BACKGROUND In recent years,approximately half of the newly diagnosed cases and mortalities attributed to hepatocellular carcinoma(HCC)have been reported in China.Despite the high incidence of HCC,there remains a pauci...BACKGROUND In recent years,approximately half of the newly diagnosed cases and mortalities attributed to hepatocellular carcinoma(HCC)have been reported in China.Despite the high incidence of HCC,there remains a paucity of data regarding the natural growth pattern and the determination of optimal surveillance intervals specific to the Chinese population.AIM To quantify the natural tumor growth pattern of HCC in regional China.METHODS A retrospective analysis was performed on patients from a single institution in Southwest China who had undergone two or more serial dynamic computed tomography or magnetic resonance imaging scans between 2014 and 2020,without having received any anti-cancer therapy.Tumor growth was assessed using tumor volume doubling time(TVDT)and tumor growth rate(TGR),with volumes measured manually by experienced radiologists.Simple univariate linear regression and descriptive analysis were applied to explore associations between growth rates and clinical factors.RESULTS This study identifies the median TVDT for HCC as 163.4 d,interquartile range(IQR)72.1 to 302.3 d,with a daily TGR of 0.42%(IQR 0.206%-0.97%).HCC growth patterns reveal that about one-third of tumors grow indolently with TVDT exceeding 270 d,another one-third of tumors exhibit rapid growth with TVDT under 90 d,and the remaining tumors show intermediate growth rates,with TVDT ranging between 3 to 9 months.CONCLUSION The identified TGRs support biannual surveillance and follow-up for HCC patients in certain regions of China.Given the observed heterogeneity in HCC growth,further investigation is warranted.展开更多
目的回顾性调查基于APP的国产家用盆底康复仪在真实世界中对盆底功能相关症状的改善情况。方法对2019年10月1日至2021年3月31日期间自由注册并按照关联APP居家使用某国产家用盆底康复仪的171000名18岁以上女性用户进行回顾性调查。通过...目的回顾性调查基于APP的国产家用盆底康复仪在真实世界中对盆底功能相关症状的改善情况。方法对2019年10月1日至2021年3月31日期间自由注册并按照关联APP居家使用某国产家用盆底康复仪的171000名18岁以上女性用户进行回顾性调查。通过公共网络平台发布分别针对盆底总体状况、性生活满意度、与松弛型盆底相关之压力性尿失禁(stress urinary incontinence,SUI)、盆腔器官脱垂(pelvic organ prolapse,POP)、阴道松弛(vaginal laxity,VL)共5份患者症状改善印象(Patient Global Impression of Improvement,PGI-I)评分问卷,由用户匿名完成自我疗效评估并上传APP中记录的使用前后阴道肌力值。根据网络后台采集的问卷分值进行疗效评估,并与阴道收缩力作配对分析。结果采用系统抽样法共收集984位用户的有效问卷,分层分析显示:使用时间达到及超过3个月的用户,其盆底总体状况(P=0.0001)、性生活满意度(P=0.009)及SUI(P=0.0001)、POP(P=0.044)、VL(P=0.034)的5项PGI-I评分与不足3个月的用户相比差异均有统计学意义,且SUI、POP的PGI-I分值与阴道肌力间具有配对相关性。而在年龄、教育水平、胎次、分娩方式各亚组中PGI-I评分的差异不具有统计学意义。结论在真实世界中,居家使用3个月及以上基于APP的国产家用盆底康复仪在中国女性人群中可观察到盆底功能相关症状的主观获益及阴道肌力的改善。展开更多
Objective To introduce the design of observational trials under real-world study(RWS),and to provide guidance for clinical development of new research trials and reference for real-world researchers.Methods Relevant l...Objective To introduce the design of observational trials under real-world study(RWS),and to provide guidance for clinical development of new research trials and reference for real-world researchers.Methods Relevant literature of RWS and observational experiments at home and abroad were reviewed and analyzed,and then the design of observational studies was summarized under RWS.Results and Conclusion The data and information provided in observational studies not only help to further verify the clinical study results obtained by randomized controlled trial(RCT)in clinical practice,but also objectively reflect the real situation in the process of clinical research and application.展开更多
