方法与临床研究雷达（2026-10-06）
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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 Bayesian Adaptive Design for Assessing Treatment Effect Consistency in Bridging Studies.
主题：临床试验方法与统计实践
相关性分数：5
期刊：Statistics in medicine
公开日期：2026-Oct
内容状态：待评估
为什么值得看：包含可供初筛的摘要；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。
Bridging studies serve as an efficient approach to evaluate the applicability of findings from an original study to a new population. We propose a Bayesian adaptive design for bridging studies with time-to-event endpoints, aiming to assess the consistency of treatment effects with those observed in the original study. The design adopts a group sequential framework, using posterior probabilities at interim and final analyses to evaluate the evidence for consistency between the bridging and original studies. If strong evidence of consistency or inconsistency is observed, the trial may be stopped early to conserve sample size. Additionally, a sample size recalculation strategy based on Bayesian predictive probability is incorporated to enhance flexibility. Simulation studies demonstrate that, compared to a commonly used method, the proposed design offers favorable operating characteristics. It maintains control of the empirical type I error rate through prespecified calibration, accounting for interim analyses (IAs) and sample size adaptation, and yields substantial sample size savings when the treatment effect in the bridging study is either highly consistent or clearly inconsistent with that of the original study.
4. A Practical Guide to Optimal Adaptive Two-Stage Designs for Various Endpoint Types.
主题：临床试验方法与统计实践
相关性分数：5
期刊：Pharmaceutical statistics
公开日期：2026
内容状态：待评估
为什么值得看：包含可供初筛的摘要；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。
Adaptive designs are frequently used to adjust sample sizes based on interim data, ensuring reliable conclusions while protecting resources. To maximize trial efficiency, design parameters can be optimized to minimize an objective function (e.g., the expected sample size) while rigorously controlling overall error rates or further operating characteristics. However, existing methodological guidance for optimal adaptive designs mainly focuses on normally distributed data or discrete optimizations for binary data, leaving a limited practical framework to practitioners who work with other common endpoint types. We address this by demonstrating how the asymptotic normal approximations of standard test statistics enable the optimization framework to be systematically applied across diverse endpoint types, including binary and time-to-event data. Furthermore, we highlight critical pitfalls in using optimal adaptive designs for two-sided testing. The results demonstrate that optimizing design parameters can substantially decrease expected sample sizes and improve conditional power compared to standard group-sequential designs. Using this guidance, we aim to enable researchers to efficiently plan clinical trials in a broad spectrum of clinical settings, a factor that is especially crucial in rare disease research where only limited patient populations are available.
5. Evaluating the Impact of Outcome Delay on the Efficiency of Sample Size Re-Estimation.
主题：临床试验方法与统计实践
相关性分数：5
期刊：Pharmaceutical statistics
公开日期：2026
内容状态：待评估
为什么值得看：包含可供初筛的摘要；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。
Sample size re-estimation (SSR) can be a powerful tool to ensure that a clinical trial meets its pre-specified power requirements when uncertainty regarding a design parameter exists at the planning stage. However, long-term primary endpoints can be harmful to the efficiency of this trial design. If recruitment is continued while treatment outcomes are awaited, long delays can potentially lead to a large number of ‘pipeline’ participants being recruited in the trial that do not contribute to the interim analysis. This may lead to a larger number of recruited participants than are actually deemed required, resulting in an over-powered trial with high cost. This paper studies the exact impact of such outcome delay on the efficiency of ‘internal pilot’ type SSR designs. The distribution of the final sample size post SSR is obtained under various delay lengths for both continuous and binary outcome data; how delay impacts the precision of the final sample size estimate is then discussed. Precisely, the impact of delay on this precision is assessed through RMSE, as well as two more novel metrics, termed the delay impact and cost. The results indicate that with an increase in delay length, the delay impact increases, inflating average sample size and power. However, the severity of the effect of delayed outcomes depends highly on the exact trial setting. Trials where the re-estimated sample size is smaller than originally planned suffer the most from delayed outcomes, often leading to an over-powered trial. However, the impact of delay is substantially less if the originally planned sample size remains smaller than the re-estimated sample size.
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. Understanding return to work after breast cancer through real-world data linkage framework: a Luxembourg digital oncology use case.
