Statistical Designs and Methods for Double-Sampling for HIV/AIDS

HIV/艾滋病双重抽样的统计设计和方法

• 批准号: 8541216
• 负责人: CONSTANTINE E FRANGAKIS
• 金额: $ 40.81万
• 依托单位: JOHNS HOPKINS UNIVERSITY
• 依托单位国家: 美国
• 财政年份: 2013
• 资助国家: 美国
• 起止时间: 2013-01-15 至 2016-12-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8541216
• 关键词: AIDS/HIV problem; Accounting; Acquired Immunodeficiency Syndrome; Address; Africa; Ambulatory Care; Caring; Characteristics; Clinical; Congresses; Data; Dropout; Emergency Situation; Enrollment; Ensure; Epidemic; Evaluation; Funding; Goals; HIV; Letters; Life; Malaria; Measures; Methods; Modeling; Monitor; Outcome; Patient Dropouts; Patients; Performance; Physicians; Program Evaluation; Public Health; Publications; Recording of previous events; Research; Research Personnel; Resources; Risk; Sampling; Stratification; Surveys; Tuberculosis; Work; base; cohort; design; experience; fighting; follow-up; improved; mortality; novel; programs; public health relevance; success; treatment program

DESCRIPTION (provided by applicant): Accurate evaluation of programs that treat and monitor HIV/AIDS patients around the world is central for fighting the epidemic. A major obstacle for program evaluation is patient dropout. An important such program is the President's Emergency Plan for AIDS Relief (PEPFAR). The US has been sponsoring PEPFAR ($63 billion for 2004-2013), and methods to accurately estimate patient survival are central to guide US management of the program. However, PEPFAR experiences high dropout rates (e.g., 39% in two years). Standard survival methods use only the observed non-dropout data, with no objective information for the dropouts. Such methods can be severely biased when dropout patients differ from nondropouts after accounting for observed information. To provide valid evaluation, in earlier work we have used a richer design known as "double-sampling". This design re-allocates increased resources to target, intensively pursue and find a subset of the dropouts. These double-sampled dropouts are intended to represent the non-double-sampled dropouts, and to provide objective information for the entire cohort. Although standard methods have been known for double-sampling in surveys, we have shown earlier that standard survival methods fail when double-sampling is used in continuous enrollment programs such as PEPFAR. Also, we have shown earlier that standard evaluation without double-sampling can dramatically underestimate mortality in PEPFAR by a factor of 5. The proposed methods will build on our earlier work with the framework of "principal stratification". The success of that framework increases the potential impact of this proposal. The proposed new methods are developed for three specific aims, motivated by PEPFAR in East Africa. (Aim 1). Develop methods to estimate the performance of follow-up programs by using data from a given double- sampling design. In this aim we will develop methods to estimate survival from double-sampling designs that select patients based on their history characteristics before dropout. This is also important for the next two aims. (Aim 2). Develop methods to create double-sampling designs that produce most accurate estimation of a pro- gram's performance given fixed resources. Evidence shows that information specific to a patient is important for what double-sampling designs provide best information about a program. Here, we will create patient-dependent double-sampling designs that maximize the accuracy given resources to estimate survival in such programs. (Aim 3). Develop double-sampling designs to best target clinical goals. Aim 1 can use the dropout patients' clinical history to predict those with highest mortality risk. These predictions can constrain the design to ensure to double-sample all such patients to better serve them medically. In Aim 3, we will create designs that maximize the accuracy of estimation and best benefit patients.

描述（由申请人提供）：准确评估治疗和监测世界各地艾滋病毒/艾滋病患者的计划对于抗击这一流行病至关重要。项目评估的一个主要障碍是患者退出。其中一个重要的计划是总统艾滋病紧急救援计划 (PEPFAR)。美国一直在赞助总统防治艾滋病紧急救援计划（2004-2013 年资助 630 亿美元），准确估计患者生存率的方法对于指导美国对该计划的管理至关重要。然而，PEPFAR 的辍学率很高（例如两年内为 39%）。标准生存方法仅使用观察到的非脱落数据，没有脱落的客观信息。当辍学患者在考虑观察到的信息后与非辍学患者不同时，这种方法可能会产生严重偏差。为了提供有效的评估，在早期的工作中，我们使用了一种更丰富的设计，称为“双采样”。这种设计将增加的资源重新分配给目标，集中追捕并找到一部分辍学者。这些双抽样的退出者旨在代表非双抽样的退出者，并为整个队列提供客观信息。尽管众所周知，标准方法在调查中采用双抽样，但我们之前已经表明，当双抽样用于连续入组计划（例如 PEPFAR）时，标准生存方法会失败。此外，我们之前已经表明，没有双重抽样的标准评估可能会大大低估 PEPFAR 的死亡率达 5 倍。所提出的方法将建立在我们早期工作的“主要分层”框架的基础上。该框架的成功增加了该提案的潜在影响。在东非总统防治艾滋病紧急救援计划的推动下，所提出的新方法是为了三个具体目标而开发的。 （目标 1）。开发使用给定双抽样设计的数据来估计后续计划绩效的方法。为此，我们将开发通过双抽样设计估计生存率的方法，该设计根据患者退出前的病史特征选择患者。这对于接下来的两个目标也很重要。 （目标 2）。开发方法来创建双采样设计，从而在给定固定资源的情况下对程序的性能进行最准确的估计。有证据表明，患者的特定信息对于双采样设计提供有关项目的最佳信息非常重要。在这里，我们将创建依赖于患者的双抽样设计，最大限度地提高给定资源的准确性，以估计此类项目的生存率。 （目标 3）。开发双采样设计以最好地实现临床目标。目标 1 可以利用退出患者的临床病史来预测死亡风险最高的患者。这些预测可以限制设计，以确保对所有此类患者进行双重采样，以便更好地为他们提供医疗服务。在目标 3 中，我们将创建能够最大限度提高估计准确性并为患者带来最大利益的设计。