方法与临床研究雷达（2026-09-19）
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1. Social cognition as an under-used behavioural endpoint in neurodevelopmental psychopharmacology: A conditional framework for integrating theory of mind into clinical trial design.
主题：临床试验方法与统计实践
相关性分数：6
期刊：European journal of pharmacology
公开日期：2026-08-20（电子公开）
期刊卷期日期：2026-Oct-15
内容状态：待评估
为什么值得看：包含可供初筛的摘要；已识别电子公开日期；通过方法学信号门槛；仍需阅读全文评价适用性与证据质量。
Social cognition, encompassing theory of mind (ToM), facial affect recognition, and mentalising under social load, is impaired across several neurodevelopmental disorders (NDDs), including autism spectrum disorder (ASD), attention-deficit/hyperactivity disorder (ADHD), developmental language disorder (DLD), and intellectual disability (ID). These impairments are plausibly downstream of neurochemical processes, particularly serotonergic, dopaminergic, neuropeptidergic, and excitatory-inhibitory modulation of prefrontal-temporal-limbic circuits, although this pathway is indirect and moderated by age, language, cognitive ability, and comorbidity. Despite this, performance-based social cognition is rarely positioned as a primary or co-primary endpoint in NDD pharmacotherapy trials. The predominant standard, the Aberrant Behavior Checklist Irritability subscale and its caregiver-rated relatives, indexes reactive behavioural output that may be construct-distant from the mechanisms through which several contemporary agents are hypothesised to act. This narrative review advances a conditional thesis: endpoint-mechanism misalignment is one plausible contributor to the uninterpretability of certain null trials, alongside insufficient power, sample heterogeneity, unverified target engagement, dosing limitations, high placebo response, and genuine inefficacy. We synthesise condition-specific social-cognitive profiles with graded evidence, the neurobiological substrates across mechanism classes, and a three-tier framework positioning social cognition as a co-primary endpoint for mechanism-matched agents and as a prespecified secondary or moderator variable otherwise. We set out the measurement and regulatory preconditions fit-for-purpose validation, adequate test-retest reliability, practice-effect control, and defined meaningful change that must precede such repositioning. We do not claim ToM should replace existing endpoints or that it suits all NDD trials.
2. Application of competing risks models in cardiovascular mortality research: findings from the Tehran lipid and glucose study.
主题：生存与复杂事件结局方法
相关性分数：6
期刊：Journal of diabetes and metabolic disorders
公开日期：2026-07-21（电子公开）
期刊卷期日期：2026-Dec
内容状态：待评估
为什么值得看：包含可供初筛的摘要；已识别电子公开日期；通过方法学信号门槛；仍需阅读全文评价适用性与证据质量。
PURPOSE: Cardiovascular diseases (CVD) are a leading cause of mortality in Iran and globally. This study aimed to provide more accurate estimates of associations between risk factors and CVD mortality by applying competing risk models within the Tehran Lipid and Glucose Study cohort.
METHODS: In this prospective analysis, 7,529 individuals aged ≥ 30 years without prevalent CVD were followed for a median of 19.87 years. The primary outcome was CVD mortality (n = 311), with non-CVD death as the competing event (n = 592). Analyses were stratified by sex and age (< 65 vs. ≥65 years). Cause-specific and Fine-Gray models estimated hazard ratios for diabetes, hypertension, hypercholesterolemia, smoking, and body mass index.
RESULTS: Diabetes and hypertension were the strongest predictors of CVD mortality across most subgroups. In the Fine-Gray model, diabetes showed the greatest impact in women < 65 years (HR: 4.83, p < 0.001), while hypertension showed the strongest association in women ≥ 65 years (HR: 3.32, p < 0.001). Hypercholesterolemia was associated with increased CVD mortality exclusively in women < 65 years (HR: 1.79, p = 0.02). Body mass index showed no significant association.
CONCLUSION: Diabetes and hypertension are the predominant risk factors for CVD mortality in the presence of competing risks, with effect magnitudes varying by sex and age. Applying competing risk models is essential for accurate risk estimation and targeted prevention in populations with high competing mortality burden.
3. A pragmatic randomized trial to evaluate the vaccine effectiveness of bivalent RSV prefusion F vaccine for preventing RSV hospitalizations in adults (DAN-RSV): Trial design update.
主题：临床试验方法与统计实践
相关性分数：6
期刊：American heart journal
公开日期：2026-06-01（电子公开）
期刊卷期日期：2026-Oct
内容状态：待评估
为什么值得看：包含可供初筛的摘要；已识别电子公开日期；通过方法学信号门槛；仍需阅读全文评价适用性与证据质量。
BACKGROUND: Respiratory syncytial virus (RSV) is a major cause of respiratory morbidity in adults, particularly among older individuals and those with comorbidities. The DAN-RSV trial was initiated to evaluate bivalent RSV prefusion F (RSVpreF) vaccine effectiveness in preventing RSV-related hospitalizations.
METHODS: DAN-RSV is a large-scale, pragmatic, randomized clinical trial that enrolled participants during the 2024/2025 (Danish adults aged ≥60 years) and 2025/2026 (adults aged ≥18 years in Denmark and Galicia, Spain) Northern hemisphere winter seasons. In Denmark, nationwide registries and the national electronic messaging system were used to identify and recruit eligible citizens; individuals could provide electronic informed consent remotely or in-person. In Galicia, participants were recruited via text message invitations and consented on-site.
