方法与临床研究雷达（2026-09-14）
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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. 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.
8. 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.
9. Sample size calculation for the sequential multiple assignment randomized trial (SMART) design with a skewed outcome: Application to the SMART+ study.
主题：临床试验方法与统计实践
相关性分数：4
期刊：Statistical methods in medical research
公开日期：2026-09-12（电子公开）
期刊卷期日期：2026-Sep-12
内容状态：待评估
为什么值得看：包含可供初筛的摘要；已识别电子公开日期；通过方法学信号门槛；仍需阅读全文评价适用性与证据质量。
With the rapid emergence of personalized healthcare, adaptive interventions have gained significant traction and relevance. Contemporary research has introduced a sophisticated trial design known as the sequential multiple assignment randomized trial (SMART) to advance the development of effective adaptive interventions. A critical element of the SMART design is the determination of the sample size. The existing literature provides sample size calculation formulas for SMART designs, which encompass a variety of approaches and types of outcome data. However, these formulas have primarily been developed under the assumption of normality for the outcome data. In practice, the fields where SMART is employed exhibit a significant prevalence of non-normality in the outcomes of interest. This paper delves into a specific scenario where the outcome demonstrates skewed behavior. We develop precision-based and power-based formulas for skewed outcomes, considering the requirements of both pilot and full-scale SMARTs. We present the operating characteristics of the formulas and perform extensive simulations under various design specifications to validate their usefulness. To demonstrate practical utility, we apply our formulas to the SMART+ study, a digital intervention trial that includes a skewed outcome among its outcomes of interest, highlighting relevance in real-world planning.
10. Clinical and economic impact of bivalent respiratory syncytial virus prefusion F (RSVpreF) vaccination in older adults: a cost-effectiveness analysis for Chile.
主题：因果推断、RWE 与卫生经济学
相关性分数：4
期刊：Expert review of vaccines
公开日期：2026-09-12（电子公开）
期刊卷期日期：2026-Dec
内容状态：待评估
为什么值得看：包含可供初筛的摘要；已识别电子公开日期；通过方法学信号门槛；仍需阅读全文评价适用性与证据质量。
BACKGROUND: Respiratory syncytial virus (RSV) is a major cause of lower respiratory tract disease in older adults in Chile. Chile’s National Immunization Technical Advisory Group (NITAG) recommended RSV vaccination for adults aged ≥60 years with risk factors for severe illness. This study estimated the cost-effectiveness of bivalent RSVpreF vaccination among at-risk older adults in Chile.
RESEARCH DESIGN AND METHODS: A population-based Markov model projected RSV-related outcomes under no vaccination and RSVpreF vaccination over the cohort lifetime. Cost-effectiveness was assessed from healthcare and societal perspectives in 2025 Chilean Pesos (CLP) and US Dollars (US$), with costs and outcomes discounted at 3% annually. Sensitivity analyses tested the model robustness.
RESULTS: RSVpreF vaccination was projected to prevent 14,956 hospitalizations, 50,419 emergency department visits, and 124,849 physician office visits; avert CLP76 billion (US$82 million) in direct medical costs and CLP305 billion (US$328 million) in indirect costs; and generate 7,079 quality-adjusted life-years (QALYs). At a threshold of CLP15,977,348 (US$17,180) per QALY gained, the maximum cost-effective vaccine price was CLP100,480 (US$108) and CLP265,261 (US$285) from the healthcare and societal perspectives respectively.
CONCLUSIONS: RSVpreF vaccination would reduce RSV burden and likely be cost-effective for older adults in Chile.
11. 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.
12. A multi-center, open-labelled, randomized controlled extended phase III non-inferiority clinical trial to evaluate the immunogenicity and tolerability of poliomyelitis vaccine (Vero cells), inactivated, Sabin strains administered with or without routine infant vaccines.
主题：临床试验方法与统计实践
相关性分数：4
期刊：Vaccine
公开日期：2026-08-27（电子公开）
期刊卷期日期：2026-Oct-03
内容状态：待评估
为什么值得看：包含可供初筛的摘要；已识别电子公开日期；通过方法学信号门槛；仍需阅读全文评价适用性与证据质量。
BACKGROUND: Oral poliovirus vaccine (OPV) has been reported to cause vaccine-derived poliovirus and vaccine-associated paralytic poliomyelitis, and the limited global supply of conventional IPV has led many countries to rely on fractional-dose IPV regimens alongside OPV. The current study aims to assess the sIPV safety and immunogenicity when given concurrently with or in a staggered manner with routine immunization.
