Statistical Methods for HIV/AIDS Studies

描述(申请人提供)：在过去的20年里，在与艾滋病毒相关的研究方面做出了很大的努力。研究结果为基于患者个体特征的疾病管理提供了有用的信息。然而，大多数这种“个性化医疗”策略都是在特别的基础上获得的。有许多无法验证的子组分析的例子。需要新的量化方法来系统地在个人层面上制定合理的治疗策略。例如，平均而言，高效抗逆转录病毒疗法(HAART)是治疗艾滋病毒感染的一种非常有效的疗法。然而，并不是每个人都从HAART中受益。利用临床试验和观察性研究的数据，重要的是建立预测模型，以确定未来将从这种有效疗法中受益的患者。目前这项研究计划的主要主题之一是系统地开发定量方法，为未来的患者反应做出有效的推断。该提案提出了五个研究目标。前三项具体解决了个性化医疗问题。最后两个目标是开发新的方法来设计、监测和分析带有事件时间响应的艾滋病毒研究，这是评估新疗法的常用终点。在过去的资助期间，国际艾滋病协会组建了一支研究小组，在设计、监测和分析与艾滋病毒有关的临床试验和观察性研究方面，在开发理论上合理和实用的量化方法方面卓有成效。PI和联合调查人员也是哈佛大学生物统计艾滋病研究中心的高级统计员。他们通过艾滋病临床试验小组(ACTG)和神经艾滋病研究联盟(NARC)密切参与艾滋病毒研究。他们可以获得过去和目前ACTG和NARC研究的数据，并可以直接将开发的方法的结果应用于正在进行的和未来的艾滋病毒研究。公共卫生相关性：将利用临床试验和观察性研究的数据，开发用于艾滋病毒疾病患者管理的个性化医学方法的量化方法。还将开发新的统计程序来设计、监测和分析临床试验，以评估艾滋病毒感染的新疗法。

DESCRIPTION (provided by applicant): Great efforts have been made over the past 20 years in HIV-related research. The results have provided useful information regarding the patient's disease management based on the individual patient characteristics. However, most of such "personalized medicine" strategies were obtained on an ad hoc basis. There are numerous examples of subgroup analyses that could not be validated. Novel quantitative methods are needed to systematically develop sound treatment strategies at a personal level. For example, on average, the highly active antiretroviral therapy (HAART) is a very potent therapy for treating HIV-infection. However, not everyone benefits from HAART. Using data from clinical trials and observational studies, it is important to establish prediction models that can identify future patients who would benefit from such potent therapies. One of the main themes for this current research plan is to systematically develop quantitative methods to make valid inferences for future patient responses. There are five research aims in the proposal. The first three specifically address the personalized medicine issues. The last two aims are to develop new methods for designing, monitoring, and analyzing HIV studies with event time responses, which are commonly used endpoints for evaluating new therapies. The PI has assembled a research team which has been very productive in developing theoretically sound and practical quantitative methods for designing, monitoring and analyzing HIV- related clinical trials and observational studies over the past funding periods. The PI and co-investigators are also senior statisticians for the Center for Biostatical AIDS Research Center at Harvard University. They are intimately involved in HIV studies via the AIDS Clinical Trails Group (ACTG) and Neurologic AIDS research Consortium (NARC). They have access to the data from past and current ACTG and NARC studies, and can directly apply the results from developed methodologies to the on-going and future HIV studies. PUBLIC HEALTH RELEVANCE: Quantitative methods will be developed for personalized medicine approaches for patient management of HIV disease using the data from clinical trials and observational studies. New statistical procedures will also be developed for designing, monitoring, and analyzing clinical trials for evaluating new therapies for HIV-infection.