主题：因果推断、RWE 与卫生经济学
相关性分数：4
期刊：ESMO real world data and digital oncology
公开日期：2026-09-23（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: Breast cancer (BC) has a substantial impact on women of working age, making return to work (RTW) a critical outcome to evaluate. Research on RTW relies on individual-level real-world data integrating clinical and occupational information. Luxembourg offers a unique setting for such studies, with a young population and a high number of crossborder workers, creating specific challenges and opportunities for studying RTW. This study aims to describe a secure data linkage framework integrating population-based cancer registry, occupational health records, and questionnaire data to evaluate RTW after BC in Luxembourg.
MATERIALS AND METHODS: A cross-sectional pilot study was conducted linking data from three sources: from Luxembourg’s National Cancer Registry (RNC), occupational health records from the Service de Santé au Travail Multisectoriel (STM), and results from a 27-item self-administered questionnaire. Eligible women were diagnosed with BC between 2013 and 2018, were younger than 65 years, employed under Luxembourgish labour law, and followed by STM. Potential participants were identified through STM records and invited to complete an electronic questionnaire and provide explicit consent for data linkage via REDCap® (Research Electronic Data Capture). A deterministic linkage between RNC and STM was enabled by using a common pseudonymisation service. Sociodemographic, clinical, and work-related characteristics were compared between linked and nonlinked participants and between women who did and did not RTW.
RESULTS: Among 452 women contacted, 171 completed the questionnaire and provided informed consent (38% response rate). Ninety-six were successfully linked with the RNC dataset. Having Luxembourg as nationality or country of residence was associated with successful linkage (P < 0.001). Among linked participants, 74 (77.1%) RTW after BC-related leave. Women who RTW were significantly younger at diagnosis than those who did not, and most resumed work in the same sector and company.
CONCLUSIONS: Pseudonymised deterministic data linkage across three real-world data sources, including population-based cancer registry data, is feasible, and provides unique insights into RTW after BC. Crossborder residency patterns and heterogeneous cancer definitions between sources were key barriers to complete linkage. The population-based approach enables a more complete picture of RTW trajectories among women with BC, supporting robust real-world evidence and informing RTW surveillance and occupational health policies in highly mobile European crossborder regions.
8. Feasibility of a prospective real-time monitoring system capturing real-world data from elderly patients with acute myeloid leukemia.
主题：因果推断、RWE 与卫生经济学
相关性分数：4
期刊：ESMO real world data and digital oncology
公开日期：2026-09-22（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: Real-world data (RWD) are increasingly applied as external controls in phase II-III clinical trials. However, retrospectively monitored RWD may suffer from limitations, including data availability and quality. Prospective monitoring of RWD may potentially overcome such limitations. We addressed the feasibility of an automated monitoring system for prospectively capturing RWD of older patients with acute myeloid leukemia (AML) and compared them with patients in the control arm of the HOVON-155 (H155) AML trial.
MATERIALS AND METHODS: The system extracted predefined data from patients receiving nonintensive therapy in 10 Dutch hospitals. Patients in the RWD cohort were further selected for comparison with patients in the H155-trial by applying the trial eligibility criteria. Caliper propensity score matching (PSM) of patients in the RWD cohort with patients in the H155-control arm was performed based on age, European LeukemiaNet 2017, and Hematopoietic Cell Transplant-Comorbidity Index.
RESULTS: Between 2020 and 2025, 193 patients in the RWD cohort were identified, of whom 120 had not participated in the H155-trial but did meet its eligibility criteria. Patients in the H155 control arm (n = 70) and patients in the RWD cohort (n = 120) differed in baseline parameters. Using PSM, 51 patients in the RWD cohort were compared with 51 patients in the H155-control arm. The 100-day mortality proportion rate was 37.3% versus 27.5% (P = 0.40) among patients in the H155 control arm and patients in the RWD cohort, respectively. Two-year overall survival was 18% ± 5% versus 18% ± 5% (hazard ratio 1.05, 95% confidence interval 0.69-1.62, P = 0.81). Bleeding and bacteremia appeared to occur more frequently among patients in the H155 control arm, whereas the frequency of other adverse events was comparable.
