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Statistical methods for non-ignorable missing data with applications to quality-of-life data.

机译:不可忽略的缺失数据的统计方法,并应用于生活质量数据。

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摘要

Researchers increasingly use more and more survey studies, and design medical studies to better understand the relationships of patients, physicians, their health care system utilization, and their decision making processes in disease prevention and management. Longitudinal data is widely used to capture trends occurring over time. Each subject is observed as time progresses, but a common problem is that repeated measurements are not fully observed due to missing response or loss to follow up. An individual can move in and out of the observed data set during a study, giving rise to a large class of distinct "non-monotone" missingness patterns. In such medical studies, sample sizes are often limited due to restrictions on disease type, study design and medical information availability. Small sample sizes with large proportions of missing information are problematic for researchers trying to understand the experience of the total population. The information in the data collected may produce biased estimators if, for example, the patients who don't respond have worse outcomes, or the patients who answered "unknown" are those without access to medical or non-medical information or care. Data modeled without considering this missing information may cause biased results.;A first-order Markov dependence structure is a natural data structure to model the tendency of changes. In my first project, we developed a Markov transition model using a full-likelihood based algorithm to provide robust estimation accounting for "non-ignorable'' missingness information, and applied it to data from the Penn Center of Excellence in Cancer Communication Research. In my second project, we extended the method to a pseudo-likelihood based approach by considering only pairs of adjacent observations to significantly ease the computational complexities of the full-likelihood based method proposed in the first project. In my third project, we proposed a two stage pseudo hidden Markov model to analyze the association between quality of life measurements and cancer treatments from a randomized phase III trial (RTOG 9402) in brain cancer patients. By incorporating selection models and shared parameter models with a hidden Markov model, this approach provides targeted identification of treatment effects.
机译:研究人员越来越多地使用越来越多的调查研究,并设计医学研究以更好地了解患者,医生,他们的医疗保健系统利用率以及他们在疾病预防和管理中的决策过程之间的关系。纵向数据被广泛用于捕获随时间变化的趋势。随着时间的流逝,每个受试者都会被观察到,但是一个常见的问题是由于缺少响应或无法进行随访,因此无法完全观察到重复的测量结果。一个人可以在研究期间移入和移出观察到的数据集,从而产生一大类独特的“非单调”缺失模式。在此类医学研究中,由于疾病类型,研究设计和医学信息可用性的限制,样本量通常受到限制。对于试图了解总人口经验的研究人员而言,小样本量和大量缺失信息是有问题的。例如,如果不响应的患者的结局较差,或者回答“未知”的患者是无法获得医学或非医学信息或护理的患者,则收集的数据中的信息可能会产生偏差估计。不考虑此缺失信息而建模的数据可能会导致偏差的结果。一阶马尔可夫依赖结构是一种自然的数据结构,用于建模变化趋势。在我的第一个项目中,我们使用基于完全似然的算法开发了一个马尔可夫转移模型,以提供针对“不可忽略的”缺失信息的可靠估计,并将其应用于宾夕法尼亚大学癌症传播研究卓越中心的数据。在我的第二个项目中,我们通过只考虑相邻的观测值对将方法扩展到基于伪似然的方法,从而显着缓解了第一个项目中提出的基于完全似然法的计算复杂性;在我的第三个项目中,我们提出了两个阶段伪隐藏马尔可夫模型,用于分析脑癌患者的一项随机III期试验(RTOG 9402)的生活质量测量结果与癌症治疗之间的关联。通过将选择模型和共享参数模型与隐藏马尔可夫模型相结合,该方法可提供针对性识别治疗效果。

著录项

  • 作者

    Liao, Kaijun.;

  • 作者单位

    University of Pennsylvania.;

  • 授予单位 University of Pennsylvania.;
  • 学科 Biology Biostatistics.
  • 学位 Ph.D.
  • 年度 2012
  • 页码 121 p.
  • 总页数 121
  • 原文格式 PDF
  • 正文语种 eng
  • 中图分类
  • 关键词

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