方法与临床研究雷达（2026-09-02）
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1. Cost-effectiveness of nicotine metabolite ratio-guided smoking cessation therapy in China: a hybrid decision-analytic modeling study based on real-world data.
主题：因果推断、RWE 与卫生经济学
相关性分数：8
期刊：Journal of medical economics
公开日期：2026-09-01（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。
AIMS: To estimate the projected cost-effectiveness of NMR-guided metabolism-informed care (MIC) compared with standard care (SC) for smoking cessation in China.
MATERIALS AND METHODS: A 24-week decision tree was linked to an annual semi-Markov cohort model that followed individuals from age 45 to age 100. Clinical inputs were informed by the China National Tobacco Cessation Cohort Study. Stabilized inverse probability of treatment weights were estimated using a multinomial propensity-score model, trimmed at the 1st and 99th percentiles, and analyzed using robust sandwich variance estimators. Under MIC, fast metabolizers (NMR ≥ 0.31) received varenicline, while slow metabolizers (NMR < 0.31) received either nicotine replacement therapy (NRT) or bupropion. Costs were expressed in 2023 Chinese yuan (CNY), and costs and health outcomes were discounted at 5% annually.
RESULTS: Over the lifetime model horizon, SC generated 13.125 QALYs per person at a total cost of CNY 40,832.29. Both MIC medication-cost scenarios generated an additional 0.003 QALYs. MIC-Bupropion cost CNY 41,033.81 and yielded an ICER of CNY 67,930.00 per QALY gained versus SC. The corresponding deterministic INMB versus SC was CNY 63.57 at the primary threshold. In the fully incremental analysis, MIC-NRT was strictly dominated by MIC-Bupropion. At CNY 89,358.00 per QALY, MIC-Bupropion had the highest net monetary benefit in 50.4% of probabilistic simulations.
LIMITATIONS: Findings depended on observational clinical inputs, literature-derived long-term parameters, and structural assumptions, and did not fully capture all smoking-related diseases, adherence, adverse effects, or implementation costs.
CONCLUSIONS: MIC-Bupropion showed favorable expected economic performance relative to SC, whereas MIC-NRT was strictly dominated. Further comparative-effectiveness, budget-impact, and implementation studies are warranted before routine adoption.
Medicines can help people quit smoking, but the right choice varies from person to person. The nicotine metabolite ratio (NMR)-a lab test that measures how quickly the body breaks down nicotine-could help doctors match each smoker to the most appropriate medication.We used real-world data from 1,100 people who sought smoking cessation treatment in China to estimate the costs and health benefits of NMR-guided care compared with standard care. Under NMR-guided strategy, fast metabolizers received varenicline, while slow metabolizers received either nicotine replacement therapy (NRT) or bupropion, resulting in two medication-cost scenarios for this economic evaluation. Because treatment assignment in the cohort was not randomized, we adjusted for measured differences between medication groups. The economic model assigned NRT and bupropion the same probability of quitting smoking.Under this assumption, the two MIC medication-cost scenarios produced the same health gains but differed in cost. The MIC-Bupropion scenario was less costly than MIC-NRT and had a cost per additional quality-adjusted life-year below the primary willingness-to-pay threshold. However, the MIC-bupropion scenario had the highest net monetary benefit in only 50.4% of probabilistic simulations at that threshold, indicating substantial decision uncertainty.This analysis estimated outcomes per person treated and provided an initial economic basis for considering NMR-guided care in China. Further studies on comparative effectiveness, budget impact, and real-world implementation are required. Nonetheless, the results suggest that NMR-guided care, especially using bupropion for slow metabolizers, is a promising and potentially cost-effective strategy worthy of further investigation and pilot implementation.
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. Clinico-genomic risk stratification for biomarker-enriched trial design in poor-prognosis metastatic castrate-resistant prostate cancer (mCRPC).
主题：临床试验方法与统计实践
相关性分数：5
期刊：The journal of liquid biopsy
公开日期：2026-08-14（电子公开）
期刊卷期日期：2026-Sep
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: An integrated prognostic risk-score (RS) based on prognostic clinical factors and plasma copy number alterations (CNAs) across independent metastatic castration-resistant prostate cancer (mCRPC) cohorts was applied as a classifier to guide development of biomarker-enriched clinical trial designs.