METHODS: A multi-country, multi-center, open-label, randomized controlled, extended phase III non-inferiority clinical trial was conducted with 1442 healthy infants aged 6-8 weeks from Bangladesh and Pakistan enrolled and randomized into four groups, i.e., co-administration group 1 (group C1), co-administration group 2 (group C2), staggered administration group 1 (group S1) and staggered administration group 2 (group S2). Antibody levels were determined using the collected sera for immunogenicity evaluation. The difference in seroconversion rates between the coadministration group and the staggered administration group is compared using the Cochran-Mantel-Haenszel χ2 (CMH-χ2) test, stratified by study site. Non-inferiority is concluded if the lower bound of the 95% confidence interval (CI) for the rate difference (coadministration group minus staggered administration group) is greater than -10%. The trial was registered prior to patient enrollment at clinicaltrials.gov (NCT05850364), and the protocol and statistical analysis plan are available at https://clinicaltrials.gov/study/NCT05850364. The trial is closed to new participants.
FINDINGS: The post-vaccination seroconversion rates for PV I were 90.3% (306/339) in group C1 and 87.0% (261/300) in group S1, for PV II, 91.7% (311/339) in group C1 and 91.3% (274/300) in group S1 and for PV III, 86.4% (293/339) in group C1 and 92.3% (277/300) in group S1. Among adverse reactions (ARs) reported within 7 days of vaccination, the incidence was similarly high in both the co-administration and staggered vaccination groups (92.8% vs. 95.5%).
INTERPRETATION: Our results demonstrated favorable safety and immunogenicity of co-administration of sIPV with other routine infant vaccines according to a 3-dose primary immunization schedule.
13. Brain-computer interface clinical trial design considerations and clinical outcome assessments in pivotal studies: a summary of the 11th BCI society meeting 2025 workshop.
主题：临床试验方法与统计实践
相关性分数：4
期刊：Journal of neural engineering
公开日期：2026-09-11（电子公开）
期刊卷期日期：2026-Sep-11
内容状态：待评估
为什么值得看：包含可供初筛的摘要；已识别电子公开日期；通过方法学信号门槛；仍需阅读全文评价适用性与证据质量。
Objective.The Brain Computer Interface (BCI) Society Meeting 2025 held a workshop in collaboration with the Implantable BCI Collaborative Community (iBCI-CC) to discuss the selection, development and validation of clinical trial outcome assessments (COAs) for pivotal studies of BCIs. The iBCI-CC Clinical Study Endpoints Workgroup aims to build consensus on a COA framework that can meet the demands of emerging iBCI trials.Approach.The workshop brought together diverse stakeholders from the iBCI-CC community and beyond, including industry, academic and government institutions to engage in pre-competitive collaborative discussion.Main results.Through presentations, panel discussions and breakout groups, workshop participants highlighted meaningful aspects of health (MAHs) relevant to iBCI users, clarified the need to define corresponding concepts of interest (COIs) and to identify or adapt COAs capable of capturing both functional impact and real-world use. Key challenges highlighted during the workshop are patient heterogeneity, the lack of widely validated and fit-for-purpose COAs, and the need to capture meaningful outcomes in home and daily-life environments. Lessons drawn from trials such as ADAPT-PD underscore the value of capturing device performance in home and daily-life settings, highlighting the need for context-sensitive and flexible metrics that reflect patient priorities.Significance.This workshop lays a foundation for the iBCI-CC Clinical Study Endpoints Workgroup to establish a transparent process for identifying MAHs and COIs that are suitable for specific iBCI technologies, patient-informed, and conducive to regulatory approval and reimbursement. By combining rigorous, quantitative assessment with patient-informed goals, iBCI clinical trials can advance toward safe, effective, and accessible neurotechnologies that enhance how a person with a severe motor impairment feels, functions and survives.