RESULTS: During the initial 2024/2025 season, 131,379 Danish adults aged ≥60 years were enrolled. Following lower-than-expected event accrual and expansion of the EU indication to adults 18 years and older, the trial was extended to continue enrollment of adults aged ≥18 years across Denmark and Galicia, Spain during the 2025/2026 RSV season. Key protocol updates include expansion of the eligibility criteria to adults aged ≥18 years, inclusion of an additional study site within the integrated public healthcare infrastructure of Galicia, Spain, and an increase in the planned sample size to up to approximately 690,000 participants across both seasons. The randomization strategy (1:1 to RSVpreF vaccine or no vaccine), primary endpoint (RSV-related respiratory tract disease hospitalization), and statistical framework, including intention-to-treat analyses and hierarchical testing, remain unchanged.
CONCLUSION: The extension of the DAN-RSV trial is expected to improve statistical precision, enhance generalizability, and strengthen the robustness of the effect estimates. The updated design will provide reliable randomized evidence on bivalent RSVpreF vaccine effectiveness to inform clinical and public health decision-making.
4. The CLTI Paradigm Shift: Implications for Clinical Trial Design and Endpoints in Endovascular Management of Infrapopliteal Disease.
主题：临床试验方法与统计实践
相关性分数：6
期刊：Journal of endovascular therapy : an official journal of the International Society of Endovascular Specialists
公开日期：2025-08-28（电子公开）
期刊卷期日期：2026-Oct
内容状态：待评估
为什么值得看：包含可供初筛的摘要；已识别电子公开日期；通过方法学信号门槛；仍需阅读全文评价适用性与证据质量。
The evolution from critical limb ischemia to chronic limb-threatening ischemia (CLTI) necessitates a reevaluation of clinical trial endpoints. Traditional measures like hemodynamic indices and lesion patency inadequately reflect CLTI complexity or patient-centered outcomes. This article explores the limitations of conventional endpoints and proposes a modified definition of clinically-driven target lesion (TL) revascularization (CD-TLR) that addresses inflow, TL, and outflow disease. By incorporating a more comprehensive assessment of vascular health and prioritizing patient-relevant outcomes, this modified CD-TLR definition may better reflect modern CLTI management strategies and improve clinical relevance of trial results in this challenging patient population.Clinical ImpactThe evolution from CLI to CLTI requires reassessing how we measure treatment success in clinical trials. This review identifies why traditional endpoints like hemodynamic indices, lesion patency, and CD-TLR may not adequately inform clinical decision-making. The importance of measuring outcomes that directly impact patient quality of life will help clinicians better evaluate and apply clinical trial results. An emphasis on limb salvage, wound healing, and functional outcomes, rather than surrogate endpoints alone, will lead to more informed treatment decisions and improved CLTI management strategies.
5. Cost-effectiveness analysis of pneumococcal vaccination of at-risk and high-risk adults aged 18-64 years in Switzerland using PCV21.
主题：因果推断、RWE 与卫生经济学
相关性分数：5
期刊：Journal of medical economics
公开日期：2026-09-10（电子公开）
期刊卷期日期：2026-Dec
内容状态：待评估
为什么值得看：包含可供初筛的摘要；已识别电子公开日期；通过方法学信号门槛；仍需阅读全文评价适用性与证据质量。
BACKGROUND: Pneumococcal disease (PD) is more common and severe among adults with underlying medical conditions. Pneumococcal vaccination is recommended but not reimbursed in Switzerland for adults 18-64 years with these risk factors.
OBJECTIVE: We estimated the cost-effectiveness of reimbursed vaccination for high-risk (immunocompromised) and at-risk (chronic medical condition) adults 18-64 years in Switzerland using 21-valent pneumococcal conjugate vaccine (PCV21).
METHODS: We updated a published state-transition Markov model to estimate lifetime health and economic outcomes for multiple adult cohorts, following individuals until death or age 100 years, from the Swiss payer perspective. Swiss demographic and health economic data were used where available and supplemented with proxy epidemiologic estimates from comparable international sources when Swiss-specific data were unavailable. Vaccination of 60% of the target population with PCV21 was compared with no pneumococcal vaccination. One-way and probabilistic sensitivity analyses assessed parameter uncertainty and model robustness.
RESULTS: Implementing PCV21 for at-risk and high-risk adults 18-64 years in Switzerland was predicted to avert 518 invasive pneumococcal disease cases, 12 post-meningitis sequelae cases, and 2,578 inpatient non-bacteremic pneumococcal pneumonia (NBPP) cases and 224 deaths. The CHF 21.4 M decrease in direct PD treatment costs partially offset the CHF 39.0 M vaccination costs, resulting in an incremental cost-effectiveness ratio (ICER) of 7,665 CHF/quality-adjusted life year (QALY) gained. The ICER was 1,750 CHF/QALY in a scenario analysis in which the proportions of adults with risk conditions increased with age. One-way sensitivity analysis found results were most sensitive to vaccine efficacy against NBPP in the at-risk group and discount rate. In probabilistic sensitivity analyses, the probability that PCV21 was cost-effective reached 100% at willingness-to-pay thresholds of approximately CHF 18,000 or higher.
CONCLUSIONS: Vaccination of at-risk and high-risk adults 18-64 years of age in Switzerland using PCV21 is likely to be cost-effective.
