for Health and Care Research ","PeriodicalId":53223,"journal":{"name":"Lancet Regional Health-Europe","volume":"61 ","pages":"101550"},"PeriodicalIF":13.0,"publicationDate":"2025-12-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12882655/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146144557","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Blood culture practices and microbiological capacity for sepsis diagnostics in Europe (2021–2022): a cross-sectional analysis of the European Sepsis Care Survey","authors":"Christian S. Scheer ,&nbsp;Evangelos J. Giamarellos-Bourboulis ,&nbsp;Djillali Annane ,&nbsp;Antonio Artigas ,&nbsp;Abdullah Tarik Aslan ,&nbsp;Gabriella Bottari ,&nbsp;Hjalmar R. Bouma ,&nbsp;Vladimir Černý ,&nbsp;Renata Curić Radivojević ,&nbsp;Ken Dewitte ,&nbsp;Daniela Filipescu ,&nbsp;Matthias Gründling ,&nbsp;Johanna Hästbacka ,&nbsp;Said Laribi ,&nbsp;Annmarie Lassen ,&nbsp;Konstantin Lebedinskii ,&nbsp;Adam Linder ,&nbsp;Jan Máca ,&nbsp;Manu L.N.G. Malbrain ,&nbsp;Gianpaola Monti ,&nbsp;Zuzanna Górska","doi":"10.1016/j.lanepe.2025.101570","DOIUrl":"10.1016/j.lanepe.2025.101570","url":null,"abstract":"<div><h3>Background</h3><div>Blood cultures (BCs) are key diagnostic elements for sepsis patients. Accurate preanalytical procedures are substantial, and results should be available as soon as possible to guide adequate antimicrobial treatment. This study aimed to evaluate BC collection practices and diagnostic capacity across European hospitals.</div></div><div><h3>Methods</h3><div>This cross-sectional survey investigated BC diagnostics in acute care hospitals across 37 European countries in the years 2021 and 2022. Analyses included BC guidelines, collection sites, number of BC sets in emergency departments (EDs), wards, and intensive care units (ICUs). We also examined transfer after collection, the use of on-site vs. external laboratories, opening hours, rapid testing capacity, and turn-around times of BCs processed in microbiology laboratories with different infrastructures.</div></div><div><h3>Findings</h3><div>Responses were collected from 907 hospitals in Europe. BC guidelines were available in 84·4% (741/878) of the hospitals. BCs were preferably collected by multiple-site sampling in EDs (62·7%, 461/735), in wards (64·0%, 513/802) and ICUs (68·5%, 518/756). One BC set was preferred in EDs in 38·4% (270/704), in wards in 40·5% (314/775), and ICUs in 34·9% (261/748). Two BC sets were preferred in EDs in 31·0% (218/704), in wards in 28·1% (218/775), and ICUs in 39·2% (293/748). 48·0% (402/838) of hospitals used on-site and 52·0% (436/838) external microbiology laboratories. Around-the-clock microbiological services were available in 10⋅0% (91/907), and rapid pathogen identification in 43·7% (396/907) of hospitals. Infrastructure with around-the-clock microbiological service and rapid testing was available in 7·4% (62/840) of hospitals, and probability of a final microbiological result within two days was highest in these hospitals compared to hospitals with limited microbiology service (for BC collected on wards: 19·6% vs. 52·7%, Odds Ratio 4·59 [95% CI 2·50–7·79], p &lt; 0·0001).</div></div><div><h3>Interpretation</h3><div>Despite the availability of BC guidelines in many hospitals, current recommendations for BC collection were often neglected. Rapid testing capacity was limited in most microbiological laboratories, and around-the-clock service for BCs was very rare. As delay in results may have a detrimental impact on patient outcomes, strategies to improve these processes are urgently needed.</div></div><div><h3>Funding</h3><div>The <span>European Sepsis Alliance</span> and a grant by Becton and Dickinson.