DESIGN: Plasma CNAs prognostic for survival were combined into a prognostic score and integrated with clinical prognostic factors to derive an integrated RS in three independent mCRPC cohorts. Biomarker-enrichment trial designs were simulated across a spectrum of enrichment fractions and enrichment thresholds for the RS to consider 1:1 randomization of high RS patients to a study treatment arm or to receive standard treatment, with either a two or a three-year follow-up. The enrichment strategy was applied to improve the trial’s ability to detect survival benefit at a Hazard Ratio 0.70; 80% power. Design trade-offs were assessed between the number of “high-risk” patients screened versus enrolled under varying enrichment thresholds and degrees of enrichment of the RS.
RESULTS: Pooled mCRPC patients (N = 561) showed RS values from 0.243 to 3.924 and a RS ≥ 1 (60th percentile) was associated with shorter survival. Elastic net models for 2- and 3-year survival were developed. At a threshold ≥1 with 2-year follow-up, a hybrid biomarker-enriched design (50% unselected, 50% high-risk) required screening 768 patients to enroll 219 per arm (n = 438), while maintaining adequate power.
CONCLUSION: An integrated clinico-genomic classifier can guide an mCRPC enrichment strategy by enriching a cohort with 50% short-survival patients balancing sample size and screening needs to achieve adequate power in a biomarker-enriched design.
5. Sample Size Reduction by Applying ML Based Causal Inference Methods.
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。
We conducted a comprehensive comparative analysis of causal machine learning (ML) methods to assess their utility in improving the efficiency of clinical trial designs with or without historical data. Specifically, we compared standard ANCOVA analysis used in a Randomized Controlled Trial (RCT) with several causal ML methods, including PROCOVA, TMLE, DML, and GRF. PROCOVA is gaining popularity in RCT design and requires a historical data for prognostic scores, but other methods can be applied with or without such data. Our primary focus was on strict RCT setting without borrowing historical control data, though we also explored the impact of borrowing data. The historical data used consisted of placebo data from two Phase 3 Ophthalmology studies with a continuous primary endpoint. We employed a generative AI approach, specifically Generative Adversarial Networks (GANs), to simulate RCT data from the historical data under various scenarios, varying treatment effects with and without treatment effect heterogeneity, RCT sizes, and bias. Results showed that causal ML methods can increase power even without borrowing historical data. For example, TMLE increased effective sample size by 21% in one scenario. In scenarios with borrowing of controls, PROCOVA increased power while controlling type 1 error, showing robustness to model misspecification.
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. Efficacy and safety of thalidomide (1 vs 2 mg/kg/d) in transfusion-dependent thalassemia: A Non-Inferiority Trial.
主题：临床试验方法与统计实践
相关性分数：4
期刊：Blood advances
公开日期：2026-09-01（电子公开）
期刊卷期日期：2026-Sep-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。
Thalidomide has emerged as a fetal hemoglobin-inducer with potential to reduce transfusion burden in transfusion-dependent thalassemia (TDT). However, optimal dosing remains undefined A prospective, randomized, open-label, multicentric clinical trial was conducted at four centres in India to compare efficacy and safety of thalidomide at 1 mg/kg/day (Group 1) versus 2 mg/kg/day (Group 2) in patients with TDT aged ≥12 years. The primary endpoint was reduction in transfusion requirement at week 24, categorized as good (>50%), moderate (25-50%), or no (<25%) response. Responders underwent stepwise dose tapering during weeks 25-72 to evaluate response sustainability. Safety assessments were performed every 4 weeks. Of 188 enrolled patients (94 per group), 82.4% completed the week-24 evaluation. The overall response rate (ORR) was 58.5%, significantly higher in Group 1 than Group 2 (67.1% vs 50.0%, p=0.012). Good, moderate, and no response were observed in 20.5%, 37.8%, and 41.6% of patients, respectively, with transfusion independence in 9 patients (5.6%). Clinical benefit was observed within 12 weeks in 71.8% of good responders and 100% moderate responders. Sustained response at week 72 was seen in 49% and 57.5% of initial responders in Groups 1 and 2, respectively. Adverse effects were mostly grade 1; drug discontinuation due to toxicity was required in 10.6 % participants. These findings suggest that Thalidomide at 1 mg/kg/day was non-inferior to 2 mg/kg/day in reducing transfusion burden in patients with TDT, with an acceptable safety profile. Low-dose thalidomide appears to be a feasible treatment option in resource-limited settings. ICMR trial registry (Trial no. CTRI/2022/05/042781).