CONCLUSION: A prospective RWD monitoring system was efficiently applied in 10 Dutch hospitals. Although patient characteristics initially differed from the H155 control arm, the outcome of the RWD cohort appeared similar following PSM. Prospective monitoring of RWD seems feasible but matching is needed for future use in clinical studies.
9. DUBHE-H-308 study protocol: rationale and adaptive design of a randomized, open-label, seamless phaseII/III trial of iparomlimab and tuvonralimab (QL1706)-based regimens versus sintilimab plus bevacizumab as first-line treatment for advanced hepatocellular carcinoma.
主题：临床试验方法与统计实践
相关性分数：4
期刊：Future oncology (London, England)
公开日期：2026-10-01（电子公开）
期刊卷期日期：2026-Oct-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。
No randomized trial has compared multiple immunotherapy-based combination strategies head-to-head in first-line advanced HCC, where approved doublet regimens achieve objective responses in fewer than one-third of patients. Iparomlimab and Tuvonralimab (QL1706), a new-type bifunctional combinedantibody (anti-PD-1/anti-CTLA-4 dual checkpoint inhibitor), deepens antitumor immunity while preserving tolerability. DUBHE-H-308 evaluates whether QL1706-based regimens, including a triplet with bevacizumab and oxaliplatin-based chemotherapy, can improve first-line outcomes in advanced HCC. DUBHE-H-308 (NCT05976568) is a prospective, randomized, open-label, multicenter, inferentially seamless phase II/III trial comparing three QL1706-based regimens against sintilimab plus bevacizumab in previously untreated advanced HCC. Three adaptive elements are incorporated: arm selection after phase II; seamless integration of phase II data into the phase III overall survival analysis; and a group-sequential interim analysis. Phase II co-primary endpoints are objective response rate and safety; the phase III primary endpoint is overall survival. Phase II enrollment is complete; interim data have guided regimen selection and confirmed transition to the confirmatory phase III stage. If successful, DUBHE-H-308 would provide the first randomized evidence supporting a triplet first-line strategy combining dual-checkpoint blockade, anti-VEGF therapy, and chemotherapy in advanced HCC, potentially establishing a new treatment standard. The adaptive framework maximizes efficiency without compromising confirmatory rigor.Clinical trial registration: ClinicalTrials.gov identifier NCT05976568.
Advanced liver cancer that cannot be removed by surgery is usually treated with drug combinations, but current two-drug regimens shrink tumors in fewer than one in three patients. QL1706 is a new “two-in-one” antibody that releases two of the brakes cancer uses to hide from the immune system. The DUBHE-H-308 study tests whether QL1706-based combinations especially a “triplet” adding bevacizumab, which cuts off the tumor’s blood supply, and platinum-based chemotherapy, improve outcomes for previously untreated patients, compared with the standard combination of sintilimab plus bevacizumab. Patients at multiple hospitals were randomly assigned to treatment groups. The trial uses an adaptive design: an early stage compared the combinations and selected the most promising one, and the study then moved into a larger confirmatory stage that measures how long patients live.Enrollment in the early stage is complete, and interim results confirmed the move into the final confirmatory stage. If positive, this would be the first randomized evidence that a three-drug strategy dual immune activation, anti-blood-vessel therapy, and chemotherapy works as a first-line treatment for advanced liver cancer, and could become a new standard of care.
10. WIse CHoices in the ICU (WICH-ICU): Protocol and Statistical Analysis Plan for a Stepped-Wedge Cluster Randomized Trial of Choosing Wisely Interventions in Swedish Intensive Care Units.
主题：临床试验方法与统计实践
相关性分数：4
期刊：Acta anaesthesiologica Scandinavica
公开日期：2026-Nov
内容状态：待评估
为什么值得看：包含可供初筛的摘要；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: Healthcare overuse, including unnecessary diagnostic testing and procedures, contributes to patient harm, increased healthcare costs, and resource waste. The Choosing Wisely initiative aims to reduce low-value care through evidence-based recommendations. However, limited evidence exists regarding the safety and effectiveness of implementing Choosing Wisely recommendations in intensive care settings.