</div></div>","PeriodicalId":53223,"journal":{"name":"Lancet Regional Health-Europe","volume":"62 ","pages":"Article 101570"},"PeriodicalIF":13.0,"publicationDate":"2025-12-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145798566","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Inequalities in availability of clinical trials for pediatric, adolescent, and young adult patients with cancer in Europe: results from the SIOPE OCEAN project","authors":"Kerstin K. Rauwolf ,&nbsp;Teresa de Rojas ,&nbsp;Miguel Martins ,&nbsp;Maria Otth ,&nbsp;Uta Dirksen ,&nbsp;Delphine Heenen ,&nbsp;Lejla Kameric ,&nbsp;Pamela Kearns ,&nbsp;Ruth Ladenstein ,&nbsp;Cormac Owens ,&nbsp;Caroline Queiroz ,&nbsp;Richard Sullivan ,&nbsp;Carmelo Rizzari ,&nbsp;Gilles Vassal ,&nbsp;European Society for Paediatric Oncology (SIOPE)","doi":"10.1016/j.lanepe.2025.101554","DOIUrl":"10.1016/j.lanepe.2025.101554","url":null,"abstract":"<div><div>There are pronounced inequalities in outcomes of children, adolescents, and young adults (AYA) with cancer across Europe. The OCEAN project aimed at describing the availability of clinical trials for this population, the inequalities between countries, and propose solutions to reduce these disparities. The <span><span>ClinicalTrials.gov</span><svg><path></path></svg></span> database was searched to identify all European cancer clinical interventional studies including patients &lt;18 years with a start date between 2010 and 2022. The study included 436 cancer-directed trials in 38 European countries. More than half were academic-sponsored (55%), and 49% included exclusively pediatric/AYA patients. Important differences in available trial numbers per country were identified, with more trials observed in Northern and Western Europe as compared to Eastern Europe. There is an urgent need to address differences in clinical trials availability both at European and national levels to advance equity and improve care, research and access to innovation for all pediatric/AYA patients with cancer in Europe.</div></div><div><h3>Funding</h3><div>This work has been performed as part of WP6 on inequalities in cancer research of the 4.UNCAN.eu Coordination and Support Action (#101069496) funded by the <span>European Union</span> and has been supported by <span>Zoé4Life</span> (MO).</div></div>","PeriodicalId":53223,"journal":{"name":"Lancet Regional Health-Europe","volume":"62 ","pages":"Article 101554"},"PeriodicalIF":13.0,"publicationDate":"2025-12-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145798567","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Real-world evidence in metabolic dysfunction-associated steatotic liver disease (MASLD): insights, challenges, and future directions","authors":"Huei-Tyng Huang,&nbsp;Michael Hewitt,&nbsp;Wenhao Li,&nbsp;William Alazawi","doi":"10.1016/j.lanepe.2025.101557","DOIUrl":"10.1016/j.lanepe.2025.101557","url":null,"abstract":"<div><div>Real-world evidence (RWE), derived from real-world data, offers key insights into metabolic dysfunction-associated steatotic liver disease (MASLD). RWE complements traditional randomised controlled trials by capturing large-scale, diverse patient populations and long-term outcomes. This review interrogates the role of RWE in understanding MASLD epidemiology, natural history, hepatic and extra-hepatic endpoints, including its co-morbid association with cardiovascular and chronic kidney disease, and its use in pharmacovigilance and precision medicine. RWE highlights large regional variations and is increasingly leveraged to advance drug development through target discovery and patient stratification. However, challenges such as data quality and confounding factors persist. In this review, key methodological gaps and future research priorities are identified and highlighted. The potential of artificial intelligence with multi-modal data linkage is considerable but requires rigorous methodologies and collaboration to fully realise the potential of RWE in MASLD research.