近年来,在医药卫生领域,利用真实世界数据生成真实世界证据的研究方法在药品研发、审批、适应证拓展等领域取得广泛的关注和认可,开展真实可信、高质量的真实世界研究证据(real world evidence,RWE),已成为循证医学的一个重要发展方向...近年来,在医药卫生领域,利用真实世界数据生成真实世界证据的研究方法在药品研发、审批、适应证拓展等领域取得广泛的关注和认可,开展真实可信、高质量的真实世界研究证据(real world evidence,RWE),已成为循证医学的一个重要发展方向。本文介绍了近年来国内外关于结直肠癌真实世界研究的现状,将真实世界研究(real world research/study,RWS)与相应的随机对照研究(randomized controlled trial,RCT)的meta分析结果进行对比和评价,以阐述RWS的特征、研究方法、相对于传统RCT研究的优势,并归纳其在肠癌领域的应用现状。在此基础上,对RWS在结直肠癌临床研究中的价值和前景进行总结,为后续研究提供新的思路。展开更多
真实世界研究(real world study,RWS)是指针对预设的临床问题,在真实世界环境下收集与研究对象健康有关的真实世界数据,通过分析获得药物的使用情况及潜在获益/风险的过程,其数据来源于卫生信息系统、医保系统、疾病登记系统和药品不良...真实世界研究(real world study,RWS)是指针对预设的临床问题,在真实世界环境下收集与研究对象健康有关的真实世界数据,通过分析获得药物的使用情况及潜在获益/风险的过程,其数据来源于卫生信息系统、医保系统、疾病登记系统和药品不良反应监测系统等。中药人用经验是2015年药物临床试验数据核查后,中药新药研发陷入低谷背景下,专家通过总结中药新药临床试验中存在问题后提出的新理念,其数据来源包括某些真实世界数据,更多是要收集中药新药处方形成、理法方药和临床应用情况等关键要素。RWS主要是用于药品上市后不良反应监测、上市药品效益评估与医疗、医保决策、特殊医疗器械和特殊药品监管审批决策,与临床随机对照试验是互补的关系。人用经验适用于中药复方制剂研发和增加功能主治,对于临床适应症和目标人群没有特殊规定,人用经验与临床试验是序贯衔接关系,通过总结人用经验为临床试验的设计与实施提供良好的支撑,是中药新药研发重要环节和普遍性方法。RWS关键在真实世界数据与研究结果的相关性和可靠性,要避免“真实世界研究不真实”。人用经验总结关键在于明确中药新药临床定位、适用人群、疗程、用法用量,避免“人用经验只讲临床经验”。在人用经验总结中采取RWS,通常采用试验性研究(pragmatic clinical trial,PCT);中药新药研发中RWS与人用经验是为了回答不同临床问题而产生的不同研究设计,研究者应根据实际情况选择合适方法。展开更多
基金Supported by Cultivate Project for the National Natural Science Foundation of China,No.gyfynsfc[2020]-27and National Natural Science Foundation of China,No.81960328.
文摘BACKGROUND In recent years,approximately half of the newly diagnosed cases and mortalities attributed to hepatocellular carcinoma(HCC)have been reported in China.Despite the high incidence of HCC,there remains a paucity of data regarding the natural growth pattern and the determination of optimal surveillance intervals specific to the Chinese population.AIM To quantify the natural tumor growth pattern of HCC in regional China.METHODS A retrospective analysis was performed on patients from a single institution in Southwest China who had undergone two or more serial dynamic computed tomography or magnetic resonance imaging scans between 2014 and 2020,without having received any anti-cancer therapy.Tumor growth was assessed using tumor volume doubling time(TVDT)and tumor growth rate(TGR),with volumes measured manually by experienced radiologists.Simple univariate linear regression and descriptive analysis were applied to explore associations between growth rates and clinical factors.RESULTS This study identifies the median TVDT for HCC as 163.4 d,interquartile range(IQR)72.1 to 302.3 d,with a daily TGR of 0.42%(IQR 0.206%-0.97%).HCC growth patterns reveal that about one-third of tumors grow indolently with TVDT exceeding 270 d,another one-third of tumors exhibit rapid growth with TVDT under 90 d,and the remaining tumors show intermediate growth rates,with TVDT ranging between 3 to 9 months.CONCLUSION The identified TGRs support biannual surveillance and follow-up for HCC patients in certain regions of China.Given the observed heterogeneity in HCC growth,further investigation is warranted.