METHODS: The WICH-ICU is a registry-based stepped-wedge cluster randomized controlled trial designed to evaluate the implementation of specific Choosing Wisely interventions in Swedish intensive care units (ICUs). The study will include adult patients treated in participating Swedish ICUs. The intervention consists of three parts of the Choosing Wisely recommendations: (1) reduced frequency of arterial blood gas sampling, (2) reduced routine laboratory testing, and (3) reduced routine chest radiography. The primary outcome is 30-day mortality. Secondary outcomes include ICU length of stay, duration of mechanical ventilation (invasive and noninvasive), continuous renal replacement therapy duration, and ICU readmission within 72 h. Intervention data, testing intensity (arterial blood gas samples, laboratory tests, and chest radiographs per patient-day), will be collected through local monitoring, while outcome data will be obtained from the Swedish Intensive Care Registry (SIR). The stepped-wedge design allows each participating ICU to serve as its own control, with randomized timing of intervention implementation.
DISCUSSION: This study will provide evidence on the safety and effectiveness of implementing Choosing Wisely recommendations in intensive care settings. The stepped-wedge cluster randomized design minimizes contamination while allowing all participating sites to eventually receive the intervention. The results will inform evidence-based de-implementation strategies in critical care.
TRIAL REGISTRATION: ClinicalTrials.gov identifier: NCT07013175.
11. Cost-effectiveness of an online personalised parenting intervention to prevent affective disorders in high-risk adolescents compared to a standard educational package.
主题：因果推断、RWE 与卫生经济学
相关性分数：4
期刊：Journal of affective disorders
公开日期：2026-09-07（电子公开）
期刊卷期日期：2026-Dec-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。
BACKGROUND: Adolescent depression is associated with substantial health and economic burden. Preventive interventions that strengthen parenting skills show promise in reducing depressive symptoms and improving adolescent well-being. However, evidence on the cost-effectiveness of scalable online parenting interventions remains limited.
OBJECTIVES: To evaluate the cost-effectiveness of an online personalised parenting (PP) programme - Partners in Parenting - designed to prevent affective disorders in high-risk adolescents, compared with a standard educational package.
METHODS: This trial-based economic evaluation was conducted alongside a randomised controlled trial of 512 parent-adolescent dyads (256 per arm). Analyses adopted UK public sector and societal perspectives over 15 months. Costs (2022-2023 GBP) were collected prospectively. Utilities from EQ-5D-Y and CHU9D were used to estimate quality-adjusted life-years (QALYs). The base case used bivariate regression of costs and QALYs with multiple imputations under intention-to-treat. Incremental cost-effectiveness ratios, net monetary benefits, and cost-effectiveness acceptability curves were estimated. Sensitivity analyses assessed robustness.
RESULTS: In the adjusted multiply imputed base-case analysis, mean incremental public-sector costs were £639 (95% CI £272 to £988) and mean incremental QALYs were 0.013 (95% CI -0.021 to 0.042), corresponding to a 13%-27% probability of cost-effectiveness at willingness-to-pay thresholds of £20,000-£30,000 per QALY. Complete-case analysis suggested lower costs and higher QALYs but with substantial uncertainty.
CONCLUSIONS: Over the 15-month trial horizon, the personalised parenting programme was associated with higher costs, a small and uncertain gain in QALYs, negative incremental net monetary benefit, and a low probability of being cost-effective at NICE-recommended willingness-to-pay thresholds.
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. Survodutide for Obesity in Chinese Adults: Phase 3 Randomized Trial Design and Baseline Characteristics (SYNCHRONIZE™-CN).
主题：临床试验方法与统计实践
相关性分数：4
期刊：Diabetes therapy : research, treatment and education of diabetes and related disorders
公开日期：2026-08-14（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。
INTRODUCTION: We aimed to describe the rationale, design, and overall baseline characteristics of the trial evaluating the efficacy, safety, and tolerability of survodutide in Chinese adults with overweight or obesity (SYNCHRONIZE™-CN, NCT06214741).