</div></div>","PeriodicalId":53223,"journal":{"name":"Lancet Regional Health-Europe","volume":"62 ","pages":"Article 101557"},"PeriodicalIF":13.0,"publicationDate":"2025-12-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145798568","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Development and validation of the NEOS2 score for prediction of long-term outcomes and improvement after first-line immunotherapy in patients with anti-NMDAR encephalitis: an international cohort study","authors":"Juliette Brenner ,&nbsp;Anna E.M. Bastiaansen ,&nbsp;Mar Guasp ,&nbsp;Sergio Muñiz-Castrillo ,&nbsp;Takahiro Iizuka ,&nbsp;Marienke A.A.M. de Bruijn ,&nbsp;Amaia Muñoz-Lopetegi ,&nbsp;Eugenia Martínez-Hernández ,&nbsp;Géraldine Picard ,&nbsp;Alberto Vogrig ,&nbsp;Mathilde Millot ,&nbsp;Carsten Finke ,&nbsp;Christian Geis ,&nbsp;Jan Lewerenz ,&nbsp;Nico Melzer ,&nbsp;Harald Prüss ,&nbsp;Saskia Räuber ,&nbsp;Marius Ringelstein ,&nbsp;Kevin Rostàsy ,&nbsp;Kurt-Wolfram Sühs ,&nbsp;Maarten J. Titulaer","doi":"10.1016/j.lanepe.2025.101562","DOIUrl":"10.1016/j.lanepe.2025.101562","url":null,"abstract":"&lt;div&gt;&lt;h3&gt;Background&lt;/h3&gt;&lt;div&gt;Anti-N-methyl-&lt;span&gt;d&lt;/span&gt;-aspartate receptor (anti-NMDAR) encephalitis is a severe disease that primarily affects young people and can improve with adequate treatment. We aimed to refine the anti-NMDAR Encephalitis One-year functional Status (NEOS) score by developing NEOS2, an updated model using readily available data at the time of diagnosis. We assessed the predictive value of the NEOS2-score for (1) improvement following first-line treatment, (2) functional outcome at one-year follow-up, and (3) resumption of school or work within three years.&lt;/div&gt;&lt;/div&gt;&lt;div&gt;&lt;h3&gt;Methods&lt;/h3&gt;&lt;div&gt;In this international (France, Germany, Japan, the Netherlands and Spain) cohort study in patients with a definite anti-NMDAR encephalitis diagnosis (according to the clinical criteria plus antibody testing in CSF), we performed logistic regression analyses to develop and validate multivariable models to predict -based upon variables available at diagnosis- short (ΔmRS two weeks after first-line treatment), middle (modified Rankin Scale [mRS] at one year), and long-term (return to school or work within three years) outcomes. We included clinical variables and biomarkers available at diagnosis.&lt;/div&gt;&lt;/div&gt;&lt;div&gt;&lt;h3&gt;Findings&lt;/h3&gt;&lt;div&gt;We included 702 patients (mean age 23 years, 95%-CI 2–69; 79% female, 21% male) diagnosed between the discovery of the disease in 2007 and 2022. Most patients (96%; 672/702) had received first-line immunotherapy, and 38% (233/615) showed improvement within two weeks. One year after diagnosis, 80% (517/644) had a favourable functional outcome (mRS≤2). At three years, 73% (203/278) had resumed work/school. In multivariable analysis, higher age (odds ratio [OR] 0·35, 95%-CI 0·29–0·43, p &lt; 0·0001), treatment delay (OR 0·49, 95%-CI 0·41–0·58, p &lt; 0·0001), movement disorders (OR 0·32, 95%-CI 0·24–0·41, p &lt; 0·0001), ICU-requirement (OR 0·34, 95%-CI 0·26–0·44, p &lt; 0·0001) and increased CSF leucocyte count (OR 0·65, 95%-CI 0·60–0·71, p &lt; 0·0001) independently predicted poorer outcomes (NEOS2, accuracy AUC 80%, 95%-CI 75–86%). The same variables, excluding age, were relevant in predicting improvement following first-line immunotherapy (NEOS2-T AUC 81–84%, 95%-CI 77–86%). Return-to-work or -school served as a useful measure of longer-term outcomes, predicted with equal accuracy as one-year functional outcome (NEOS2-W AUC 80%, 95%-CI 75–85%). The NEOS2-score, applied as an ordinal measure, enabled nuanced predictions of outcome probabilities across the score spectrum, ranging from a high (80%; n = 20/25) likelihood of improving after first-line immunotherapy and achieving a good outcome (100%; n = 32/32) to a high risk of first-line treatment failure (97%; n = 77/79) and no return to school/work (94%; n = 15/16).