8. [Evaluation of the efficacy and safety of multi-wavelength semiconductor laser for oral soft tissue incision and gingivectomy: a prospective, multicenter, randomized, parallel-controlled, non-inferiority clinical trial].
主题：临床试验方法与统计实践
相关性分数：4
期刊：Zhonghua kou qiang yi xue za zhi = Zhonghua kouqiang yixue zazhi = Chinese journal of stomatology
公开日期：2026-09-01（电子公开）
期刊卷期日期：2026-Sep-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。
Objective: To evaluate the clinical efficacy and safety of the multi-wavelength semiconductor laser therapeutic instrument in oral soft tissue incision and gingivectomy. Methods: A total of 156 patients who received mandibular impacted wisdom tooth extraction combined with oral soft tissue incision or gingivectomy at the Stomatological Hospital of Zhejiang University School of Medicine, Zhejiang Hospital and Wenzhou Central Hospital between June 2023 and March 2024 were enrolled and divided into experimental group (n=78) and control group (n=78) by stratified block randomization method. During the operation, the experimental group used the multi-wavelength semiconductor laser therapeutic instrument to complete soft tissue incision and gingivectomy in the surgical area, while the control group used the single-wavelength semiconductor laser therapeutic instrument for the corresponding procedures. Follow-up assessments were conducted one week and one month postoperatively for both groups of patients. Primary outcomes were the excellent and good rate of intraoperative hemostasis and wound healing at one week postoperatively, which were analyzed by non-inferiority test. Secondary outcomes included laser cutting efficiency, convenience of disinfection, operational convenience, operational safety and stability, which were assessed using the Wilcoxon rank-sum test, χ2 test or Fisher’s exact probability method. Safety indicator was the incidence of adverse events, which was analyzed using Fisher’s exact probability method. Results: Primary efficacy evaluation: the excellent and good rate of intraoperative hemostasis [100.0% (78/78) vs. 100.0% (78/78)] and wound healing [100.0% (78/78) vs 98.7% (77/78)] in the experimental group were both non-inferior to those in the control group. Secondary efficacy evaluation: the laser cutting efficiency in experimental group [(0.08±0.07) mm/s] was significantly higher than that in control group [(0.06±0.04) mm/s] (Z=-2.87, P=0.004). There were no significant differences in disinfection convenience, operational convenience, safety, and stability between the two groups (all P>0.05). Regarding safety evaluation, no adverse events occurred in the experimental group, while 2 cases (2.6%) of mild or moderate adverse events and 1 case (1.3%) of serious adverse events occurred in the control group, with no significant difference (P>0.05). Conclusions: The efficacy and safety of the multi-wavelength semiconductor laser therapeutic instrument are non-inferior to the control products, meeting the standards for clinical validation.
目的： 评价多波长半导体激光治疗仪用于口腔软组织切开、牙龈切除术的临床有效性和安全性。 方法： 纳入2023年6月至2024年3月在浙江大学医学院附属口腔医院、浙江医院、温州市中心医院就诊，需拔除下颌阻生智齿且需进行口腔软组织切开、牙龈切除的156例患者，采用分层区组随机法将患者分为试验组（78例）和对照组（78例），术中试验组使用多波长半导体激光治疗仪完成术区软组织切开及牙龈切除操作，对照组使用单波长半导体激光治疗仪完成术区软组织切开及牙龈切除操作。对两组患者术后1周、1个月进行随访。主要有效性评价指标为术中止血效果优良率、术后1周伤口愈合情况优良率，采用非劣效检验；次要有效性评价指标为激光切割效率、设备的消毒便利性、操作便利性、操作安全性及稳定性，采用Wilcoxon秩和检验、χ2检验或Fisher精确概率法检验；安全性指标为不良事件发生率，采用Fisher精确概率法检验。 结果： 主要有效性评价：试验组术中激光止血效果优良率为100.0%（78/78），术后1周伤口愈合情况优良率为100.0%（78/78），均非劣于对照组的100.0%（78/78）、98.7%（77/78）。次要有效性评价：试验组激光切割效率［（0.08±0.07）mm/s］显著高于对照组［（0.06±0.04）mm/s］（Z=-2.87，P=0.004），消毒便利性、操作便利性、安全性及稳定性评价与对照组相比差异均无统计学意义（均P>0.05）。安全性评价方面，试验组无不良事件发生，对照组发生3例不良事件，2例（2.6%）为轻、中度不良事件，1例（1.3%）为严重不良事件，两组不良事件发生率差异无统计学意义（P>0.05）。 结论： 多波长半导体激光治疗仪用于下颌阻生智齿拔除患者口腔软组织切开、牙龈切除术的有效性和安全性良好，与对照组相当。.