6. Cost-effectiveness of finotonlimab plus bevacizumab versus sorafenib as first-line therapy in unresectable hepatocellular carcinoma in China.
主题：因果推断、RWE 与卫生经济学
相关性分数：5
期刊：Human vaccines & immunotherapeutics
公开日期：2026-07-27（电子公开）
期刊卷期日期：2026-Dec-31
内容状态：待评估
为什么值得看：包含可供初筛的摘要；已识别电子公开日期；通过方法学信号门槛；仍需阅读全文评价适用性与证据质量。
Hepatocellular carcinoma (HCC) imposes a substantial health burden in China. Finotonlimab plus bevacizumab recently prolonged progression-free survival (PFS) and overall survival (OS) vs. sorafenib, but its economic value remains unknown. Here we evaluated the cost-effectiveness of finotonlimab plus bevacizumab vs. sorafenib from the Chinese healthcare system perspective. Parametric survival models were fitted to extrapolate PFS and OS. Total costs, life-years (LYs), quality-adjusted life-years (QALYs), incremental cost-effectiveness ratios (ICERs), incremental net monetary benefit (INMB), and incremental net health benefit (INHB) were estimated at a willingness-to-pay (WTP) threshold of $27,906 per QALY. Uncertainty was evaluated by one-way and two-way sensitivity analyses, probabilistic sensitivity analysis (PSA), subgroup analyses, scenario analyses, and price simulations. In the base-case analysis, sorafenib yielded 1.74 LYs and 1.25 QALYs at a total cost of $10,303.10, whereas finotonlimab plus bevacizumab yielded 3.01 LYs and 2.18 QALYs at a total cost of $58,595.49. Compared with sorafenib, the combination increased costs by $48,292.39 and generated gains of 1.27 LYs and 0.93 QALYs, resulting in ICERs of $38,203.46 per LY and $51,899.31 per QALY. INMB (-$22,325.82) and INHB (-0.80 QALYs) were negative. Sensitivity analyses identified PFS utility and bevacizumab cost as key drivers, but all ICERs remained above the WTP threshold. In PSA, the mean ICER was $50561.81 per QALY, and the probability of cost-effectiveness was 0% at the prespecified threshold. Based on the assumptions and inputs used in the present model, finotonlimab plus bevacizumab was unlikely to be cost-effective compared with sorafenib at the prespecified WTP in China.
7. Wound-Only versus Weight-Based Rabies Immunoglobulin in Post-Exposure Prophylaxis: A Quasi-Randomized Asessor Blind Non-Inferiority Trial.
主题：临床试验方法与统计实践
相关性分数：4
期刊：The American journal of tropical medicine and hygiene
公开日期：2026-09-17（电子公开）
期刊卷期日期：2026-Sep-17
内容状态：待评估
为什么值得看：包含可供初筛的摘要；已识别电子公开日期；通过方法学信号门槛；仍需阅读全文评价适用性与证据质量。
Rabies is a fatal but preventable zoonotic disease that causes more than 20,000 deaths annually in India. Post-exposure prophylaxis (PEP) involves rabies vaccination and passive immunization with rabies immunoglobulin (RIG). Traditional RIG dosing is based on patient weight, whereas wound-only administration targets local viral neutralization at the exposure site. In the present study, antibody responses were compared after a three-dose anti-rabies vaccination in patients receiving either wound-only or weight-based RIG infiltration as part of PEP to assess the noninferiority of the wound-only approach. Patients with category III rabies exposure were enrolled in this noninferiority clinical trial, all of whom received a standard three-dose rabies vaccination. Rabies immunoglobulin was administered either according to body weight or infiltrated only around the wound. Serum rabies-neutralizing antibody titers were measured at predefined intervals. The primary outcome was the proportion of patients exhibiting protective antibody titers; secondary outcomes included RIG usage volume, adverse events, and feasibility in resource-limited settings. Both groups achieved adequate seroconversion with protective antibody titers. Wound-only RIG infiltration produced protective antibody responses comparable to weight-based RIG dosing while substantially reducing RIG volume used and could improve feasibility. Larger studies, including formal cost-effectiveness analyses, are needed to confirm the clinical and programmatic implications.
8. REmotely monitored, Mobile health-supported Multidomain Rehabilitation with High Intensity Interval Training for COVID-19 (REMM-HIIT-CoV): Trial Design and Rationale.
主题：临床试验方法与统计实践
相关性分数：4
期刊：Physical therapy
公开日期：2026-09-17（电子公开）
期刊卷期日期：2026-Sep-17
内容状态：待评估
为什么值得看：包含可供初筛的摘要；已识别电子公开日期；通过方法学信号门槛；仍需阅读全文评价适用性与证据质量。
IMPORTANCE: Individuals hospitalized with COVID-19 are at increased risk for prolonged physical, cognitive, and psychosocial impairments. Timely post-acute rehabilitation may reduce these sequelae and accelerate recovery.
OBJECTIVE: The objective is to determine whether a REmotely monitored, Mobile health-supported Multidomain Rehabilitation Program incorporating High-intensity Interval Training (REMM-HIIT-CoV) after COVID-19 hospitalization improves functional exercise capacity.
DESIGN: This study is a multicenter, single-blinded, attention controlled, prospective randomized controlled trial.
SETTING: Five academic medical centers will enroll participants for remotely delivered and monitored rehabilitation.
PARTICIPANTS: The study will enroll 140 community-dwelling adults discharged within 60 days of COVID-19 hospitalization.
