方法与临床研究雷达（2026-09-12）
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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. Sample-size justification, differential post-randomization exclusion, and multiplicity in a randomized trial of epidural versus wound catheter analgesia after pancreatoduodenectomy.
主题：临床试验方法与统计实践
相关性分数：6
期刊：Pancreatology : official journal of the International Association of Pancreatology (IAP) … [et al.]
公开日期：2026-09-09（电子公开）
期刊卷期日期：2026-Sep-09
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。
3. 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.
4. 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.
5. 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.
6. 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.
7. Covariate-adaptive randomization with dropouts.
主题：临床试验方法与统计实践
相关性分数：5
期刊：Statistical methods in medical research
公开日期：2026-09-10（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。
While clinical trials are often regarded as the gold standard by medical researchers and regulatory agencies, the statistical properties of typical randomized controlled trials heavily depend on large sample sizes, which may not always be achievable due to budget constraints or other practical limitations. To address this issue, adaptive designs have gained increasing attention since the 1970s. These designs incorporate modifications in trial procedures or statistical methodologies, aiming to fulfill study objectives more efficiently using smaller sample sizes and shorter durations compared to traditional clinical trials. Specifically, covariate-adaptive designs are developed to reduce the risk of significant covariate imbalances among treatment groups, particularly in studies with limited sample sizes. Additionally, patient dropout during trials significantly impacts statistical inference regarding treatment effects and diminishes statistical power. Ignoring dropout events can result in undesirable participant allocation and substantial treatment imbalances. In this article, we propose novel strategies that explicitly incorporate dropout information into covariate-adaptive randomization. We thoroughly investigate the statistical properties of covariate-adaptive designs adjusted for dropouts. Extensive simulation studies highlight the advantages of the proposed approaches. Our research lays a robust foundation for future developments in covariate-adaptive randomization, specifically addressing dropout challenges.
8. Cost-effectiveness analysis of elranatamab versus physician’s choice of treatment (non-BCMA-directed regimens) in patients with triple class exposed multiple myeloma in Japan.
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：2026-09-08（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。
AIM: To evaluate the cost-effectiveness of elranatamab versus physician’s choice of treatment (PCT) for patients with triple class exposed multiple myeloma in Japan.
MATERIALS AND METHODS: A cost-effectiveness analysis was conducted comparing elranatamab with PCT using a partitioned survival model with three health states from the Japanese public healthcare payer perspective. A weekly cycle length and a 25-year lifetime horizon were applied, with costs and health outcomes discounted at 2% annually in accordance with Japanese HTA guidelines. Clinical inputs for elranatamab were derived from the phase 2 MagnetisMM-3 trial. Comparative effectiveness versus PCT was estimated using an unanchored matching-adjusted indirect comparison (MAIC) with the prospective real-world LocoMMotion study. Utility values were calculated by applying the Japanese value set to EQ-5D-5L data collected in MagnetisMM-3. Scenario analyses were performed using MAIC-based comparisons versus teclistamab and idecabtagene vicleucel (ide-cel).
RESULTS: In the base-case analysis, elranatamab increased quality-adjusted life-years (QALYs) compared with PCT (2.59 vs 0.82) at a higher total cost (JPY 36,901,695 (USD 246,570) vs 30,899,696 (USD 206,466)), resulting in an incremental cost of JPY 6,001,999 (USD 40,104) and an incremental QALY gain of 1.77. The incremental cost-effectiveness ratio (ICER) was JPY 3,394,966 (USD 22,684) per QALY, remaining within cost-effectiveness thresholds in Japan. Sensitivity analyses demonstrated the robustness of the base-case results. In scenario analyses, elranatamab was dominant versus teclistamab and ide-cel.
LIMITATIONS: Limitations include reliance on the clinical opinion of a single hematology expert for Japanese clinical practice patterns, extrapolation beyond observed trial follow-up, and inherent constraints of MAIC due to the lack of head-to-head trials.
CONCLUSION: From the healthcare payer perspective, elranatamab was cost-effective versus PCT in the base case and scenario analyses, with ICERs consistently below commonly accepted thresholds in Japan; contingent on the unanchored MAIC and long-term extrapolation, this should be interpreted with caution.
