Dixi Xue
Dixi Xue
Sequential monitoring of efficacy and safety data has become a vital component of modern clinical trials. It affords companies the opportunity to stop studies early in cases when it appears as if the primary objective will not be achieved o...
Comparative Study
Pharmaceutical statistics. 2012 Nov-Dec;11(6):468-75. DOI:10.1002/pst.1538 2012
A bivariate Bayesian dose-finding procedure applied to a seamless phase I/II trial in rheumatoid arthritis [0.03%]
应用于类风湿关节炎的一期/二期无缝试验的二元贝叶斯剂量寻找方法
Helene Thygesen,Vladimir Dragalin,Anne Whitehead et al.
Helene Thygesen et al.
We describe a dose escalation procedure for a combined phase I/II clinical trial. The procedure is based on a Bayesian model for the joint distribution of the occurrence of a dose limiting event and of some indicator of efficacy (both consi...
Joint modeling of hierarchically clustered and overdispersed non-gaussian continuous outcomes for comet assay data [0.03%]
高斯连续型层级聚类与过度离散非 gauss comet 裂隙实验数据的联合建模方法研究
Aklilu Habteab Ghebretinsae,Christel Faes,Geert Molenberghs et al.
Aklilu Habteab Ghebretinsae et al.
Multivariate longitudinal or clustered data are commonly encountered in clinical trials and toxicological studies. Typically, there is no single standard endpoint to assess the toxicity or efficacy of the compound of interest, but co-primar...
Comparative Study
Pharmaceutical statistics. 2012 Nov-Dec;11(6):449-55. DOI:10.1002/pst.1533 2012
Application of a stochastic model to response assessments in a study of squamous cell carcinoma of the head and neck [0.03%]
头颈部鳞状细胞癌研究中对随机模型反应进行评估的应用
Habib Hassani
Habib Hassani
This paper discusses the application of a stochastic model in the analysis of response assessments made at various time points in a clinical trial of patients with squamous cell carcinoma of the head and neck. The transition rates and proba...
Clinical Trial
Pharmaceutical statistics. 2012 Nov-Dec;11(6):462-7. DOI:10.1002/pst.1537 2012
A structured approach to choosing estimands and estimators in longitudinal clinical trials [0.03%]
纵向临床试验中选择估计量和统计量的结构化方法
C H Mallinckrodt,Q Lin,I Lipkovich et al.
C H Mallinckrodt et al.
An important evolution in the missing data arena has been the recognition of need for clarity in objectives. The objectives of primary focus in clinical trials can often be categorized as assessing efficacy or effectiveness. The present inv...
Mark Janssens,Geert Molenberghs,René Kerstens
Mark Janssens
Missing data in clinical trials is a well-known problem, and the classical statistical methods used can be overly simple. This case study shows how well-established missing data theory can be applied to efficacy data collected in a long-ter...
Multivariate extreme value modelling of laboratory safety data from clinical studies [0.03%]
基于临床研究实验数据的多元极值模型安全分析
Harry Southworth,Janet E Heffernan
Harry Southworth
Generally, in the interpretation of clinical safety laboratory data, it is extreme values that indicate potential safety issues. We illustrate the application of multivariate extreme value modelling to such data. Applying the methods to a c...
Measurement error and outcomes defined by exceeding a threshold: biased findings in comparative effectiveness trials [0.03%]
度量误差与超出阈值判定的结局指标：非劣效性临床试验中的研究偏倚问题分析
Petra Bůžková
Petra Bůžková
In medical studies, the long-term level of a risk factor exceeding a threshold is often an outcome of interest. In practice, such a risk factor may not be directly measurable. Instead, outcome variables are based on a single or multiple bio...
Dealing with excess of zeros in the statistical analysis of magnetic resonance imaging lesion count in multiple sclerosis [0.03%]
处理统计分析中多发性硬化症磁共振成像病变计数过多零的方法
Mercier Francois,Chin Peter,Francis Gordon
Mercier Francois
Lesion count observed on brain magnetic resonance imaging scan is a common end point in phase 2 clinical trials evaluating therapeutic treatment in relapsing remitting multiple sclerosis (MS). This paper compares the performances of Poisson...
Randomized Controlled Trial
Pharmaceutical statistics. 2012 Sep-Oct;11(5):417-24. DOI:10.1002/pst.1529 2012
Sara Hughes,Julia Harris,Nancy Flack et al.
Sara Hughes et al.
Considerable statistical research has been performed in recent years to develop sophisticated statistical methods for handling missing data and dropouts in the analysis of clinical trial data. However, if statisticians and other study team ...
Meta-Analysis
Pharmaceutical statistics. 2012 Sep-Oct;11(5):410-6. DOI:10.1002/pst.1528 2012