Innovative Thinking on Endpoint Selection in Clinical Trials

ABSTRACT

In clinical trials, selection of appropriate study endpoints is critical for an accurate and reliable evaluation of safety and effectiveness of a test treatment under investigation. In practice, however, there are usually multiple endpoints available for measurement of disease status and/or therapeutic effect of the test treatment under study. For example, in cancer clinical trials, overall survival, response rate, and/or time to disease progression are usually considered as primary clinical endpoints for evaluation of safety and effectiveness of the test treatment under investigation. Once the study endpoints have been selected, sample size required for achieving a desired power is then determined. It, however, should be noted that different study endpoints may result in different sample sizes. In practice, it is usually not clear which study endpoint can best inform the disease status and measure the treatment effect. Moreover, different study endpoints may not translate one another although they may be highly correlated one another. In this article, we intend to develop an innovative endpoint namely therapeutic index based on a utility function to combine and utilize information collected from all study endpoints. Statistical properties and performances of the proposed therapeutic index are evaluated theoretically. A numerical example concerning a cancer clinical trial is given to illustrate the use of the proposed therapeutic index.     

《艾滋病诊疗指南第三版(2015版)》更新解读

2015年中华医学会感染病学分会艾滋病学组发布了第三版《艾滋病诊疗指南》。新版指南强调抗病毒治疗时点前移:一旦成人确诊感染人类免疫缺陷病毒(HIV), 若无禁忌宜尽早启动抗HIV治疗。对于合并机会性感染的HIV感染者, 在感染控制、病情稳定后也应及早开始抗病毒治疗。尤其强调HIV合并结核患者在CD4阳性淋巴细胞数少于200/μL的情况下, 建议抗结核两周内即开始抗病毒治疗。在抗HIV治疗用药中, 淘汰了一些毒副作用大、依从性较差的药物, 如司他夫定、去羟肌苷、茚地那韦等, 优选抗病毒效力强、服药方便的组合, 如拉米夫定、替诺福韦、依非韦伦组合。对于HIV感染的婴幼儿, 亦主张及早抗HIV治疗。对于五岁以内的幼儿, 主张确诊后即启动抗病毒治疗。对于HIV感染的孕产妇, 建议尽快予以全程、联合抗HIV治疗, 寓防于治。     

腹腔镜与开腹手术治疗老年胆囊炎患者的分析

目的观察在治疗老年急性胆囊炎患者采取开腹手术与腹腔镜手术的治疗效果并分析。方法将2017年1月—2018年9月期间医院收治的74例急性胆囊炎患者采用随机数字表法分为观察组和对照组,对照组37例采取开腹手术治疗,观察组37例采取腹腔镜治疗,对比两组治疗效果。结果观察组并发症发生率5.4%、治疗总有效率97.3%优于对照组的24.3%、78.4%,P<0.05。观察组术中出血量(15.7±3.4)mL、手术时间(40.7±4.4)min、住院时间(7.1±1.8)d、术后肠道恢复时间(3.3±1.2)d均低于对照组的(46.4±3.5)mL、(68.6±14.7)min、(12.6±2.3)d、(6.1±1.5)d,P<0.05。观察组躯体疼痛(87.3±0.5)分、总体健康(85.2±1.2)分、精神状况(86.4±1.3)分、社会活动评分(90.2±1.3)分均高于于对照组(78.3±1.2)分、(70.3±10.8)分、(71.3±0.5)分、(75.5±2.1)分,对比有统计学意义(P<0.05)。结论腹腔镜手术治疗老年急性胆囊炎患者效果良好,可有效减少患者病程,促进康复和提高生活质量,值得推广。     

Electronic clinical records in primary care for estimating disease burden and management. An example of COPD

Chronic obstructive pulmonary disease (COPD) is a significant health problem in developed countries. We aimed to estimate the prevalence of COPD in a single Spanish healthcare area. We also aimed to assess if there are any differences in prevalence and spirometry use among primary care services by utilizing already registered information. We designed a cross-sectional study to determine the prevalence of COPD and the performance of spirometries in each primary care service. A total of 8,444 patients were diagnosed with COPD, with a prevalence of 2.6% for individuals older than 39 years. The prevalence increased with age and was much higher in men. Significant heterogeneity was found in the prevalence of COPD and spirometry use among primary care services. COPD was underdiagnosed and there was wide variability in spirometry use in our area. Greater efforts are needed to diagnose COPD in order to improve its clinical outcomes and to refine registries so that they can be used as reliable sources of information     

微波配合序贯扩肛治疗陈旧性肛裂患者的临床分析

目的:探讨微波配合序贯扩肛治疗陈旧性肛裂的临床疗效。方法:选取陈旧性肛裂患者78例,随机分为对照组和治疗组,每组各39例。对照组患者采用后卫内括约肌部分切断治疗,治疗组患者采用微波配合序贯扩肛治疗。结果:观察组患者术中出血量、切口愈合时间均少于对照组(P<0.05),并发症发生率低于对照组(P<0.05)。结论:采用微波配合序贯扩肛治疗陈旧性肛裂,临床疗效显著。