9. 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.
10. Ultrasound-Guided Infraspinatus-Teres Minor Interfascial Block versus Interscalene Brachial Plexus Block for Analgesia After Arthroscopic Shoulder Surgery: A Randomized 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：Drug design, development and therapy
公开日期：2026-09-03（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。
BACKGROUND: This randomized non-inferiority trial was designed to verify whether ultrasound-guided infraspinatus-teres minor interfascial block (ITMB) could produce non-inferior postoperative analgesia in adult patients undergoing arthroscopic shoulder surgery compared with ultrasound-guided interscalene brachial plexus block (ISB), while also comparing the early respiratory safety profiles and postoperative adverse events of the two techniques.
METHODS: This trial adopted a two-arm parallel-group design with blinding for participants and all outcome assessors. A total of 82 eligible patients were randomly assigned 1:1 to receive either ITMB (n=41) or ISB (n=41) with 25 mL 0.375% ropivacaine for each nerve block. Two participants in the ITMB group were excluded from the per-protocol (PP) analysis due to intraoperative conversion to open surgery and unplanned intensive care unit admission, leaving 39 ITMB patients and 41 ISB patients for primary PP analysis; intention-to-treat (ITT) analysis covering all 82 randomized patients was additionally conducted to validate robustness. The primary prespecified outcome was 24-hour postoperative total oxycodone consumption, with a predefined non-inferiority margin of -5 mg (ISB minus ITMB). Non-inferiority was established if the lower limit of the 95% confidence interval (CI) for the between-group mean difference was no less than -5 mg. Secondary endpoints comprised the maximum 11-point resting NRS pain score within 24 h, incidence of rebound pain, and rescue analgesia requirements. Safety endpoints included dyspnea, diaphragmatic paralysis, Horner’s syndrome, and postoperative nausea and vomiting.
RESULTS: PP analysis showed the mean 24-hour oxycodone consumption was 11.26 (SD 4.38) mg in the ISB group (95% CI 9.89 to 12.65) versus 10.92 (SD 3.48) mg in the ITMB group (95% CI 9.79 to 12.05), with a between-group mean difference of 0.34 mg (95% CI -1.42 to 2.11, P<0.001). The lower bound of the 95% CI (-1.42 mg) substantially exceeded the pre-specified non-inferiority margin of -5 mg, satisfying the non-inferiority criterion (one-sided non-inferiority P<0.001). ITT analysis yielded consistent non-inferiority results (mean difference 0.26 mg, 95% CI -1.46 to 1.99, P<0.001). For secondary pain outcomes, the median worst resting NRS score within 24 h was significantly lower in the ITMB group [3.0 (IQR 3.0-4.0)] than the ISB group [4.0 (IQR 3.0-7.0), median difference 1.0, 95% CI 0 to 1.0, P<0.001]. ITMB also brought significantly lower rebound pain rate (2.6% vs 26.8%, P=0.006) and less frequent rescue analgesia requirements (7.7% vs 29.3%, P=0.016). Safety outcomes revealed significantly lower early hemidiaphragmatic paralysis and dyspnea in the ITMB group: hemidiaphragmatic paralysis occurred in only 2.6% of ITMB patients vs 90.2% of ISB patients (P<0.001); dyspnea incidence was 2.6% (ITMB) vs 19.5% (ISB, P=0.016); Horner’s syndrome was absent in the ITMB group while occurring in 29% of ISB patients (P<0.001). Postoperative nausea and vomiting rates were comparable between two groups (P=0.655). No severe block-related complications such as nerve injury, local anesthetic systemic toxicity or pneumothorax were observed in either group.
CONCLUSION: Ultrasound-guided ITMB provided non-inferior 24-hour postoperative opioid analgesia compared with ISB and reduced the incidence of early hemidiaphragmatic paralysis in patients undergoing arthroscopic shoulder surgery. The comparable total 24-hour oxycodone consumption reflected distinct time-dependent analgesic profiles, with ISB providing greater analgesic benefit during the early postoperative period, whereas ITMB demonstrated more sustained analgesic effects during the later postoperative period.