INTERVENTION: Participants are randomized to either (1) a 12-week, individually tailored, structured and progressive multidomain physical rehabilitation intervention-targeting deficits in balance, mobility, strength, and endurance-delivered via videoconference and remotely monitored using activity trackers and a mobile health application; or (2) an exercise education control group without individualized exercise prescription.
MAIN OUTCOMES AND MEASURES: The primary outcome is functional exercise capacity, measured by 6-minute walk distance at 3 months post-randomization. Secondary outcomes include cardiorespiratory fitness (VO2peak), physical function (Short Physical Performance Battery, 30-second Sit-to-Stand, Grip Strength), frailty (Fried Frailty Index), and cognitive function (Montreal Cognitive Assessment), self-reported quality of life (EQ-5D-5L), functional status (Duke Activity Status Index, Lawton Instrumental Activities of Daily Living), mental health (Hospital Anxiety and Depression Scale), and health-related social needs (Accountable Health Communities Health-related Social Needs Screening Tool). Exploratory outcomes include blood-derived biomarkers of inflammation and mitochondrial function, and ultrasound measures of lower extremity skeletal muscle structure.
CONCLUSIONS: REMM-HIIT-CoV is the first randomized trial to evaluate a remotely delivered, structured, and tailored multidomain rehabilitation program incorporating HIIT in post-hospitalized COVID-19 survivors.
RELEVANCE: Findings may inform evidence-based guidelines and policy-relevant models for home-based remote rehabilitation after severe respiratory infections.
9. Adaptive design strategies for ethical and efficient clinical trials using simulated annealing.
主题：临床试验方法与统计实践
相关性分数：4
期刊：Statistical methods in medical research
公开日期：2026-09-14（电子公开）
期刊卷期日期：2026-Sep-14
内容状态：待评估
为什么值得看：包含可供初筛的摘要；已识别电子公开日期；通过方法学信号门槛；仍需阅读全文评价适用性与证据质量。
We introduce a flexible adaptive design strategy for treatment allocation in clinical trials based on a randomized simulated annealing algorithm. The proposed approach provides a unified and modular framework for implementing a broad range of adaptive randomization objectives, including covariate-adaptive (CA), response-adaptive (RA), covariate-adjusted response-adaptive (CARA), and hybrid RA+CA and CARA+CA designs. The procedure accommodates multiple treatments, different outcome types, and any baseline covariate information available. Its implementation is compatible with both parametric and nonparametric predictive models; in this work, we use Bayesian additive regression trees to capture complex covariate-response relationships without committing to a low-dimensional parametric specification. The allocation rule combines a simulated-annealing-based recommendation with a fallback randomization step used to ensure positive assignment probabilities. In a finite stratified setting, we establish strong consistency of the resulting stratified sample mean estimators. Extensive simulation studies with homogeneous and heterogeneous treatment effects show that our proposal achieves a favorable trade-off between ethical allocation and statistical efficiency. A redesign of the ACTG 175 clinical trial further illustrates the practical advantages and flexibility of the proposed framework.
10. Sample size calculation for the sequential multiple assignment randomized trial (SMART) design with a skewed outcome: Application to the SMART+ study.
主题：临床试验方法与统计实践
相关性分数：4
期刊：Statistical methods in medical research
公开日期：2026-09-12（电子公开）
期刊卷期日期：2026-Sep-12
内容状态：待评估
为什么值得看：包含可供初筛的摘要；已识别电子公开日期；通过方法学信号门槛；仍需阅读全文评价适用性与证据质量。
With the rapid emergence of personalized healthcare, adaptive interventions have gained significant traction and relevance. Contemporary research has introduced a sophisticated trial design known as the sequential multiple assignment randomized trial (SMART) to advance the development of effective adaptive interventions. A critical element of the SMART design is the determination of the sample size. The existing literature provides sample size calculation formulas for SMART designs, which encompass a variety of approaches and types of outcome data. However, these formulas have primarily been developed under the assumption of normality for the outcome data. In practice, the fields where SMART is employed exhibit a significant prevalence of non-normality in the outcomes of interest. This paper delves into a specific scenario where the outcome demonstrates skewed behavior. We develop precision-based and power-based formulas for skewed outcomes, considering the requirements of both pilot and full-scale SMARTs. We present the operating characteristics of the formulas and perform extensive simulations under various design specifications to validate their usefulness. To demonstrate practical utility, we apply our formulas to the SMART+ study, a digital intervention trial that includes a skewed outcome among its outcomes of interest, highlighting relevance in real-world planning.
11. Clinical and economic impact of bivalent respiratory syncytial virus prefusion F (RSVpreF) vaccination in older adults: a cost-effectiveness analysis for Chile.
主题：因果推断、RWE 与卫生经济学
相关性分数：4
期刊：Expert review of vaccines
公开日期：2026-09-12（电子公开）
期刊卷期日期：2026-Dec
内容状态：待评估
为什么值得看：包含可供初筛的摘要；已识别电子公开日期；通过方法学信号门槛；仍需阅读全文评价适用性与证据质量。
BACKGROUND: Respiratory syncytial virus (RSV) is a major cause of lower respiratory tract disease in older adults in Chile. Chile’s National Immunization Technical Advisory Group (NITAG) recommended RSV vaccination for adults aged ≥60 years with risk factors for severe illness. This study estimated the cost-effectiveness of bivalent RSVpreF vaccination among at-risk older adults in Chile.