14. Transitions in depressive symptom states among adults with subthreshold depression: Associations with lifetime trauma exposure and combined lifestyle in a multistate Markov study.
主题：生存与复杂事件结局方法
相关性分数：4
期刊：Journal of affective disorders
公开日期：2026-08-04（电子公开）
期刊卷期日期：2026-Dec-01
内容状态：待评估
为什么值得看：包含可供初筛的摘要；已识别电子公开日期；通过方法学信号门槛；仍需阅读全文评价适用性与证据质量。
BACKGROUND: Subthreshold depression is common, but whether lifetime trauma exposure and combined lifestyle are differentially associated with symptom transitions remains unclear.
METHODS: We analyzed a prospective community cohort in Shenzhen, China, of adults aged 18-65 years with a Patient Health Questionnaire-9 (PHQ-9) score ≥ 5 and no depressive disorder at baseline. Depressive symptom states (Normal, Mild, and Moderate/Severe) were assessed repeatedly over 36 months. Continuous-time multi-state Markov models estimated transition intensities, probabilities, and hazard ratios (HRs) for lifetime trauma exposure and a five-component combined lifestyle score, adjusting for demographic, socioeconomic, and health-related covariates.
RESULTS: Among 2361 participants (mean age, 37.30 years; 61.9% women), recovery transitions were more frequent than worsening transitions. The monthly Mild-to-Normal intensity was 0.131 (95% CI, 0.100-0.171), 2.47 times the Mild-to-Moderate/Severe intensity; Moderate/Severe-to-Mild was highest (0.205; 95% CI, 0.158-0.272). Lifetime trauma exposure was associated with lower Mild-to-Normal (HR, 0.54; 95% CI, 0.42-0.69) and Moderate/Severe-to-Mild rates (HR, 0.52; 95% CI, 0.41-0.65). Unfavorable lifestyle was associated with a lower Mild-to-Normal rate (HR, 0.70; 95% CI, 0.53-0.92) and a higher Mild-to-Moderate/Severe rate (HR, 1.60; 95% CI, 1.13-2.25). Those with both exposures had the lowest Mild-to-Normal rate (HR, 0.39; 95% CI, 0.26-0.59) and a higher Mild-to-Moderate/Severe rate (HR, 1.69; 95% CI, 1.05-2.73).
CONCLUSIONS: Lifetime trauma exposure was associated with lower recovery rates, whereas an unfavorable lifestyle was associated with lower recovery and higher worsening rates. These associations may inform trauma-informed assessment and lifestyle support for adults with subthreshold depression.
15. Benefit of Linked-Color Imaging in Artificial Intelligence-Assisted Diagnosis of Early Gastric Cancer: A Pilot Study With Propensity Score Adjustment.
主题：因果推断、RWE 与卫生经济学
相关性分数：4
期刊：DEN open
公开日期：2026-07-28（电子公开）
期刊卷期日期：2027-Apr
内容状态：待评估
为什么值得看：包含可供初筛的摘要；已识别电子公开日期；通过方法学信号门槛；仍需阅读全文评价适用性与证据质量。
BACKGROUND AND AIMS: Artificial intelligence (AI)-assisted endoscopy represents a promising approach for lesion detection, yet frequent false-positive detections impair clinical utility by disrupting examinations and diminishing physician confidence. Linked-color imaging (LCI), an image-enhanced endoscopy technique that amplifies mucosal and vascular contrast, may address this limitation. This investigation evaluated whether LCI reduces false-positive AI detections compared with white-light imaging (WLI).
METHODS: This retrospective study analyzed consecutive AI-assisted upper endoscopies performed between March 2024 and June 2025. WLI and LCI were performed sequentially within the same endoscopic session in each patient. False-positive AI detections were compared between modalities using two computer-aided detection (CAD) versions. Propensity score adjustment was used as a sensitivity analysis for baseline differences between CAD Versions I and II.
RESULTS: Of 66 initially screened cases, 63 remained after excluding patients with prior gastric surgery. LCI reduced false-positive AI detections compared with WLI (median 2 vs. 5; p < 0.001). In CAD version-stratified sensitivity analyses, LCI reduced false-positive AI detections in both Version I (5 to 2; p = 0.01) and Version II (2 to 0; p = 0.03). This reduction remained consistent across atrophic grades. Both imaging modalities identified all gastric lesions, achieving 100% detection sensitivity.