9. Efficacy and safety of semaglutide injection in Indian patients with type 2 diabetes mellitus inadequately controlled on metformin: A phase 3, randomized, active-controlled, multicenter, non-inferiority trial (WIN-IND study).
主题：临床试验方法与统计实践
相关性分数：4
期刊：Metabolism open
公开日期：2026-08-05（电子公开）
期刊卷期日期：2026-Sep
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: Type 2 diabetes mellitus (T2DM) is a chronic metabolic disorder with a rapidly rising global burden. Present study established the non-inferiority and evaluated the safety of Intas semaglutide (test) compared to Innovator semaglutide (reference) in patients with T2DM inadequately controlled on metformin.
METHODS: This prospective, randomized, open-label, multicenter phase 3 trial, conducted between July-2025 and February-2026, enrolled adult patients (18-65 years) with T2DM and HbA1c level ≥7%-<10.5% at baseline, who had been on diet, exercise and metformin (≥1500 mg/day). Randomized (1:1) patients received either test or reference product, once weekly from 1 to 24 weeks. Primary endpoint was the change in HbA1c level. Secondary endpoints were the change in fasting blood glucose (FBG) level, post-prandial blood glucose (PPBG) level, bodyweight, body mass index (BMI), and lipid parameters. Safety assessment included monitoring of treatment emerged adverse events (TEAEs).
RESULTS: Study analyzed 223 (safety set) and 217 (ITT set) patients. Baseline characteristics were comparable between treatment groups. In the ITT (LOCF) set, at week-24, LSM change in HbA1c was -1.50 vs. -1.65 in test vs. reference group, respectively; LSM difference was 0.16 (95% CI: -0.16 to 0.47, P = 0.3266), confirming non-inferiority of the test product. Other efficacy parameters (FBG and PPBG levels, bodyweight and BMI) were also similarly reduced in the test group. The incidence, severity, and pattern of TEAEs were similar, without new safety signals identified.
CONCLUSION: Intas semaglutide showed non-inferiority to reference semaglutide with comparable efficacy, immunogenicity, and safety which may provide a cost-effective alternative treatment option to Indian patients with T2DM.
CLINICAL TRIAL REGISTRATION NUMBER: CTRI/2025/06/089290.
10. Comparison of the efficacy and safety of acetaminophen versus NSAIDs for the treatment of chronic pain in older adults with osteoarthritis of the hip and knee: Findings from the randomized, double-blind, parallel-group, non-inferiority RETHINK study.
主题：临床试验方法与统计实践
相关性分数：4
期刊：Osteoarthritis and cartilage open
公开日期：2026-07-06（电子公开）
期刊卷期日期：2026-Sep
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: This study aimed to evaluate the non-inferiority of acetaminophen compared with non-steroidal anti-inflammatory drugs (NSAIDs) for the treatment of chronic osteoarthritis-related pain in older adults.
DESIGN: This multicenter, randomized, double-blind, parallel-group study enrolled patients aged 65 years or older with osteoarthritis-related pain. Participants were randomly assigned to receive acetaminophen (1800 mg/day) or NSAIDs (loxoprofen 180 mg/day or celecoxib 200 mg/day). The primary endpoint was the change in Brief Pain Inventory (BPI) item 3 (worst pain) score from baseline to week 8. The secondary endpoints included the change in BPI item 3 score from baseline to week 4, quality of life, gastrointestinal disorders, and renal and liver function parameters.