RESEARCH DESIGN AND METHODS: A population-based Markov model projected RSV-related outcomes under no vaccination and RSVpreF vaccination over the cohort lifetime. Cost-effectiveness was assessed from healthcare and societal perspectives in 2025 Chilean Pesos (CLP) and US Dollars (US$), with costs and outcomes discounted at 3% annually. Sensitivity analyses tested the model robustness.
RESULTS: RSVpreF vaccination was projected to prevent 14,956 hospitalizations, 50,419 emergency department visits, and 124,849 physician office visits; avert CLP76 billion (US$82 million) in direct medical costs and CLP305 billion (US$328 million) in indirect costs; and generate 7,079 quality-adjusted life-years (QALYs). At a threshold of CLP15,977,348 (US$17,180) per QALY gained, the maximum cost-effective vaccine price was CLP100,480 (US$108) and CLP265,261 (US$285) from the healthcare and societal perspectives respectively.
CONCLUSIONS: RSVpreF vaccination would reduce RSV burden and likely be cost-effective for older adults in Chile.
12. A multi-center, open-labelled, randomized controlled extended phase III non-inferiority clinical trial to evaluate the immunogenicity and tolerability of poliomyelitis vaccine (Vero cells), inactivated, Sabin strains administered with or without routine infant vaccines.
主题：临床试验方法与统计实践
相关性分数：4
期刊：Vaccine
公开日期：2026-08-27（电子公开）
期刊卷期日期：2026-Oct-03
内容状态：待评估
为什么值得看：包含可供初筛的摘要；已识别电子公开日期；通过方法学信号门槛；仍需阅读全文评价适用性与证据质量。
BACKGROUND: Oral poliovirus vaccine (OPV) has been reported to cause vaccine-derived poliovirus and vaccine-associated paralytic poliomyelitis, and the limited global supply of conventional IPV has led many countries to rely on fractional-dose IPV regimens alongside OPV. The current study aims to assess the sIPV safety and immunogenicity when given concurrently with or in a staggered manner with routine immunization.
METHODS: A multi-country, multi-center, open-label, randomized controlled, extended phase III non-inferiority clinical trial was conducted with 1442 healthy infants aged 6-8 weeks from Bangladesh and Pakistan enrolled and randomized into four groups, i.e., co-administration group 1 (group C1), co-administration group 2 (group C2), staggered administration group 1 (group S1) and staggered administration group 2 (group S2). Antibody levels were determined using the collected sera for immunogenicity evaluation. The difference in seroconversion rates between the coadministration group and the staggered administration group is compared using the Cochran-Mantel-Haenszel χ2 (CMH-χ2) test, stratified by study site. Non-inferiority is concluded if the lower bound of the 95% confidence interval (CI) for the rate difference (coadministration group minus staggered administration group) is greater than -10%. The trial was registered prior to patient enrollment at clinicaltrials.gov (NCT05850364), and the protocol and statistical analysis plan are available at https://clinicaltrials.gov/study/NCT05850364. The trial is closed to new participants.
FINDINGS: The post-vaccination seroconversion rates for PV I were 90.3% (306/339) in group C1 and 87.0% (261/300) in group S1, for PV II, 91.7% (311/339) in group C1 and 91.3% (274/300) in group S1 and for PV III, 86.4% (293/339) in group C1 and 92.3% (277/300) in group S1. Among adverse reactions (ARs) reported within 7 days of vaccination, the incidence was similarly high in both the co-administration and staggered vaccination groups (92.8% vs. 95.5%).
INTERPRETATION: Our results demonstrated favorable safety and immunogenicity of co-administration of sIPV with other routine infant vaccines according to a 3-dose primary immunization schedule.
13. Brain-computer interface clinical trial design considerations and clinical outcome assessments in pivotal studies: a summary of the 11th BCI society meeting 2025 workshop.
主题：临床试验方法与统计实践
相关性分数：4
期刊：Journal of neural engineering
公开日期：2026-09-11（电子公开）
期刊卷期日期：2026-Sep-11
内容状态：待评估
为什么值得看：包含可供初筛的摘要；已识别电子公开日期；通过方法学信号门槛；仍需阅读全文评价适用性与证据质量。
Objective.The Brain Computer Interface (BCI) Society Meeting 2025 held a workshop in collaboration with the Implantable BCI Collaborative Community (iBCI-CC) to discuss the selection, development and validation of clinical trial outcome assessments (COAs) for pivotal studies of BCIs. The iBCI-CC Clinical Study Endpoints Workgroup aims to build consensus on a COA framework that can meet the demands of emerging iBCI trials.Approach.The workshop brought together diverse stakeholders from the iBCI-CC community and beyond, including industry, academic and government institutions to engage in pre-competitive collaborative discussion.Main results.Through presentations, panel discussions and breakout groups, workshop participants highlighted meaningful aspects of health (MAHs) relevant to iBCI users, clarified the need to define corresponding concepts of interest (COIs) and to identify or adapt COAs capable of capturing both functional impact and real-world use. Key challenges highlighted during the workshop are patient heterogeneity, the lack of widely validated and fit-for-purpose COAs, and the need to capture meaningful outcomes in home and daily-life environments. Lessons drawn from trials such as ADAPT-PD underscore the value of capturing device performance in home and daily-life settings, highlighting the need for context-sensitive and flexible metrics that reflect patient priorities.Significance.This workshop lays a foundation for the iBCI-CC Clinical Study Endpoints Workgroup to establish a transparent process for identifying MAHs and COIs that are suitable for specific iBCI technologies, patient-informed, and conducive to regulatory approval and reimbursement. By combining rigorous, quantitative assessment with patient-informed goals, iBCI clinical trials can advance toward safe, effective, and accessible neurotechnologies that enhance how a person with a severe motor impairment feels, functions and survives.