TRIAL REGISTRATION: This trial was registered at the Chinese Clinical Trial Registry (ChiCTR2400084716). Date of registration: May 23, 2024.
11. 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).
12. [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。.
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. Computational surveillance of Colombian public pharmaceutical procurement using public administrative data: A reproducible analysis of a closed 2020-2025 cohort.
主题：可复现研究与开放科学
相关性分数：3
期刊：PloS one
公开日期：2026-09-11（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。
INTRODUCTION: BigLoI monitors Colombian public pharmaceutical procurement from 2015 onward. For this manuscript, the source cohort comprised 162,271 candidate pharmaceutical contracts from 2020 to 2025. After contract-level review excluded 441 records explicitly concerning veterinary, animal-health, or agricultural use, the corrected closed analytical cohort comprised 161,830 contracts.
OBJECTIVE: To describe the design, implementation, and findings of a reproducible computational infrastructure for surveillance of Colombian public pharmaceutical procurement.
METHODS: Public APIs from SECOP-II, INVIMA, and SISMED were integrated into a reproducible architecture combining PostgreSQL, statistical analysis, and a public-facing observatory. Candidate records whose supplier names indicated possible veterinary or agricultural activity underwent contract-object review; 441 records with explicit animal, veterinary, or agricultural scope were excluded, while human-health and ambiguous records were retained conservatively. A Z-score engine was implemented to flag contracts with atypical total values within therapeutic categories. As a clearly secondary technical-feasibility module, smart-contract automation of a payment workflow was tested on the Sepolia testnet only to verify predefined digital state transitions under simulated conditions. The corrected closed analytical cohort included 161,830 pharmaceutical contracts from 2020 to 2025, while post-2025 records remained available only for live platform monitoring. Monetary results were reported primarily in COP; secondary USD equivalents were included only as approximate interpretive references, with detailed conversions relegated to S1 Table.
RESULTS: Among 146,594 contracts analyzed in categories with at least 10 observations, 664 contracts (0.45%) triggered a statistical alert with absolute Z-score greater than or equal to 1.5 sigma. The alert rate among Z-score-eligible contracts increased from 0.30% in 2021 to 1.31% in 2025. Antibiotics showed a category-level maximum Z-score of 8.89. The top 3% of suppliers concentrated 85.8% of total contracted value. As a clearly secondary module, the Sepolia smart-contract prototype confirmed only that predefined digital state transitions could be executed under simulated testnet conditions; it provides no evidence of real-world payment-cycle reduction, realized savings, or institutional deployability.
CONCLUSIONS: A reproducible national-scale computational infrastructure identified atypical procurement patterns and documented marked market concentration in Colombian public pharmaceutical procurement. These descriptive findings may inform auditing, public health policy discussions, and health data governance, but they do not by themselves establish wrongdoing or prescribe specific reforms. Statistical alerts remain exploratory prioritization signals rather than evidence of corruption or fraud. The blockchain module should be interpreted strictly as a complementary technical proof of concept tested under simulated conditions and not as operational evidence on real-world payment performance, savings, or implementation readiness.
18. Advantages of a Two-Stage Randomized Trial Design to Evaluate Antimicrobial Treatment Strategies: a Simulation Study.
主题：临床试验方法与统计实践
相关性分数：3
期刊：American journal of epidemiology
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。
Antimicrobial prescribing policies affect treated patients and, through altered transmission, their contacts. Two-stage randomized (2SR) designs can estimate these spillover effects but have not been applied to antimicrobial strategies. We built a stochastic agent-based model of a hospital ward with two competing strains (susceptible and resistant) to emulate a 2SR trial. Drug A covers only the susceptible strain, while Drug B covers both. Six clusters were randomized to a 90/10 or 50/50 Drug A/Drug B allocation, with individuals randomized to treatments within clusters. We estimated direct, indirect, total, and overall effects on mortality. Drug A recipients had higher mortality than Drug B recipients in every cluster, because Drug A does not cover the resistant strain (direct effect +4.75 to +7.08 percentage points [pp]). Among Drug A recipients, mortality was higher when the ward followed 90/10 than 50/50 (indirect effect +2.10 pp), driven by higher resistant-strain prevalence; the effect on Drug B recipients was minimal. The 50/50 strategy reduced total mortality (overall effect -4.00 pp), but this concealed a redistribution: resistant-strain deaths fell while susceptible-strain deaths rose. Findings were robust across sensitivity scenarios. The 2SR design captures spillover effects invisible to individually randomized trials.