文摘目的回顾性调查基于APP的国产家用盆底康复仪在真实世界中对盆底功能相关症状的改善情况。方法对2019年10月1日至2021年3月31日期间自由注册并按照关联APP居家使用某国产家用盆底康复仪的171000名18岁以上女性用户进行回顾性调查。通过公共网络平台发布分别针对盆底总体状况、性生活满意度、与松弛型盆底相关之压力性尿失禁(stress urinary incontinence,SUI)、盆腔器官脱垂(pelvic organ prolapse,POP)、阴道松弛(vaginal laxity,VL)共5份患者症状改善印象(Patient Global Impression of Improvement,PGI-I)评分问卷,由用户匿名完成自我疗效评估并上传APP中记录的使用前后阴道肌力值。根据网络后台采集的问卷分值进行疗效评估,并与阴道收缩力作配对分析。结果采用系统抽样法共收集984位用户的有效问卷,分层分析显示:使用时间达到及超过3个月的用户,其盆底总体状况(P=0.0001)、性生活满意度(P=0.009)及SUI(P=0.0001)、POP(P=0.044)、VL(P=0.034)的5项PGI-I评分与不足3个月的用户相比差异均有统计学意义,且SUI、POP的PGI-I分值与阴道肌力间具有配对相关性。而在年龄、教育水平、胎次、分娩方式各亚组中PGI-I评分的差异不具有统计学意义。结论在真实世界中,居家使用3个月及以上基于APP的国产家用盆底康复仪在中国女性人群中可观察到盆底功能相关症状的主观获益及阴道肌力的改善。
文摘Objective To introduce the design of observational trials under real-world study(RWS),and to provide guidance for clinical development of new research trials and reference for real-world researchers.Methods Relevant literature of RWS and observational experiments at home and abroad were reviewed and analyzed,and then the design of observational studies was summarized under RWS.Results and Conclusion The data and information provided in observational studies not only help to further verify the clinical study results obtained by randomized controlled trial(RCT)in clinical practice,but also objectively reflect the real situation in the process of clinical research and application.
文摘近年来,在医药卫生领域,利用真实世界数据生成真实世界证据的研究方法在药品研发、审批、适应证拓展等领域取得广泛的关注和认可,开展真实可信、高质量的真实世界研究证据(real world evidence,RWE),已成为循证医学的一个重要发展方向。本文介绍了近年来国内外关于结直肠癌真实世界研究的现状,将真实世界研究(real world research/study,RWS)与相应的随机对照研究(randomized controlled trial,RCT)的meta分析结果进行对比和评价,以阐述RWS的特征、研究方法、相对于传统RCT研究的优势,并归纳其在肠癌领域的应用现状。在此基础上,对RWS在结直肠癌临床研究中的价值和前景进行总结,为后续研究提供新的思路。
文摘真实世界研究(real world study,RWS)是指针对预设的临床问题,在真实世界环境下收集与研究对象健康有关的真实世界数据,通过分析获得药物的使用情况及潜在获益/风险的过程,其数据来源于卫生信息系统、医保系统、疾病登记系统和药品不良反应监测系统等。中药人用经验是2015年药物临床试验数据核查后,中药新药研发陷入低谷背景下,专家通过总结中药新药临床试验中存在问题后提出的新理念,其数据来源包括某些真实世界数据,更多是要收集中药新药处方形成、理法方药和临床应用情况等关键要素。RWS主要是用于药品上市后不良反应监测、上市药品效益评估与医疗、医保决策、特殊医疗器械和特殊药品监管审批决策,与临床随机对照试验是互补的关系。人用经验适用于中药复方制剂研发和增加功能主治,对于临床适应症和目标人群没有特殊规定,人用经验与临床试验是序贯衔接关系,通过总结人用经验为临床试验的设计与实施提供良好的支撑,是中药新药研发重要环节和普遍性方法。RWS关键在真实世界数据与研究结果的相关性和可靠性,要避免“真实世界研究不真实”。人用经验总结关键在于明确中药新药临床定位、适用人群、疗程、用法用量,避免“人用经验只讲临床经验”。在人用经验总结中采取RWS,通常采用试验性研究(pragmatic clinical trial,PCT);中药新药研发中RWS与人用经验是为了回答不同临床问题而产生的不同研究设计,研究者应根据实际情况选择合适方法。