14. Transitions in depressive symptom states among adults with subthreshold depression: Associations with lifetime trauma exposure and combined lifestyle in a multistate Markov study.
主题：生存与复杂事件结局方法
相关性分数：4
期刊：Journal of affective disorders
公开日期：2026-08-04（电子公开）
期刊卷期日期：2026-Dec-01
内容状态：待评估
为什么值得看：包含可供初筛的摘要；已识别电子公开日期；通过方法学信号门槛；仍需阅读全文评价适用性与证据质量。
BACKGROUND: Subthreshold depression is common, but whether lifetime trauma exposure and combined lifestyle are differentially associated with symptom transitions remains unclear.
METHODS: We analyzed a prospective community cohort in Shenzhen, China, of adults aged 18-65 years with a Patient Health Questionnaire-9 (PHQ-9) score ≥ 5 and no depressive disorder at baseline. Depressive symptom states (Normal, Mild, and Moderate/Severe) were assessed repeatedly over 36 months. Continuous-time multi-state Markov models estimated transition intensities, probabilities, and hazard ratios (HRs) for lifetime trauma exposure and a five-component combined lifestyle score, adjusting for demographic, socioeconomic, and health-related covariates.
RESULTS: Among 2361 participants (mean age, 37.30 years; 61.9% women), recovery transitions were more frequent than worsening transitions. The monthly Mild-to-Normal intensity was 0.131 (95% CI, 0.100-0.171), 2.47 times the Mild-to-Moderate/Severe intensity; Moderate/Severe-to-Mild was highest (0.205; 95% CI, 0.158-0.272). Lifetime trauma exposure was associated with lower Mild-to-Normal (HR, 0.54; 95% CI, 0.42-0.69) and Moderate/Severe-to-Mild rates (HR, 0.52; 95% CI, 0.41-0.65). Unfavorable lifestyle was associated with a lower Mild-to-Normal rate (HR, 0.70; 95% CI, 0.53-0.92) and a higher Mild-to-Moderate/Severe rate (HR, 1.60; 95% CI, 1.13-2.25). Those with both exposures had the lowest Mild-to-Normal rate (HR, 0.39; 95% CI, 0.26-0.59) and a higher Mild-to-Moderate/Severe rate (HR, 1.69; 95% CI, 1.05-2.73).
CONCLUSIONS: Lifetime trauma exposure was associated with lower recovery rates, whereas an unfavorable lifestyle was associated with lower recovery and higher worsening rates. These associations may inform trauma-informed assessment and lifestyle support for adults with subthreshold depression.
15. Benefit of Linked-Color Imaging in Artificial Intelligence-Assisted Diagnosis of Early Gastric Cancer: A Pilot Study With Propensity Score Adjustment.
主题：因果推断、RWE 与卫生经济学
相关性分数：4
期刊：DEN open
公开日期：2026-07-28（电子公开）
期刊卷期日期：2027-Apr
内容状态：待评估
为什么值得看：包含可供初筛的摘要；已识别电子公开日期；通过方法学信号门槛；仍需阅读全文评价适用性与证据质量。
BACKGROUND AND AIMS: Artificial intelligence (AI)-assisted endoscopy represents a promising approach for lesion detection, yet frequent false-positive detections impair clinical utility by disrupting examinations and diminishing physician confidence. Linked-color imaging (LCI), an image-enhanced endoscopy technique that amplifies mucosal and vascular contrast, may address this limitation. This investigation evaluated whether LCI reduces false-positive AI detections compared with white-light imaging (WLI).
METHODS: This retrospective study analyzed consecutive AI-assisted upper endoscopies performed between March 2024 and June 2025. WLI and LCI were performed sequentially within the same endoscopic session in each patient. False-positive AI detections were compared between modalities using two computer-aided detection (CAD) versions. Propensity score adjustment was used as a sensitivity analysis for baseline differences between CAD Versions I and II.
RESULTS: Of 66 initially screened cases, 63 remained after excluding patients with prior gastric surgery. LCI reduced false-positive AI detections compared with WLI (median 2 vs. 5; p < 0.001). In CAD version-stratified sensitivity analyses, LCI reduced false-positive AI detections in both Version I (5 to 2; p = 0.01) and Version II (2 to 0; p = 0.03). This reduction remained consistent across atrophic grades. Both imaging modalities identified all gastric lesions, achieving 100% detection sensitivity.
CONCLUSIONS: LCI assessment performed after WLI observation yielded fewer false-positive CAD-EYE detections while maintaining lesion detection sensitivity. However, because the observation sequence was fixed, these findings should be interpreted cautiously and require confirmation in prospective or counterbalanced studies.
UNLABELLED: Trial Registration: N/A (retrospective study).
16. Transcutaneous auricular vagus nerve stimulation combined with ciprofol for sedation in patients undergoing same-session bidirectional endoscopy: a randomized, double-blind, placebo-controlled, three-arm non-inferiority trial protocol.