RESULTS: Of the 400 patients enrolled, 191 and 197 were in the acetaminophen (mean age 73.6 years; 83.2% female) and NSAID groups (mean age 73.3 years; 74.6% female), respectively. The least-squares mean change in BPI item 3 scores at 8 weeks was -1.79 in the acetaminophen group and -1.94 in the NSAIDs group. The between-group difference in BPI item 3 scores change was 0.14 (95% CI, -0.33 to 0.61). No major safety concerns were identified; however, gastrointestinal disorders occurred more frequently with NSAIDs and were the most common cause of treatment discontinuation.
CONCLUSIONS: In this RETHINK study, acetaminophen achieved a similar reduction in osteoarthritis-related pain to NSAIDs in older adults after eight weeks; however, non-inferiority was not demonstrated. In terms of adverse events, acetaminophen was associated with fewer gastrointestinal disorders. These findings suggest that treatment choice may depend on the balance between analgesic efficacy and safety considerations.
TRIAL REGISTRATION NUMBER: The study is registered in the Japan Registry of Clinical Trials (jRCTs071200112).
11. 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).
12. 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.
13. Resuscitation in paediatric septic shock using vitamin C and hydrocortisone (RESPOND): The RESPOND randomised controlled trial statistical analysis plan.
主题：临床试验方法与统计实践
相关性分数：4
期刊：Critical care and resuscitation : journal of the Australasian Academy of Critical Care Medicine
公开日期：2026-06-16（电子公开）
期刊卷期日期：2026-Sep
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 Resuscitation in Paediatric Septic Shock using Vitamin C and Hydrocortisone (RESPOND) trial is a multicentre randomised controlled trial exploring whether the use of hydrocortisone alone, or in combination with vitamin C, increases time alive and free of vasopressors for critically ill children.
OBJECTIVE: To present the prespecified statistical analysis plan (SAP) for the RESPOND trial prior to finalising recruitment and locking the trial dataset.
DESIGN SETTING AND PARTICIPANTS: The RESPOND trial is a three-arm, parallel group, open-label, randomised controlled trial, recruiting in paediatric intensive care units in Australia, New Zealand, India, and Brazil. The planned sample size is 384 participants.
MAIN OUTCOME MEASURES: The primary outcome is time alive and free of inotropes/vasopressors, censored at 7 days post-randomisation. Secondary outcomes include clinical (e.g. alive and free of multi-organ dysfunction, length of stay), safety, health economics (e.g. incremental costs, quality-adjusted life years), and long-term outcomes (measured at 6 months post-randomisation; e.g. health-related quality of life).
RESULTS AND CONCLUSIONS: The SAP was designed by the Chief Investigators and approved by the RESPOND Steering Committee. Statistical analyses are summarised. The primary outcome will be analysed using quantile regression adjusted for stratification variables. Appropriate statistical comparisons between groups were planned and described in a way that is transparent, available to the public, verifiable, and predetermined before completion of data collection. The trial statistician, RESPOND Steering Committee members, and SAP authors remain blind to treatment allocation throughout the study. Data Safety and Monitoring Board members were provided with safety data with masked group identifiers during interim analyses. The RESPOND trial commenced recruitment in December, 2021, and aims to complete recruitment by mid-2026.
TRIAL REGISTRATION: ACTRN12621000247875.
14. Endoscopic Surveillance in Serrated Polyposis Syndrome, Two or Three-Year Intervals: A Non-inferiority Randomized Trial.
主题：临床试验方法与统计实践
相关性分数：4
期刊：Digestive diseases and sciences
公开日期：2026-04-21（电子公开）
期刊卷期日期：2026-Sep
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: Serrated polyposis syndrome, the most prevalent colonic polyposis, confers an increased colorectal cancer risk. Guidelines recommend close colonoscopy surveillance, but recent data suggest low neoplasia rates, supporting longer colonoscopy intervals.
AIMS: Compare advanced neoplasia incidence between two- and three-year surveillance.
METHODS: A multicentre, randomized non-inferiority trial was conducted (May 2021-November 2024) in six Spanish hospitals. Patients fulfilling the 2019 WHO criteria for serrated polyposis syndrome, including newly diagnosed individuals and those already under surveillance, with no advanced neoplasia and fewer than five relevant polyps at their previous colonoscopy, were randomized to surveillance at 2 or 3 years. The primary endpoint was advanced neoplasia incidence.
