{"title":"用不完全重复有序数据评估参数估计的不确定性","authors":"Claudio J. Verzilli, J. Carpenter","doi":"10.1191/1471082x02st033oa","DOIUrl":null,"url":null,"abstract":"Data collected in clinical trials involving follow-up of patients over a period of time will almost inevitably be incomplete. Patients will fail to turn up at some of the intended measurement times or will not complete the study, giving rise to various patterns of missingness. In these circumstances, the validity of the conclusions drawn from an analysis of available cases depends crucially on the mechanism driving the missing data process; this in turn cannot be known for certain. For incomplete categorical data, various authors have recently proposed taking into account in a systematic way the ignorance caused by incomplete data. In particular, the idea of intervals of ignorance has been introduced, whereby point estimates for parameters of interest are replaced by intervals or regions of ignorance (Vansteelandt and Goetghebeur, 2001; Kenward et al., 2001; Molenberghs et al., 2001). These are identified by the set of estimates corresponding to possible outcomes for the missing data under little or no assumptions about the missing data mechanism. Here we extend this idea to incomplete repeated ordinal data. We describe a modified version of standard algorithms used for fitting marginal models to longitudinal categorical data, which enables calculation of intervals of ignorance for the parameters of interest. The ideas are illustrated using dental pain measurements from a longitudinal clinical trial.","PeriodicalId":354759,"journal":{"name":"Statistical Modeling","volume":"1990 1","pages":"0"},"PeriodicalIF":0.0000,"publicationDate":"2002-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"8","resultStr":"{\"title\":\"Assessing uncertainty about parameter estimates with incomplete repeated ordinal data\",\"authors\":\"Claudio J. Verzilli, J. Carpenter\",\"doi\":\"10.1191/1471082x02st033oa\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"Data collected in clinical trials involving follow-up of patients over a period of time will almost inevitably be incomplete. Patients will fail to turn up at some of the intended measurement times or will not complete the study, giving rise to various patterns of missingness. In these circumstances, the validity of the conclusions drawn from an analysis of available cases depends crucially on the mechanism driving the missing data process; this in turn cannot be known for certain. For incomplete categorical data, various authors have recently proposed taking into account in a systematic way the ignorance caused by incomplete data. In particular, the idea of intervals of ignorance has been introduced, whereby point estimates for parameters of interest are replaced by intervals or regions of ignorance (Vansteelandt and Goetghebeur, 2001; Kenward et al., 2001; Molenberghs et al., 2001). These are identified by the set of estimates corresponding to possible outcomes for the missing data under little or no assumptions about the missing data mechanism. Here we extend this idea to incomplete repeated ordinal data. We describe a modified version of standard algorithms used for fitting marginal models to longitudinal categorical data, which enables calculation of intervals of ignorance for the parameters of interest. The ideas are illustrated using dental pain measurements from a longitudinal clinical trial.\",\"PeriodicalId\":354759,\"journal\":{\"name\":\"Statistical Modeling\",\"volume\":\"1990 1\",\"pages\":\"0\"},\"PeriodicalIF\":0.0000,\"publicationDate\":\"2002-10-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"8\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Statistical Modeling\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.1191/1471082x02st033oa\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"\",\"JCRName\":\"\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Statistical Modeling","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.1191/1471082x02st033oa","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"","JCRName":"","Score":null,"Total":0}
引用次数: 8
摘要
在临床试验中收集的数据涉及一段时间内对患者的随访,几乎不可避免地是不完整的。患者将无法在某些预定的测量时间出现或无法完成研究,从而产生各种类型的缺失。在这种情况下,从现有案例分析中得出的结论的有效性主要取决于驱动缺失数据处理的机制;这反过来又不能确定。对于不完整的分类数据,最近有许多作者提出要系统地考虑不完整数据所造成的无知。特别是,引入了无知区间的概念,即对感兴趣参数的点估计被无知区间或区域所取代(Vansteelandt和Goetghebeur, 2001;Kenward et al., 2001;Molenberghs et al., 2001)。在对缺失数据机制很少或没有假设的情况下,通过一组与缺失数据的可能结果相对应的估计来识别这些数据。这里我们把这个思想扩展到不完全重复有序数据。我们描述了用于拟合边缘模型到纵向分类数据的标准算法的修改版本,它可以计算感兴趣参数的忽略区间。这些想法是用纵向临床试验的牙痛测量来说明的。
Assessing uncertainty about parameter estimates with incomplete repeated ordinal data
Data collected in clinical trials involving follow-up of patients over a period of time will almost inevitably be incomplete. Patients will fail to turn up at some of the intended measurement times or will not complete the study, giving rise to various patterns of missingness. In these circumstances, the validity of the conclusions drawn from an analysis of available cases depends crucially on the mechanism driving the missing data process; this in turn cannot be known for certain. For incomplete categorical data, various authors have recently proposed taking into account in a systematic way the ignorance caused by incomplete data. In particular, the idea of intervals of ignorance has been introduced, whereby point estimates for parameters of interest are replaced by intervals or regions of ignorance (Vansteelandt and Goetghebeur, 2001; Kenward et al., 2001; Molenberghs et al., 2001). These are identified by the set of estimates corresponding to possible outcomes for the missing data under little or no assumptions about the missing data mechanism. Here we extend this idea to incomplete repeated ordinal data. We describe a modified version of standard algorithms used for fitting marginal models to longitudinal categorical data, which enables calculation of intervals of ignorance for the parameters of interest. The ideas are illustrated using dental pain measurements from a longitudinal clinical trial.