METHODS: In this 76-week, multicenter, randomized, double-blind, placebo-controlled, phase 3 trial, Chinese participants were randomized 1:1:1 to once-weekly subcutaneous survodutide (3.6 mg or 4.8 mg) or placebo plus lifestyle intervention. Participants with either body mass index (BMI) ≥ 28 kg/m2 or BMI ≥ 24 kg/m2 and at least one obesity-related complication were eligible. The primary endpoints were the percentage change in body weight and achievement of body weight reduction ≥ 5% from baseline to week 52.
RESULTS: At baseline, participants (N = 306) had a mean age of 36.9 years, body weight 93.9 kg, waist circumference 105.6 cm, BMI 33.2 kg/m2, triglycerides 2.2 mmol/L. 49% were women. 72.9% had at least one obesity-related complication, with metabolic dysfunction‑associated steatotic liver disease (48.4%), dyslipidemia (42.2%), hypertension (27.8%), and type 2 diabetes mellitus (16.7%) being the most prevalent, followed by obstructive sleep apnea (4.6%) and cardiovascular diseases (4.2%). In total, 19.6% had prediabetes.
CONCLUSIONS: This trial was designed to provide a robust evaluation of efficacy and safety of survodutide for the treatment of obesity or overweight in Chinese adults.
TRIAL REGISTRATION: Clinical Trials.gov identifier, NCT06214741.
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. From electronic health records to real-world evidence: Savana Next-Generation Registry digital solution.
主题：因果推断、RWE 与卫生经济学
相关性分数：3
期刊：ESMO real world data and digital oncology
公开日期：2026-09-22（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: The secondary use of electronic health records (EHRs) offers a major opportunity to generate real-world evidence (RWE) to complement clinical trials and support regulatory and clinical decision making. However, a large proportion of clinically relevant information remains embedded in unstructured text, limiting its systematic reuse, particularly in complex areas such as oncology. This article presents the Savana Next-Generation Registry (SNGR), a methodology for building oncology-focused registries through the extraction and structuring of unstructured EHR data.
MATERIALS AND METHODS: SNGR uses EHRead®, a clinical natural language processing engine trained on multilingual EHRs, to transform free-text data into standardized clinical variables. By extracting clinically relevant information from unstructured clinical narratives, the approach enriches existing structured EHR data, enabling more complete and clinically meaningful datasets. The solution integrates medical expert input, terminology curation, and a multilayer quality assurance framework designed to support data validity, reproducibility, and traceability.
RESULTS: SNGR is aligned with European regulatory frameworks, including the European Health Data Space, General Data Protection Regulation, and Data Act, enabling compliant and interoperable secondary data use. To date, it has supported over 65 real-world use cases across multiple therapeutic areas, including oncology, generating >30 peer-reviewed publications and analysis-ready datasets in multicenter and international collaborations.
CONCLUSIONS: SNGR demonstrates how unstructured EHR data can be transformed into analysis-ready datasets for RWE generation. It provides a scalable and methodologically robust infrastructure for next-generation registries, particularly suited for oncology and other data-rich clinical domains, supporting research, drug development, and health care decision making. As the framework evolves, future studies will continue to expand its evidence base.
18. 36.25 Gy versus 40 Gy in five fractions of curative stereotactic body radiotherapy for prostate cancer: a propensity score-matched analysis.
主题：因果推断、RWE 与卫生经济学
相关性分数：3
期刊：Clinical and translational radiation oncology
公开日期：2026-09-19（电子公开）
期刊卷期日期：2027-Jan
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: This study aimed to determine whether 36.25 Gy was inferior to 40 Gy in terms of prostate-specific antigen (PSA) recurrence following stereotactic body radiation therapy (SBRT) for prostate cancer.
METHODS: We retrospectively reviewed the medical records of 863 consecutive patients with prostate cancer treated at a single institution between 2016 and 2025. Propensity score (PS) matching based on age, pre-treatment PSA level, biopsy Gleason score, clinical T-stage, National Comprehensive Cancer Network (NCCN) risk classification, and concomitant androgen-deprivation therapy (ADT) yielded 530 patients for analysis. Patients received SBRT at a total dose of 36.25 or 40 Gy in five fractions administered every other day. The prespecified non-inferiority margin was a hazard ratio of 1.40.