RESULTS: A total of 131 patients with serrated polyposis syndrome were included (47.3% women; mean age 66.1). Seventy-two were assigned to 2-year and 59 to 3-year colonoscopy. Among 771 resected lesions, 2.4% were advanced adenomas or advanced serrated polyps; no colorectal cancer was detected. The proportion of patients with advanced neoplasia in the surveillance colonoscopy was 6.9% (2-year) vs 13.6% (3-year), with no statistical difference (p = 0.208) but with a risk difference of + 6.7% (95% CI -4.1 to 17.5%) exceeding the pre-specified non-inferiority margin of + 10%. Time since serrated polyposis syndrome diagnosis ≤ 3 years was associated with advanced neoplasia (OR 4.4; 95% CI 1.56-14.71; p = 0.024).
CONCLUSIONS: In patients with serrated polyposis syndrome, extending colonoscopy surveillance to a three-year compared with a two-year interval yielded inconclusive evidence regarding non-inferiority for advanced neoplasia incidence. The early years following serrated polyposis syndrome diagnosis were identified as a risk factor for advanced neoplasia.
TRIAL REGISTRATION: Clinical Trial Registry ClinicalTrials.gov (NCT04906343). Date: 5-10-2021.
15. STAT3 phosphorylation as an endpoint in a functional cell-based neutralizing antibody assay with potential for broad application.
主题：临床试验方法与统计实践
相关性分数：3
期刊：Bioanalysis
公开日期：2026-09-01（电子公开）
期刊卷期日期：2026-Sep-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: PF-07314470 is an antibody that was tested in a first-in-human study to evaluate its safety, pharmacokinetics, and pharmacodynamics. Because PF-07314470 is an interleukin-27 receptor (IL-27 R) agonist, the feasibility of a cell-based neutralizing antibody (NAb) assay was assessed as a tool to characterize anti-drug antibodies (ADA). Identifying a suitable assay endpoint is critical to NAb assay development. Phosphorylation of signal transducer and activator of transcription 3 (STAT3) was chosen, because it reflected activation of IL-27 R and could be measured by different means.
METHODS: PF-07314470 was used to activate IL-27 R on engineered Chinese Hamster ovary (CHO) cells, and resultant phospho-STAT3 (pSTAT3) levels were measured using a Meso Scale Discovery (MSD) assay. Experiments were conducted to confirm specificity of the pSTAT3 response and its inhibition by a NAb positive control. Biotin-Drug Extraction and Acid Dissociation (BEAD) was used to enhance drug tolerance.
RESULTS: PF-07314470 induced pSTAT3 that was reliably measured by MSD. The pSTAT3 response was specific to PF-07314470 and could only be inhibited by a positive control against PF-07314470. Assay sensitivity and drug tolerance were achieved through implementation of BEAD.
CONCLUSIONS: Experiments demonstrated the feasibility of a cell-based NAb assay for PF-07314470. The assay system could be used for other biotherapeutics that modulate pSTAT3.
When a biotherapeutic is given to a person, that person’s body may produce antibodies against the biotherapeutic. Some of these antibodies may prevent the biotherapeutic from doing its job; they are called “neutralizing antibodies.” In clinical trials, blood samples are collected from participants. When appropriate, these samples are used in an assay (or test) to detect the neutralizing antibodies. In general, assays to detect neutralizing antibodies are difficult to develop. PF-07314470 is a biotherapeutic that was tested in a clinical trial. We performed experiments to develop the framework of an assay that would detect neutralizing antibodies against PF-07314470. The system used in our assay may be suitable for other biotherapeutics that work like PF-07314470. However, this should be tested for each biotherapeutic, and some modifications (within the assay framework) may be needed.
16. Generalized linear multiple kernel learning for high-dimensional image data.
主题：统计建模与试验设计方法
相关性分数：3
期刊：Statistics & probability letters
公开日期：2026-08-21（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。
We propose GLIMARK, a sparse generalized linear multiple-kernel framework for high-dimensional structured predictors. It extends additive kernel learning to exponential-family outcomes, supports multi-view and hierarchical feature representations, improves scalability via Nyström approximation, and yields interpretable feature-kernel and representer selection.
17. Clinical Utility of Anti-reflux Mucosal Intervention for Refractory Gastroesophageal Reflux Disease in Patients With a Hiatal Hernia.