CONCLUSIONS: LCI assessment performed after WLI observation yielded fewer false-positive CAD-EYE detections while maintaining lesion detection sensitivity. However, because the observation sequence was fixed, these findings should be interpreted cautiously and require confirmation in prospective or counterbalanced studies.
UNLABELLED: Trial Registration: N/A (retrospective study).
16. Transcutaneous auricular vagus nerve stimulation combined with ciprofol for sedation in patients undergoing same-session bidirectional endoscopy: a randomized, double-blind, placebo-controlled, three-arm non-inferiority trial protocol.
主题：临床试验方法与统计实践
相关性分数：4
期刊：Annals of medicine
公开日期：2026-07-01（电子公开）
期刊卷期日期：2026-Dec
内容状态：待评估
为什么值得看：包含可供初筛的摘要；已识别电子公开日期；通过方法学信号门槛；仍需阅读全文评价适用性与证据质量。
BACKGROUND: Transcutaneous auricular vagus nerve stimulation (taVNS) provides targeted modulation of the autonomic nervous system and descending pain pathways, exerting analgesic potential. However, high-quality evidence remains insufficient to determine whether taVNS can effectively replace opioids in sedation regimens for same-session bidirectional endoscopy while maintaining the quality of early postoperative recovery.
DISCUSSION: This study is a single-center, prospective, randomized, double-blind, placebo-controlled, three-arm non-inferiority trial. A total of 181 patients scheduled for painless same-session bidirectional endoscopy will be enrolled and randomly assigned using dynamic block randomization to one of three groups: Group S (sufentanil 0.1 µg/kg plus sham taVNS), Group T (normal saline plus active taVNS), and Group P (normal saline plus sham taVNS). All participants will receive ciprofol for sedation induction and maintenance. The primary outcome will be the quality of recovery at 24 h postoperatively, assessed using the 15-item Quality of Recovery scale (QoR-15), with a predefined non-inferiority margin (δ) of 6 points, which corresponds to the minimal clinically important difference of the QoR-15 scale. Secondary outcomes will include perioperative adverse events (pre-, intra-, and postoperative, including taVNS-related events), QoR-15 score at 1 h postoperatively, procedural and recovery efficiency indices, sedative dosage, and patient and endoscopist satisfaction scores. Blinding effectiveness will be assessed in all participants, and statistical analyses will follow the modified intention-to-treat principle.
CONCLUSION: This study protocol will rigorously assess the effectiveness and safety of taVNS as an alternative to opioid analgesics for sedation during same-session bidirectional endoscopy using, to our knowledge, the first three-arm design.Trial registration: Chinese Clinical Trial Registry (ChiCTR2600117962).
This study will provide the first systematic evaluation of the feasibility and efficacy of transcutaneous auricular vagus nerve stimulation (taVNS) as an opioid-sparing alternative to sufentanil for ciprofol-based sedation during same-session bidirectional endoscopy.A three-arm randomized design incorporating a prespecified trial-sensitivity analysis will be used to rigorously validate the effectiveness of the standard regimen, enhancing the interpretability and credibility of the non-inferiority conclusion.An “active placebo” taVNS stimulation model combined with an improved standardized questionnaire for blinding assessment will be employed to address the well-recognized limitations of inadequate blinding in taVNS research.
17. Efficacy and safety of ciprofol versus remimazolam combined with alfentanil for sedation during outpatient colonoscopy: a randomized double-blind noninferiority trial.
主题：临床试验方法与统计实践
相关性分数：3
期刊：Brazilian journal of anesthesiology (Elsevier)
公开日期：2026-09-12（电子公开）
期刊卷期日期：2026-Sep-12
内容状态：待评估
为什么值得看：包含可供初筛的摘要；已识别电子公开日期；通过方法学信号门槛；仍需阅读全文评价适用性与证据质量。
INTRODUCTION: Colonoscopy often causes significant discomfort to patients. To improve patient experience, sedation-assisted colonoscopy is being increasingly adopted. This study was designed to assess the efficacy and safety of ciprofol versus remimazolam combined with alfentanil during outpatient colonoscopy.