主题：临床试验方法与统计实践
相关性分数：4
期刊：Annals of medicine
公开日期：2026-07-01（电子公开）
期刊卷期日期：2026-Dec
内容状态：待评估
为什么值得看：包含可供初筛的摘要；已识别电子公开日期；通过方法学信号门槛；仍需阅读全文评价适用性与证据质量。
BACKGROUND: Transcutaneous auricular vagus nerve stimulation (taVNS) provides targeted modulation of the autonomic nervous system and descending pain pathways, exerting analgesic potential. However, high-quality evidence remains insufficient to determine whether taVNS can effectively replace opioids in sedation regimens for same-session bidirectional endoscopy while maintaining the quality of early postoperative recovery.
DISCUSSION: This study is a single-center, prospective, randomized, double-blind, placebo-controlled, three-arm non-inferiority trial. A total of 181 patients scheduled for painless same-session bidirectional endoscopy will be enrolled and randomly assigned using dynamic block randomization to one of three groups: Group S (sufentanil 0.1 µg/kg plus sham taVNS), Group T (normal saline plus active taVNS), and Group P (normal saline plus sham taVNS). All participants will receive ciprofol for sedation induction and maintenance. The primary outcome will be the quality of recovery at 24 h postoperatively, assessed using the 15-item Quality of Recovery scale (QoR-15), with a predefined non-inferiority margin (δ) of 6 points, which corresponds to the minimal clinically important difference of the QoR-15 scale. Secondary outcomes will include perioperative adverse events (pre-, intra-, and postoperative, including taVNS-related events), QoR-15 score at 1 h postoperatively, procedural and recovery efficiency indices, sedative dosage, and patient and endoscopist satisfaction scores. Blinding effectiveness will be assessed in all participants, and statistical analyses will follow the modified intention-to-treat principle.
CONCLUSION: This study protocol will rigorously assess the effectiveness and safety of taVNS as an alternative to opioid analgesics for sedation during same-session bidirectional endoscopy using, to our knowledge, the first three-arm design.Trial registration: Chinese Clinical Trial Registry (ChiCTR2600117962).
This study will provide the first systematic evaluation of the feasibility and efficacy of transcutaneous auricular vagus nerve stimulation (taVNS) as an opioid-sparing alternative to sufentanil for ciprofol-based sedation during same-session bidirectional endoscopy.A three-arm randomized design incorporating a prespecified trial-sensitivity analysis will be used to rigorously validate the effectiveness of the standard regimen, enhancing the interpretability and credibility of the non-inferiority conclusion.An “active placebo” taVNS stimulation model combined with an improved standardized questionnaire for blinding assessment will be employed to address the well-recognized limitations of inadequate blinding in taVNS research.
17. Effects of Intranasal Dexmedetomidine on the Night Before Surgery on Postoperative Delirium in Elderly Patients Undergoing Total Knee/Hip Arthroplasty. A Parallel-Arm, Randomized Controlled, Non-Inferiority Trial.
主题：临床试验方法与统计实践
相关性分数：3
期刊：Drug design, development and therapy
公开日期：2026-09-13（电子公开）
期刊卷期日期：2026
内容状态：待评估
为什么值得看：包含可供初筛的摘要；已识别电子公开日期；通过方法学信号门槛；仍需阅读全文评价适用性与证据质量。
IMPORTANCE: It is crucial to identify preoperative treatment methods that can safely and effectively reduce the incidence of postoperative delirium.
OBJECTIVE: To evaluate whether preoperative intranasal dexmedetomidine administration for preventing postoperative delirium is non-inferior to intravenous dexmedetomidine infusion on the operative day before induction for elderly patients undergoing elective knee/hip arthroplasty.
DESIGN: This randomized clinical trial was performed at a tertiary hospital in China between January 10, 2025 to August 30, 2025. The follow-up for the last subject was completed on September 6, 2025.
SETTING: This was a single center trial.
PARTICIPANTS: After qualification screening, 535 patients scheduled for unilateral total knee arthroplasty and total hip arthroplasty under general anesthesia were invited to participate in the study. Ultimately, 316 patients were randomly assigned to the experimental group and the control group.
INTERVENTIONS: Patients were randomized to receive intranasal dexmedetomidine (the night before surgery)+intravenous normal saline (before induction) or intranasal normal saline (the night before surgery)+intravenous dexmedetomidine (before induction).
MAIN OUTCOMES AND MEASURES: The primary outcome was incidence of postoperative delirium within 3 days after surgery assessed by the Confusion Assessment Method.
RESULTS: In the intention-to-treat analysis, the incidence of postoperative delirium within 3 days was 9.5% in experimental group and 7.6% in control group. The rate difference was 0.02 (95% CI: -0.04 to 0.08), meeting the pre-specified non-inferiority criterion (P < 0.001). Patients in experimental group showed significantly improved sleep quality on the night before surgery and lower preoperative anxiety and pain scores. Furthermore, the incidence of adverse events was significantly lower in experimental group.
CONCLUSIONS AND RELEVANCE: For elderly patients undergoing major joint arthroplasty, intranasal dexmedetomidine administered the night before surgery is non-inferior to pre-induction intravenous infusion for preventing postoperative delirium. The intranasal route offers additional benefits, including improved preoperative sleep, reduced anxiety, and superior hemodynamic stability.