&lt;/div&gt;&lt;/div&gt;&lt;div&gt;&lt;h3&gt;Interpretation&lt;/h3&gt;&lt;div&gt;The NEOS2-score, readily available at diagnosis and easy to apply, can identify patients with either a favourable or poor prognosis, and those who may ","PeriodicalId":53223,"journal":{"name":"Lancet Regional Health-Europe","volume":"62 ","pages":"Article 101562"},"PeriodicalIF":13.0,"publicationDate":"2025-12-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145749855","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Patient-reported, psychosocial and health economic outcomes in mild to moderate Friedreich's ataxia: baseline results of the PROFA study","authors":"Marcus Grobe-Einsler ,&nbsp;Stéphanie Borel ,&nbsp;Maresa Buchholz ,&nbsp;Sabrina Sayah ,&nbsp;Rania Hilab ,&nbsp;Lucie Pierron ,&nbsp;Audrey Iskandar ,&nbsp;Brittany Humphries ,&nbsp;Claire Ewenczyk ,&nbsp;Anna Heinzmann ,&nbsp;Mariana Atencio ,&nbsp;Katrin Feldmann ,&nbsp;Vivian Maas ,&nbsp;Jennifer Faber ,&nbsp;Sylvia Boesch ,&nbsp;Elisabetta Indelicato ,&nbsp;Kathrin Reetz ,&nbsp;Jörg B. Schulz ,&nbsp;Almut T. Bischoff ,&nbsp;Thomas Klopstock ,&nbsp;Bernhard Michalowsky","doi":"10.1016/j.lanepe.2025.101552","DOIUrl":"10.1016/j.lanepe.2025.101552","url":null,"abstract":"<div><h3>Background</h3><div>Friedreich ataxia (FA) is the most common autosomal recessive ataxia. Little attention has been paid to FA's impact on patient-reported, psychosocial, and health-economic outcomes. This study aimed to report these outcomes across FA's disability stages 1–5.</div></div><div><h3>Methods</h3><div>We assessed patients in Germany, France, and Austria as part of the PROFA study, a European multicenter observational study. The protocol included a study center visit followed by a remote mobile assessment capturing ataxia severity (SARA), daily living deficits (FARS-ADL), cognitive and affective impairments (CCAS), health-related quality of life (HRQoL: PROM-Ataxia short-form, EQ-5D-5L), mental well-being (WEMWBS), communication disabilities (COMATAX), and healthcare and informal care utilization. FARS disability stages were used to demonstrate outcomes with effect size measures (Eta-Squared, Cramér's V). Multivariate regression models evaluated associations between z-standardized outcomes and disability stages.</div></div><div><h3>Findings</h3><div>One hundred one patients (mean [SD]: age 35.0 [11.5]; GAA-repeat size 657 [299]; 50.5% women) were included. Activities of daily living, HRQoL, communication disabilities, and informal care utilization worsened significantly across disability stages with moderate to high effect sizes. Cognitive-affective impairments and mental well-being showed significant associations with small effect sizes. Twenty-three patients (33.3%) received formal care, while 40 (58.0%) received informal care (mean 12.2 h/week). Omaveloxolone was used by 33 patients (32.7%). Annual healthcare costs excluding Omaveloxolone were €13,620 (payer) and €32,679 (societal perspective, including informal care and productivity losses).</div></div><div><h3>Interpretation</h3><div>The results emphasize the multidimensional patient, societal, and economic burden of FA and the need for comprehensive care addressing physical, mental, and psychosocial health.</div></div><div><h3>Funding</h3><div><span>European Joint Programme on Rare Diseases</span> (EJP RD).</div></div>","PeriodicalId":53223,"journal":{"name":"Lancet Regional Health-Europe","volume":"61 ","pages":"Article 101552"},"PeriodicalIF":13.0,"publicationDate":"2025-12-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145747382","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}