METHODS: A randomized, double-blind controlled clinical trial was performed among patients scheduled for elective outpatient colonoscopy. Patients were randomly allocated at a 1:1 ratio to two groups: the ciprofol group (Group C, 0.4 mg.kg-1 intravenously, n = 88) and the remimazolam group (Group R, 0.3 mg.kg-1 intravenously, n = 88). Intravenous alfentanil (3 μg.kg-1) was administered to all patients in conjunction with the study drug. The primary outcome was the success rate of colonoscopy. Secondary outcomes included induction time, discharge time, satisfaction levels (patients and endoscopists), and adverse events.
RESULTS: In the ITT population, colonoscopy success rates were 100.0% (88/88) in Group C and 98.9% (87/88) in Group R (treatment difference [95% CI]: 0.011 [-0.024 to 0.048]). The lower bound of the two-sided 95% CI was above the prespecified noninferiority margin of -0.08, confirming the noninferiority of ciprofol relative to remimazolam. After Bonferroni correction (adjusted threshold p < 0.0028), discharge time was longer in Group C (8.5 [6.0‒12.0] vs. 7.0 [5.0‒9.0] minutes; p = 0.002). Group R showed numerically higher MAP and HR at several time points.
CONCLUSIONS: This study demonstrated that intravenous ciprofol (0.4 mg.kg-1) or remimazolam (0.3 mg.kg-1), combined with alfentanil (3 μg.kg-1), provided effective sedation for outpatient colonoscopy.
18. Mitochondrial Function-Related Genes in Sleep Disorders: A Multi-Omics Mendelian Randomization Study.
主题：临床试验方法与统计实践
相关性分数：3
期刊：Journal of molecular neuroscience : MN
公开日期：2026-09-12（电子公开）
期刊卷期日期：2026-Sep-12
内容状态：待评估
为什么值得看：包含可供初筛的摘要；已识别电子公开日期；通过方法学信号门槛；仍需阅读全文评价适用性与证据质量。
Mitochondrial dysfunction is linked to sleep disorders in previous report, but the potential roles of specific genes remain unclear. This study aimed to dissect different subtype-specific genetic associations and their underlying mechanisms. A multi-omics Summary-data-based Mendelian Randomization (SMR) approach was performed to identify potential causal links between mitochondrial function-related genes and sleep disorders. We integrated GWAS data from FinnGen database (the discovery set), independent GWAS datasets (covering different sleep-disorder subtypes and used for validation), and cis-QTLs (including mQTLs, eQTLs, and pQTLs) to perform systematic exploration. Specially, we performed targeted validation of tissue-specific effects, leveraging gene expression data from disease-relevant brain regions within the GTEx database. Our SMR analysis identified mitochondrial function-related genes potentially modulating sleep disorders across biological layers, initially identifying 102 genes at the methylation level, 48 at the gene expression level, and 6 at the protein abundance level. Integrative analysis subsequently prioritized DCXR and ACADVL and revealed their distinct, subtype-specific associations. DCXR exhibited a protective role in sleep apnea while ACADVL showed a paradoxical risk conferring role in daytime sleepiness. In addition, the analysis identified an epigenetic regulatory mechanism for DCXR in which its expression and protein levels are modulated by DNA methylation. Finally, validation in brain-hypothalamus tissue confirmed DCXR as a significant potential protective factor (OR = 0.929, 95% CI: 0.887-0.973, P_HEIDI = 0.999, FDR = 0.2449). Our findings implicate key mitochondrial genes, particularly DCXR and ACADVL, in the pathophysiology of specific sleep disorder subtypes, highlighting potential avenues for precision medicine. Clinical trial number: Not applicable.
