目的介绍基于限制性平均生存时间(restricted mean survival time,RMST)的思想构建非比例风险模型及其SAS软件实现。方法以肺癌临床试验数据为实例,先进行比例风险假定的检验,再利用LIFETEST、RMSTREG在非比例风险生存数据模型下进行分...目的介绍基于限制性平均生存时间(restricted mean survival time,RMST)的思想构建非比例风险模型及其SAS软件实现。方法以肺癌临床试验数据为实例,先进行比例风险假定的检验,再利用LIFETEST、RMSTREG在非比例风险生存数据模型下进行分析。结果通过LIFETEST程序可对RMST进行组间非参数比较,RMSTREG程序可拟合线性或对数RMST回归模型,旨在研究RMST和自变量之间的关系,从而获得组间比较的预期寿命差或预期寿命比的统计效应量。结论当比例风险假定不成立时,风险比(hazard ratio,HR)难解释其临床意义,RMST可以作为HR的替代方法之一。基于RMST统计量的假设检验与统计效应量为非比例风险的生存数据提供了一种统计分析方法。展开更多
Background:The vast majority of patients with cholangiocarcinoma(CC)have advanced disease at diagnosis and are candidates for palliative treatment only.The robustness of the randomized controlled trials regarding the ...Background:The vast majority of patients with cholangiocarcinoma(CC)have advanced disease at diagnosis and are candidates for palliative treatment only.The robustness of the randomized controlled trials regarding the treatment of CC are assessed.Methods:A systematic review of all randomized control trials(RCT)of treatments for both intra-and extrahepatic CC between 2010 and 2020 was performed.The survival-inferred fragility index(SIFI;the minimum number of reassignments of the best survivors between arms that would overturn the statistical outcomes)was calculated.In addition,the gain,or loss,in survival in RCTs was evaluated by the restricted mean survival time(RMST)difference.Finally,the level of spin i.e.,misrepresentation of study outcomes,was measured in inconclusive studies to assess distorted reporting strategies.Results:Out of 6,167 studies retrieved,11 could be retained for full text revision(7 with both intra-and extrahepatic CC,3 with peri-hilar CC,and 1 with peri-hilar or distal CC).Only 3 studies included resected patients(2 with both intra-and extrahepatic CC and 1 with peri-hilar or distal CC).Nine studies investigated systemic chemotherapy(including 3 after surgical resection),one study evaluated photodynamic therapy,and another investigated the use of an endoscopically inserted stent in the biliary tract.The median SIFI was−2[interquartile range(IQR):−6.25,−0.25]across all studies.Overall,the median RMST difference was 0.56 months(IQR:0.10,0.95).Finally,for inconclusive studies,the level of spin was high,moderate,and low in respectively 12.5%,25%,and 62.5% of the studies.Conclusions:RCTs of CC showed a low degree of robustness with a frequent proportion of associated spin.展开更多
文摘目的介绍基于限制性平均生存时间(restricted mean survival time,RMST)的思想构建非比例风险模型及其SAS软件实现。方法以肺癌临床试验数据为实例,先进行比例风险假定的检验,再利用LIFETEST、RMSTREG在非比例风险生存数据模型下进行分析。结果通过LIFETEST程序可对RMST进行组间非参数比较,RMSTREG程序可拟合线性或对数RMST回归模型,旨在研究RMST和自变量之间的关系,从而获得组间比较的预期寿命差或预期寿命比的统计效应量。结论当比例风险假定不成立时,风险比(hazard ratio,HR)难解释其临床意义,RMST可以作为HR的替代方法之一。基于RMST统计量的假设检验与统计效应量为非比例风险的生存数据提供了一种统计分析方法。
文摘Background:The vast majority of patients with cholangiocarcinoma(CC)have advanced disease at diagnosis and are candidates for palliative treatment only.The robustness of the randomized controlled trials regarding the treatment of CC are assessed.Methods:A systematic review of all randomized control trials(RCT)of treatments for both intra-and extrahepatic CC between 2010 and 2020 was performed.The survival-inferred fragility index(SIFI;the minimum number of reassignments of the best survivors between arms that would overturn the statistical outcomes)was calculated.In addition,the gain,or loss,in survival in RCTs was evaluated by the restricted mean survival time(RMST)difference.Finally,the level of spin i.e.,misrepresentation of study outcomes,was measured in inconclusive studies to assess distorted reporting strategies.Results:Out of 6,167 studies retrieved,11 could be retained for full text revision(7 with both intra-and extrahepatic CC,3 with peri-hilar CC,and 1 with peri-hilar or distal CC).Only 3 studies included resected patients(2 with both intra-and extrahepatic CC and 1 with peri-hilar or distal CC).Nine studies investigated systemic chemotherapy(including 3 after surgical resection),one study evaluated photodynamic therapy,and another investigated the use of an endoscopically inserted stent in the biliary tract.The median SIFI was−2[interquartile range(IQR):−6.25,−0.25]across all studies.Overall,the median RMST difference was 0.56 months(IQR:0.10,0.95).Finally,for inconclusive studies,the level of spin was high,moderate,and low in respectively 12.5%,25%,and 62.5% of the studies.Conclusions:RCTs of CC showed a low degree of robustness with a frequent proportion of associated spin.