主题：因果推断、RWE 与卫生经济学
相关性分数：3
期刊：DEN open
公开日期：2026-08-31（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: Surgical fundoplication is the standard treatment for refractory gastroesophageal reflux disease (GERD) with large (≥3 cm) hiatal hernias; however, its invasiveness is problematic in older adults and high-risk patients. Anti-reflux mucosal intervention (ARMI) is a less invasive endoscopic alternative; however, patients with large hernias have previously been excluded from studies, limiting available evidence. We assessed the feasibility, short-term clinical outcomes, and safety of ARMI in this population.
METHODS: We retrospectively analyzed consecutive patients with proton pump inhibitor (PPI)/potassium-competitive acid blocker (P-CAB)-refractory GERD and sliding hiatal hernias ≥3 cm who underwent ARMI (anti-reflux mucosectomy; anti-reflux mucoplasty) between April 2024 and April 2025. GERD-Health-Related Quality of Life (GERD-HRQL), GERD Questionnaire (GerdQ), and Frequency Scale for the Symptoms of GERD (FSSG) scores were compared before ARMI and 3 months afterward. PPI/P-CAB discontinuation and treatment-related adverse events were also assessed.
RESULTS: Ten patients (mean age 74.3 years; 80% women; hernia size 3-6 cm) were included, all with American Society of Anesthesiologists physical status ≥ II. At 3 months, mean GERD-HRQL scores decreased from 21.1 to 4.6 (p = 0.002), FSSG from 22.6 to 6.2 (p = 0.004), and GerdQ from 10.9 to 4.6 (p = 0.008). PPI/P-CAB was discontinued in seven patients. No clinically significant treatment-related adverse events, including perforation, delayed bleeding, or dysphagia, occurred.
CONCLUSIONS: ARMI was technically feasible, showed favorable short-term outcomes in patients with refractory GERD and 3-6-cm hiatal hernias, and may represent a less invasive option for older or high-risk patients.
TRIAL REGISTRATION: N/A.
18. Reverse total shoulder arthroplasty versus open reduction internal fixation for proximal humerus fractures in elderly patients: an age-stratified 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：Journal of shoulder and elbow arthroplasty
公开日期：2026-07-17（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 optimal surgical management of displaced proximal humerus fractures in elderly patients remains controversial. Reverse total shoulder arthroplasty (rTSA) has gained increasing acceptance for complex fracture patterns, yet comparative data against open reduction and internal fixation (ORIF) across age strata are limited. This study compared reoperation rates, emergency department (ED) utilization, and dislocation following rTSA versus ORIF in propensity score-matched cohorts stratified by age.
METHODS: Using the TriNetX multi-institutional research network, patients aged 65-74 years and 75-84 years who underwent rTSA or ORIF for proximal humerus fractures were identified. Propensity score matching was performed using nearest-neighbor matching with a 0.150 standard deviation caliper. The primary outcome was coded shoulder-related return to surgery at 1 and 2 years. Secondary outcomes included all-cause ED utilization and coded dislocation when available.
RESULTS: After matching, 748 patients (374 per group) were analyzed in the 65 to 74-year cohort and 882 patients (441 per group) in the 75 to 84-year cohort. Patients undergoing rTSA had significantly more 4-part fractures (34.2% vs. 6.4% in the 65 to 74-year cohort, P < .001). At 2-year follow-up, reoperation rates did not differ significantly in either age group (65-74 years: rTSA 5.62% vs. ORIF 3.48%, risk ratio 1.62, P = .16; 75-84: rTSA 4.54% vs. ORIF 5.67%, risk ratio 0.80, P = .444). Kaplan-Meier survival analysis confirmed no significant differences in freedom from surgical failure. ED utilization did not differ significantly between groups, and dislocation was comparable in the 75 to 84-year cohort for which dislocation data were available.
CONCLUSION: In propensity score-matched cohorts, rTSA and ORIF demonstrated comparable coded reoperation rates and all-cause ED utilization at 1- and 2-year follow-up. These findings persisted despite rTSA patients having more complex fracture patterns; however, because fracture morphology and patient-reported outcomes were not directly matched or captured, the results should be interpreted as hypothesis-generating real-world associations rather than evidence of treatment superiority.
19. Validation of Ultra-Widefield Autofluorescence Imaging as an Endpoint for Disease Progression in Choroideremia: A Comparative Study.
