Multivariate modelling of responses to conditional items: New possibilities for latent class analysis

Questionnaire data may contain missing values because certain questions do not apply to all respondents. For instance, questions addressing particular attributes of a symptom, such as frequency, triggers or seasonality, are only applicable to those who have experienced the symptom, while for those who have not, responses to these items will be missing. This missing information does not fall into the category ‘missing by design’, rather the features of interest do not exist and cannot be measured regardless of survey design. Analysis of responses to such conditional items is therefore typically restricted to the subpopulation in which they apply. This article is concerned with joint multivariate modelling of responses to both unconditional and conditional items without restricting the analysis to this subpopulation. Such an approach is of interest when the distributions of both types of responses are thought to be determined by common parameters affecting the whole population. By integrating the conditional item structure into the model, inference can be based both on unconditional data from the entire population and on conditional data from subjects for whom they exist. This approach opens new possibilities for multivariate analysis of such data. We apply this approach to latent class modelling and provide an example using data on respiratory symptoms (wheeze and cough) in children. Conditional data structures such as that considered here are common in medical research settings and, although our focus is on latent class models, the approach can be applied to other multivariate models. Copyright © 2009 John Wiley & Sons, Ltd.     

早期糖尿病视网膜病变药物治疗的药物经济学分析

目的：探讨不同方案治疗早期糖尿病视网膜病变的经济效果,找出既经济又有效的方案。方法：对目前临床常用的4种不同治疗方案（导升明、胰激肽原酶、复方血栓通胶囊、复方丹参滴丸,治疗时间均为90d）运用药物经济学方法进行成本-效果分析。结果：4种方案所需费用分别为1417.50元、810.00元、891.00元、656.10元;治疗有效率分别为94.25%、85.09%、85.17%、83.53%;成本-效果比分别为15.04、9.52、10.46、7.85。结论：复方丹参滴丸治疗效果较好,不良反应少,成本低廉。     

结节性甲状腺肿合并其他甲状腺疾病临床分析

目的探讨结节性甲状腺肿合并其他甲状腺疾病的临床特点。方法回顾性分析我院手术治疗的4622例结节性甲状腺肿中并存其他甲状腺疾病的临床资料。结果4622例结节性甲状腺肿中700例合并其他甲状腺疾病，1418例存在不同的诊断失误。结论在诊断结节性甲状腺肿的同时应注意其合并的其他甲状腺疾病。     

One‐ and two‐stage design proposals for a phase II trial comparing three active treatments with control using an ordered categorical endpoint

Phase II clinical trials are performed to investigate whether a novel treatment shows sufficient promise of efficacy to justify its evaluation in a subsequent definitive phase III trial, and they are often also used to select the dose to take forward. In this paper we discuss different design proposals for a phase II trial in which three active treatment doses and a placebo control are to be compared in terms of a single‐ordered categorical endpoint. The sample size requirements for one‐stage and two‐stage designs are derived, based on an approach similar to that of Dunnett. Detailed computations are prepared for an illustrative example concerning a study in stroke. Allowance for early stopping for futility is made. Simulations are used to verify that the specified type I error and power requirements are valid, despite certain approximations used in the derivation of sample size. The advantages and disadvantages of the different designs are discussed, and the scope for extending the approach to different forms of endpoint is considered. Copyright © 2008 John Wiley & Sons, Ltd.     

Measurement in clinical trials: A neglected issue for statisticians?

Biostatisticians have frequently uncritically accepted the measurements provided by their medical colleagues engaged in clinical research. Such measures often involve considerable loss of information. Particularly, unfortunate is the widespread use of the so‐called ‘responder analysis’, which may involve not only a loss of information through dichotomization, but also extravagant and unjustified causal inference regarding individual treatment effects at the patient level, and, increasingly, the use of the so‐called number needed to treat scale of measurement. Other problems involve inefficient use of baseline measurements, the use of covariates measured after the start of treatment, the interpretation of titrations and composite response measures. Many of these bad practices are becoming enshrined in the regulatory guidance to the pharmaceutical industry. We consider the losses involved in inappropriate measures and suggest that statisticians should pay more attention to this aspect of their work. Copyright © 2009 John Wiley & Sons, Ltd.     

Is the political system really related to health?

We analyze whether the political system and its stability are related to cross-country differences in health. We apply factor analysis on various national health indicators for a large sample of countries over the period 2000–2005 and use the outcomes of the factor analysis to construct two new health measures, i.e., the health of individuals and the quality of the health care sector. Using a cross-country structural equation model with various economic and demographic control variables, we examine the relationship between the type of regime and political stability on the one hand and health on the other. The political variables and the control variables are measured as averages over the period 1980–1999. Our results suggest that democracy has a positive relationship with the health of individuals, while regime instability has a negative relationship with the health of individuals. Government instability is negatively related to individual health via its link with the quality of the health care sector, while democracy is positively related with individual health through its link with income. Our main findings are confirmed by the results of a panel model and various sensitivity tests.     

《实用美容整形外科杂志》（1999—2003年）载文分析

目的 探讨《实用美容整形外科杂志》的载文特点。方法 利用文献计量学方法统计1999～2003年出版的30期《实用美容整形外科杂志》的多项文献计量学指标。结果 5年的总载文量为975篇，总页数为1680页。有较为固定的十几个栏目，平均发表时滞为242．6天，平均基金论文比为0．05。合著的论文占总数的84．8％，平均论文作者合作度为3．26。作者分布在全国29个省、自治区或直辖市。工作在省市医院、医药院校附属医院和部队医院的作者较多。结论 《实用美容整形外科杂志》是以省市医院、医药院校附属医院和部队医院的中级以上职称的中青年作者为主要作者群的国家级美容整形外科专业核心期刊。     

NSCLC患者行PET检查的成本效益分析

目的研究非小细胞肺癌（NSCLC）患者行PET和CT检查的成本效益比,寻找更低成本效益比的检查.方法采用决策分析法,对国内现有NSCLC患者PET及CT诊断效率研究中的数据进行分析,比较其正确检出费用、正确分期费用和正确治疗费用.结果①就检出率而言,CT的成本效益比更低;②就分期准确性而言,CT的成本效益比仍较PET低;③就诊疗费用的成本效益比而言,PET较CT为低;④PET与CT相比,其优势在于诊断Ⅰ期的患者.结论CT适合NSCLC的筛选,而PET适用于临床分期,指导临床治疗.     

305例尿石成份分析及预防对策

目的研究一种快速、简便的方法对泌尿系结石进行分析,并探讨其防治方法。方法用化学常量法对305例患者的尿石标本进行化学成份测定,并结合临床资料进行分析。结果发现结石发病男性多于女性(比例为3.2∶1),20~50岁多发,上尿路结石多于下尿路结石(5.4∶1)。结石中草酸钙检出率达82%,尿酸盐55.2%,磷酸钙48.6%,磷酸镁铵与碳酸磷灰石则多见于泌尿系感染病例,胱氨酸结石少见。结论化学常量法分析泌尿系结石简便、快捷、准确。结石成份分析对于了解结石成因、预防结石复发具有一定的意义。