18. A cost-effectiveness analysis of selective decontamination of the digestive tract in mechanically ventilated patients in the intensive care unit.
主题：因果推断、RWE 与卫生经济学
相关性分数：3
期刊：Critical care and resuscitation : journal of the Australasian Academy of Critical Care Medicine
公开日期：2026-09-09（电子公开）
期刊卷期日期：2026-Dec
内容状态：待评估
为什么值得看：包含可供初筛的摘要；已识别电子公开日期；通过方法学信号门槛；仍需阅读全文评价适用性与证据质量。
OBJECTIVE: To evaluate the 24-months cost-effectiveness of selective decontamination of the digestive tract (SDD) versus standard care in mechanically ventilated Intensive Care Unit (ICU) patients.
DESIGN: Within‑trial cost‑effectiveness analysis using linked administrative healthcare datasets.
SETTING: Nineteen Australian ICUs participating in the Australian Selective Decontamination of the Digestive Tract in the ICU (SuDDICU Australia) cluster crossover randomised trial (2018-2021).
PARTICIPANTS: 2845 adult patients enrolled from New South Wales, Australia with 24‑month follow‑up (SDD, n=1252; standard care, n=1593).
INTERVENTIONS: SDD plus standard care versus standard care alone.
MAIN OUTCOME MEASURES: All‑cause mortality at 24 months; life‑years gained; healthcare resource use; healthcare costs (Australian dollars [A$]) and cost-effectiveness expressed as incremental cost-effectiveness ratio (cost per life-year gained); subgroup analyses in acute brain injuries/conditions patients.
RESULTS: At 24 months, mortality (SDD: 521/1252 [41.6%] vs standard care: [697/1593] 43.8%; p=.25), and survival probabilities (hazard ratio 0.93, 95% confidence interval: 0.83-1.04; p=.22) did not differ between groups. SDD was associated with shorter median mechanical ventilation duration (4.0 vs 4.3 days) and lower mean non-SDD antibiotics use (11.7 vs 12.9) versus standard care. Mean per-patient healthcare costs were higher with SDD (A$294,529 vs A$268,493), yielding an incremental cost-effectiveness ratio of A$669,726 per life-year gained and ∼1% probability of SDD being cost-effectiveness at the Australian willingness-to-pay threshold. Incremental cost-effectiveness ratio was lower in traumatic brain injury patients (A$169,887 per life-year gained).
CONCLUSION: In mechanically ventilated ICU patients, SDD use didn’t improve 24-month survival and healthcare costs were higher, resulting in low probability of cost-effectiveness within Australian healthcare context.
19. Ultrasensitive quantification of serum IL-5 in systemic lupus erythematosus does not reveal utility as a biomarker for disease activity or damage.
主题：因果推断、RWE 与卫生经济学
相关性分数：3
期刊：Journal of translational autoimmunity
公开日期：2026-09-07（电子公开）
期刊卷期日期：2026-Dec
内容状态：待评估
为什么值得看：包含可供初筛的摘要；已识别电子公开日期；通过方法学信号门槛；仍需阅读全文评价适用性与证据质量。
BACKGROUND: Interleukin-5 (IL-5) is the principal regulator of eosinophil biology and a key mediator of type 2 immune responses. Despite its recognized role in autoimmunity, its contribution to the pathophysiology of systemic lupus erythematosus (SLE) remains poorly defined, and no previous study has quantified IL-5 using ultrasensitive methodology in this setting. The present study aimed to measure serum IL-5 levels by ultrasensitive assay in a large, well-characterized cohort of SLE patients and to assess its relationship with a broad range of disease manifestations.
METHODS: A comprehensive characterization of 313 patients with SLE was performed, including autoantibody profiles and indices of disease activity (SLE-DAS, SLEDAI-2K, and LLDAS), damage (SLICC-DI), and remission (DORIS). Full hematological profiles were also assessed. IL-5 serum levels were measured using Simoa (Single Molecule Array) technique. A multivariable linear regression analysis was performed to examine the associations between the disease characteristics and circulating IL-5 as the dependent variable.
RESULTS: In 22 patients with SLE (7%), IL-5 levels were undetectable despite the use of an ultrasensitive assay. Therefore, the relationship between IL-5 and disease-related features was first analyzed after imputing these values, followed by a sensitivity analysis restricted to patients with detectable IL-5 levels. Overall, associations between IL-5 and most disease characteristics, including disease activity, damage, remission/low disease activity states, complement levels, and interferon-related biomarkers, were weak or inconsistent across analyses. Anti-extractable nuclear antigen antibody positivity showed a significant positive association with IL-5 in both the primary and sensitivity analyses. Likewise, no hematological cell line showed a consistent association with IL-5 across both analytical approaches.
CONCLUSION: Serum IL-5, as measured by ultrasensitive methodology, does not appear to be a robust biomarker of global disease activity, organ damage, remission status, or hematologic involvement in SLE. Accordingly, our findings do not support the clinical investigation of therapeutic IL-5 blockade in this disease.
20. Author Correction: Improving computational reproducibility in the social sciences.
主题：可复现研究与开放科学
相关性分数：3
期刊：Nature human behaviour
公开日期：2026-09-15（电子公开）
期刊卷期日期：2026-Sep-15
内容状态：待评估
为什么值得看：暂缺摘要，需回到 PubMed 核验；已识别电子公开日期；通过方法学信号门槛；仍需阅读全文评价适用性与证据质量。