RESULTS: After PS matching, no significant differences remained in baseline characteristics that had differed significantly between the two groups before matching. In the 36.25-Gy and 40-Gy groups, respectively, 16 and 10 PSA recurrences and 18 and 10 deaths occurred. Four PSA recurrences (15%) occurred over 5 years. The 5-year event-free survival (EFS) rates were 90% with 36.25 Gy and 88% with 40 Gy (p = 0.37). The hazard ratio for EFS with 36.25 Gy versus 40 Gy was 0.76 (95% confidence interval [CI], 0.42-1.39; p = 0.37). The 5-year PSA recurrence-free rates were 95% and 93%, respectively (p = 0.76). Among the 298 patients receiving 36.25 Gy, grade 3 gastrointestinal (GI) and genitourinary (GU) toxicities occurred in 2.3% and 1.3%, respectively. Among the 565 patients receiving 40 Gy, grade 3 or GI toxicity occurred in 1.9%, and grade ≥ 3 GU toxicity occurred in 0.8%. No grade 5 toxicity occurred. Urinary and bowel quality of life (QOL) scores decreased at 1 month and returned to near-baseline levels by 3 months.
CONCLUSIONS: In this retrospective study, the findings suggest that the 36.25-Gy regimen may be non-inferior to 40 Gy for EFS and may be associated with fewer grade 4 toxicities and better urinary QOL. Given the retrospective design, these findings should be considered hypothesis-generating.
19. Surgery followed by adjuvant radiotherapy versus definitive radiotherapy for locally advanced squamous cell carcinoma of the pharynx and larynx: a propensity score-matched analysis.
主题：因果推断、RWE 与卫生经济学
相关性分数：3
期刊：Clinical and translational radiation oncology
公开日期：2026-09-10（电子公开）
期刊卷期日期：2027-Jan
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。
Aim: Locally advanced squamous cell carcinoma (SCC) of the pharynx and larynx is commonly treated with either primary surgery followed by adjuvant radiotherapy (RT) or definitive RT with or without concurrent systemic therapy. Comparative outcome data between these treatment strategies remain limited because retrospective analyses are frequently affected by treatment-selection bias. This study compared oncologic outcomes between primary surgery followed by adjuvant RT and definitive RT using propensity score matching to reduce baseline confounding. Methods: A retrospective analysis was performed on 309 patients with locally advanced SCC of the oropharynx, hypopharynx, or larynx treated at our center. Propensity scores were generated using a multivariable logistic regression model including T stage, N stage, and age at diagnosis. One-to-one nearest-neighbor matching with a caliper of 2% resulted in 61 matched patient pairs treated with primary surgery followed by adjuvant RT or definitive RT. Survival outcomes were analyzed using the Kaplan-Meier method and compared using log-rank tests. Multivariable Cox regression analyses were performed to account for imbalances in previous malignancies after matching. Results: After propensity score matching, 61 patients treated with primary surgery followed by adjuvant RT and 61 patients treated with definitive RT were included, with improved balanced baseline characteristics for the matching variables. Median OS was 81 months in the surgery followed by adjuvant RT group and was not reached in the definitive RT group, with no statistically significant difference between groups (p = 0.70). Numerical differences favored adjuvant RT for progression-free survival (median PFS: 81.0 vs. 43.4 months; p = 0.09) and loco-regional recurrence-free survival (p = 0.08), although these differences were not statistically significant. Lymphedema was the only toxicity significantly more frequent in the adjuvant RT cohort (33% vs. 13%, p = 0.017), while rates of other treatment-related toxicities were comparable. Multivariable analyses adjusted for previous malignancies confirmed the absence of significant associations between treatment modality and survivals. Conclusion: Primary surgery followed by adjuvant RT and definitive RT achieved comparable overall survival in patients with locally advanced SCC of the pharynx and larynx after propensity score matching. However, the surgery plus adjuvant RT strategy demonstrated trends toward improved disease control and local control. These findings support individualized multidisciplinary treatment selection while prospective comparative studies remain warranted.
20. Comment on “A pragmatic trial evaluating the impact of the anumana clinical decision support tool for guideline-directed management of heart failure (ACT-HF): Clinical trial design and methods”.
主题：临床试验方法与统计实践
相关性分数：3
期刊：American heart journal plus : cardiology research and practice
公开日期：2026-09-16（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：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。