19. ‘Methodological considerations regarding propensity score matching and interpretation of oncologic outcomes in ultrasound-guided versus CT-guided microwave ablation for liver metastases’.
主题：因果推断、RWE 与卫生经济学
相关性分数：3
期刊：International journal of hyperthermia : the official journal of European Society for Hyperthermic Oncology, North American Hyperthermia Group
公开日期：2026-09-11（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：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。
20. Efficacy and Safety of an Insulin Patch Pump Compared to Multiple Daily Injections in Pediatric Patients With Diabetes: A Randomized, Open-Label, Crossover, Non-Inferiority Clinical Trial.
主题：临床试验方法与统计实践
相关性分数：3
期刊：Clinical medicine insights. Endocrinology and diabetes
公开日期：2026-09-07（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。
BACKGROUND: Insulin patch pumps offer advantages over multiple daily injections (MDI) for pediatric diabetes, but randomized trial evidence in Chinese children remains limited.
OBJECTIVES: To evaluate the efficacy and safety of a tubeless insulin patch pump (Equil™) compared with MDI in children and adolescents with diabetes.
METHODS: This multicenter, open-label, randomized, crossover, non-inferiority trial was conducted at seven pediatric endocrinology centers in China (September 2021 - October 2022). Patients aged 3-17 years with type 1 diabetes mellitus (T1DM) or type 2 diabetes mellitus (T2DM) were randomized 1:1 to receive continuous subcutaneous insulin infusion (CSII) via Equil™ or MDI via insulin pen for 5 days, followed by crossover. The primary outcome was mean blood glucose (MBG). Non-inferiority margin was pre-specified as 0.386 mmol/L. Secondary outcomes included glycemic variability (standard deviation of blood glucose, SDBG), glycated albumin (GA), total daily insulin dose (TDD), hypoglycemia frequency, and adverse events. Patient satisfaction was assessed using a study-specific 11-item questionnaire.
RESULTS: Of 74 enrolled patients, 72 (97.3%) completed the study. CSII demonstrated non-inferiority to MDI for MBG (8.43±1.88 mmol/L, n=74, vs 9.00±2.04 mmol/L, n=72); mean difference -0.606 mmol/L; 95% CI -0.988 to -0.223; upper CI limit -0.223 < non-inferiority margin 0.386. No significant differences were observed for secondary outcomes (SDBG: 3.019 vs 3.357; GA change: 2.52±3.03% vs 2.02±3.25%, p=0.184; TDD: 132.17±78.43 vs 147.52±91.59 IU, p=0.159; hypoglycemia events: 114 vs 106). Adverse event rates were similar (75.7% vs 69.4%, p=0.399); no serious adverse events occurred. Patient satisfaction was significantly higher with CSII (total score 21.03±4.47 vs 22.00±3.99; mean difference -0.97, 95% CI -1.75 to -0.19; p=0.015), with 68.1% of patients preferring the patch pump.
CONCLUSION: The Equil™ patch pump is non-inferior to MDI in terms of glycemic control and safety for pediatric diabetes patients, with higher patient satisfaction. These findings support the patch pump as an effective alternative for children and adolescents requiring exogenous insulin therapy.
This study compared two ways of giving insulin to children and teenagers with diabetes: an insulin patch pump (a small device that sticks to the skin and delivers insulin continuously) and daily insulin shots (multiple daily injections, or MDI). A total of 74 children and teenagers (aged 3–17 years) from seven hospitals in China took part. Each participant used both methods for 5 days each. The study found that the patch pump worked just as well as daily shots for controlling blood sugar levels. There were no major safety concerns with either method. Patients reported higher satisfaction with the patch pump, especially regarding flexibility and daily life impact, and 68 out of every 100 participants preferred the patch pump over daily shots.