Assessment of Growth Horomone Use in Short Children

描述（由申请人提供）：父母、初级保健医生（pcp）和专家（内分泌学家）如何决定用生长激素（GH）治疗矮小儿童（特发性矮小；ISS） ？关于生长激素使用的决定将影响多达200万儿童，每年的潜在成本高达400亿美元。在我们之前的工作基础上，我们确定了一组4个决定，共同决定生长激素的使用：是否讨论孩子的生长，将孩子转介给内分泌学家，开始生长激素治疗，继续生长激素。通过判断分析，我们假设有5个领域会影响这些关键决策；这些领域包括生理特征、态度和信念、结构和组织因素、知识和经验以及社会人口因素。在目标1中，我们关注的是家庭和他们的pcp所做的决定，即是否讨论和采取行动，以促进身高低于年龄10%的儿童的成长。目的1的方法包括分析在初级保健中为矮个子儿童做出的实际决定（n=534），使用来自3个来源的数据：与父母和pcp的访谈、图表回顾和标准化问卷调查。进行定性和定量分析。在目标2中，我们重点关注pcp向内分泌学家的转诊。我们对pcp （n = 1920）的分层样本进行了一项全国性研究，使用因子实验设计的调查来确定将矮个子儿童转介给内分泌学家的决定因素。Aim 1的研究结果将有助于构建Aim 2的调查。在Aim 3中，我们关注内分泌学家关于是否开始和是否继续生长激素（作为个体化治疗试验）的决定。我们进行了一项全国范围的儿科内分泌学家普查研究（n= 1200），以确定他们建议ISS患者开始和继续/停止GH的决定因素，采用病例情景因子实验设计。我们还检查了纵向数据库，以评估个别儿童生长激素治疗的持续时间。在所有3个目标中，我们将为决策者获取5个领域的信息，并评估他们对所研究判断的解释力。这个项目将提供关于如何决定生长激素使用的基本知识。它将确定生长激素使用的关键驱动因素，并确定可以调节生长激素使用的关键点。它将为将来对身材矮小的前瞻性纵向研究提供基础，包括帮助家庭和医生做出明智的管理决策的干预措施。此外，获得的关于治疗使用决定因素的知识可能适用于许多开始和结束不明确，但受到患者高度重视的新兴疗法。

DESCRIPTION (provided by the applicant): How do parents, primary care physicians (PCPs), and specialists (endocrinologists) make decisions to treat short children (idiopathic short stature; ISS) with growth hormone (GH)? Decisions about GH use will affect up to 2 million children, with potential costs of up to $40 billion per year. Building on our previous work, we identify a set of 4 decisions that, together, govern GH use: whether to discuss the child's growth, refer the child to an endocrinologist, initiate GH treatment, and continue GH. Using Judgment Analysis, we hypothesize that 5 domains will influence these key decisions; the domains are physiological characteristics, attitudes and beliefs, structural and organizational factors, knowledge and experience, and sociodemographic factors. In Aim 1, we are concerned with decisions actually made by families and their PCPs about whether to discuss and action about growth in children whose heights are below the 10th percentile for age. The methods for Aim 1 involve analyses of actual decisions made for short children in primary care (n=534), using data from 3 sources: interviews with parents and PCPs, chart review, and standardized questionnaires. Qualitative and quantitative analyses be performed. In Aim 2, we focus on referrals by PCPs to endocrinologists. We conduct a national study of a stratified sample of PCPs (n = 1920) to define the determinants of decisions to refer short children to endocrinologists, using surveys with factorial experimental design. Findings from Aim 1 will help to structure the Aim 2 survey. In Aim 3, we focus on endocrinologists' decisions about whether to initiate and whether to continue GH (as individualized therapeutic trials). We conduct a national study of a census of pediatric endocrinologists (n= 1200) to define the determinants of their recommendations for initiation and continuation/discontinuation of GH in ISS, using case scenarios factorial experimental design. We also examine a longitudinal database to assess duration of GH treatment in individual children. In all 3 aims, we will obtain information on the 5 domains for decision-makers and assess their explanatory power for the judgments under study. This project will provide fundamental knowledge about how decisions are made that determine GH use. It will identify key drivers of GH use, and define pivotal points at which GH use can be modulated. It will provide a foundation future prospective, longitudinal studies of short stature- including interventions that can help families and physicians make informed management decisions. Moreover, the knowledge gained on determinants of treatment use will likely be applicable to many emerging therapies that have ambiguous beginnings and endings, but are highly valued by patients.