主题：临床试验方法与统计实践
相关性分数：3
期刊：Ophthalmology science
公开日期：2026-07-24（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。
OBJECTIVE: Promising gene therapy studies in patients with choroideremia (CHM) have failed to meet the primary endpoint of best-corrected visual acuity, but other therapeutic effects in the peripheral fundus may not have been discovered. Ultra-widefield fundus autofluorescence (UWF-FAF) is able to capture peripheral retinal changes as potential structural endpoint. We compared UWF-FAF with conventional FAF in patients with CHM to evaluate its potential as additional imaging modality for future clinical trials.
DESIGN: Retrospective cohort study.Subjects: Participants with genetically confirmed CHM and both central 30° and 55° FAF and UWF-FAF imaging available.
METHODS: Conventional FAF (30° or 55°) and UWF-FAF images obtained on the same day were retrospectively aligned for direct comparison and then analyzed. Two independent graders outlined atrophic areas and preserved retinal pigment epithelium (RPE) islands. Between-modality differences with standard errors, 95% confidence intervals (CIs), and 2-sided P values (α = 0.05) were calculated. Intergrader agreement was assessed using intraclass correlation coefficients (ICCs).
MAIN OUTCOME MEASURES: Between-modality differences in area measurements of FAF and UWF-FAF with standard errors, 95% CIs and ICCs.
RESULTS: Ninety-six FAF images from 15 patients were included (35 FAF 30°, 20 FAF 55°, and 41 UWF-FAF). Intergrader ICCs ranged from 0.625 to 0.998, indicating good-to-excellent agreement. Within the 30° field, no significant difference was found for the total cohort (0.01; 95% CI, -0.02 to 0.04; P = 0.438) or for atrophy (-0.06; 95% CI, -0.14 to 0.01; P = 0.089), whereas preserved RPE islands differed significantly (0.04; 95% CI, 0.01-0.06; P = .005). Within the 55° field, differences did not reach statistical significance for the total cohort (0.08; 95% CI, -0.01 to 0.18; P = 0.077), atrophy (0.10; 95% CI, -0.09 to 0.28; P = 0.260), or islands (0.07; 95% CI, -0.001 to 0.15; P = 0.054).
CONCLUSION: In our current study UWF-FAF provided comprehensive atrophy measurements quantitatively comparable to conventional FAF in CHM, supporting its usefulness as a structural clinical trial endpoint for disease progression. Due to significant differences in preserved RPE island measurements the modalities should not be used interchangeably.
FINANCIAL DISCLOSURES: The authors have no proprietary or commercial interest in any materials discussed in this article.
20. Laryngeal mask airway use during liver transplantation: perioperative outcomes in a propensity score-matched cohort.
主题：因果推断、RWE 与卫生经济学
相关性分数：3
期刊：Annals of medicine
公开日期：2026-08-30（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: Whether a laryngeal mask airway (LMA) can be safely used during liver transplantation remains unclear. However, in clinical practice, the choice of an airway device may also reflect a broader recovery-oriented perioperative strategy rather than an isolated technical substitution.
METHODS: We retrospectively reviewed adult patients who underwent primary elective liver transplantation at our center between January 2024 and November 2025. The patients were grouped according to their primary intraoperative airway device (LMA or endotracheal tube [ETT]). Propensity score matching was used to reduce baseline imbalance. Intraoperative variables, postoperative airway-related events, postoperative pulmonary complications (PPCs), intensive care unit (ICU) stays, and postoperative hospital stays were compared.
RESULTS: After matching, 25 and 44 patients in the LMA and ETT groups, respectively, were analyzed. The LMA group showed a higher rate of immediate airway device removal, lower rocuronium use during anesthetic maintenance, less postoperative noninvasive ventilation, less postoperative sore throat, and a shorter postoperative hospital stay. PPCs were numerically less frequent in the LMA group, but the between-group difference was not statistically significant after matching. No increase in major airway-related adverse events was observed in the LMA group.
CONCLUSIONS: In carefully selected liver transplant recipients, use of LMA within a recovery-oriented perioperative context appeared feasible and was associated with several favorable early postoperative outcomes.
Laryngeal mask airway was feasible in carefully selected adult liver transplant recipients.Laryngeal mask airway increased immediate airway device removal after transplantation.Laryngeal mask airway reduced sore throat and noninvasive ventilation after surgery.Laryngeal mask airway was associated with shorter postoperative hospital stay.