19. Improving Clinical Trial Design in Low Back Pain: Insights from Studies of Gabapentinoids.
主题：临床试验方法与统计实践
相关性分数：3
期刊：Pain and therapy
公开日期：2026-09-12（电子公开）
期刊卷期日期：2026-Sep-12
内容状态：待评估
为什么值得看：包含可供初筛的摘要；已识别电子公开日期；通过方法学信号门槛；仍需阅读全文评价适用性与证据质量。
Low back pain (LBP) is one of the leading causes of disability worldwide, affecting hundreds of millions of people globally. Scientific literature continues to highlight key issues in managing these patients, including accuracy of diagnostic terminology, identification of the specific pain mechanisms, and lack of effective licensed treatment options. Using published clinical trials of gabapentinoids to provide insights, we discuss key methodological and clinical factors, such as the accurate use of terminology, appropriate patient selection, effective application of screening tools, and mechanism-based selection of medicines at therapeutic doses. It is important to select a patient population appropriately for the specific pain mechanism treated by the medicine under assessment. Current evidence suggests that identification of a neuropathic component in clinical trials is often inadequate, with many studies either failing to use available validated screening tools or applying them without sufficient methodological rigor. Alongside this, spontaneous resolution of acute pain in low back patients remains an important confounder that is often not thoroughly accounted for. Patients with LBP represent a highly heterogeneous group, and the potential disconnect between diagnosis and mechanism is not helped by older nonspecific terminology. The clinical community continues to improve the terminology, such as the recent International Association for the Study of Pain (IASP) definition of “spine-related leg pain” updating the older diagnosis of “sciatica.” Defining or further stratifying a patient population (for example, using neuropathic screening tool scores) and then evaluating multiple treatments effective for nociceptive or neuropathic pain may improve a study’s discriminatory power while reducing the number of patients required. Once initial drug efficacy has been established in randomized, placebo-controlled trials, additional study designs targeting specific patient populations can still provide valuable insight into key clinical questions, often requiring substantially fewer resources.
20. Racial and ethnic survival disparities in primary liver cancer in the United States: a National Cancer Database propensity score-matched study.
主题：因果推断、RWE 与卫生经济学
相关性分数：3
期刊：Hepatic oncology
公开日期：2026-09-12（电子公开）
期刊卷期日期：2026-Dec
内容状态：待评估
为什么值得看：包含可供初筛的摘要；已识别电子公开日期；通过方法学信号门槛；仍需阅读全文评价适用性与证据质量。
INTRODUCTION: Primary liver cancer is a leading cause of cancer mortality worldwide. We evaluated whether racial and ethnic survival disparities among US patients with primary liver cancer persist after adjustment for clinical, treatment, socioeconomic, and access-related factors.
METHODS: We performed a retrospective cohort study using the National Cancer Database. Race was analyzed as White versus Black, and race/ethnicity groups were defined as non-Hispanic White (NHW), non-Hispanic Black (NHB), and Hispanic of any race. Overall survival (OS) was assessed using Kaplan-Meier analysis, log-rank testing, multivariable Cox regression, and 1:1 propensity score matching for the White-versus-Black comparison.
RESULTS: Among 122,049 patients diagnosed between 2004 and 2016, Black patients more often presented with advanced disease and were less likely to receive surgery, chemotherapy, or radiotherapy. In the unmatched analysis, White patients had better OS. After matching, race was not an independent predictor of survival (HR 0.98, p = 0.11). Hispanic ethnicity remained associated with improved survival compared with NHW and NHB patients (HR 1.32 and 1.29; both p < 0.001).
CONCLUSION: The unadjusted survival difference between White and Black patients was attenuated after adjustment for clinical, treatment, socioeconomic, and access-related factors. Hispanic ethnicity remained associated with improved survival in secondary analyses, warranting further investigation.
This National Cancer Database study included 122,049 adults with primary liver cancer diagnosed between 2004 and 2016.Black patients presented more often with advanced disease, received surgery, chemotherapy, and radiotherapy less often, and had worse unadjusted overall survival than White patients.After multivariable adjustment and 1:1 propensity score matching of 20,680 White and 20,680 Black patients, race was not independently associated with overall survival (HR 0.98, p = 0.11).Hispanic ethnicity remained associated with longer overall survival than non-Hispanic White and non-Hispanic Black groups in secondary analyses; these findings should be interpreted cautiously because the matching model was designed for the White–Black comparison.The attenuation of the White–Black survival difference after adjustment highlights measured clinical, treatment, socioeconomic, healthcare-access, and facility-level factors as important potential targets for reducing disparities.