A new paradigm of short stature: Incorporating parent and youth characteristics into understanding GH-related decision making and trajectories of quality of life and self-esteem

项目摘要/摘要 生长激素(GH)治疗是一种“扩展生物技术”的范例，其中一种生物医学技术 最初设计用于治疗疾病(GH缺乏症；GHD)的扩大，在井- 预期的医生和行业的支持，用于治疗模糊了 疾病和变异(特发性矮小；ISS)，估计潜在的国家成本为400亿美元。在……里面 我们之前的研究表明，父母对身材矮小对心理社会功能的影响的担忧， 无论是在儿童期还是成年期，都对他们寻求医疗保健的决定产生了重大影响 对于一个孩子的矮小身材来说。然而，到目前为止，身高一直是生长激素治疗的主要结果指标，甚至 尽管身高不能很好地预测心理社会适应和健康相关的生活质量(QOL)。长的- 这项研究的长期目标是确定是什么推动了青少年更好的生活质量和心理社会适应 身材矮小。虽然普遍的假设认为，身高的增加直接提供了 利益，我们假设身高对生活质量和自尊的影响被 无论有没有生长激素治疗，父母和年轻人的身高增加的影响都超过了这一程度。 因此，拟议的纵向混合方法研究将对8-14岁的青少年进行为期两年的跟踪 在我们的成长中心和家长参与者中接受挑衅性的生长激素测试。使用半结构化 访谈和新颖的量化测量，具体目标1将探索青少年和家长对Short的看法 身高及其对生活质量、对生长激素治疗和决策的认知和期望的影响 关于矮小身材的评价和生长激素治疗，重点是青年在决策中的作用， 它还没有被研究，但在慢性(数年)的选择性治疗(ISS)的背景下是关键的， 侵入性的(每日注射)，并有潜在的副作用。使用QOL的量化测量和 自尊的积极方面，具体目标2将描述青少年生活质量和自尊的轨迹2 刺激性生长激素检测数年后，包括患有生长激素缺乏症的青少年，接受生长激素治疗的ISS，以及未接受治疗的ISS 天哪。具体目标3将确定具体的父母和青年特征，以改变身高与 这两年的生活质量和自尊。这项申请中提出的研究将解决一个关键差距 在我们对耐心和父母特征如何改变矮小的潜在影响的理解中，以及 将目前持有的身材矮小的传统疾病模型转变为更丰富的框架，包括 父母特征、父母教养方式、青少年特征以及身高。拟议的工作将 从而为未来测试生长激素疗效的确定性、长期、随机对照试验奠定了基础 或其他将生活质量和心理社会适应作为主要结果的矮小干预措施， 并使现场和临床护理能够以循证、价值驱动的方式向前发展。

PROJECT SUMMARY/ABSTRACT Growth hormone (GH) treatment is a paradigm of “expansive biotechnology,” wherein a biomedical technology originally designed for treatment of disease (GH deficiency; GHD) expanded, with the encouragement of well- intended physicians and support of industry, into treatment of conditions that blur the boundary between disease and variation (idiopathic short stature; ISS), with an estimated potential national cost of $40 billion. In our prior research, parents indicated that concerns about the impact of short stature on psychosocial function, both in childhood and projected into adulthood, have a major influence on their decision to seek medical care for a child's short stature. Yet thus far height has been the primary outcome measure of GH treatment, even though height is a poor predictor of psychosocial adjustment and health-related quality of life (QoL). The long- term goal of this research is to determine what drives better QoL and psychosocial adjustment in youth with short stature. While the prevailing assumption holds that the attainment of increased height directly provides benefit, we hypothesize that the impact of height on QoL and self-esteem is modified by characteristics of the parent and youth to a degree that exceeds the impact from height gain itself, with or without GH treatment. Thus, the proposed longitudinal mixed-methods study will follow for two years youth aged 8-14 years who undergo provocative GH testing in our Growth Center and a parent participant. Using semi-structured interviews and novel quantitative measures, Specific Aim 1 will explore youth and parent perceptions of short stature and its impact on QoL, perceptions and expectations vis-a-vis GH treatment, and decision-making regarding evaluation of short stature and GH treatment, with a focus on the youth's role in decision-making, which has not been studied but is critical in the context of an elective treatment (ISS) that is chronic (years), invasive (daily injections), and carries potential side effects. Using quantitative measures of both QoL and the positive aspects of self-esteem, Specific Aim 2 will describe trajectories of youth QoL and self-esteem for 2 years after provocative GH testing, including youth with GHD, ISS treated with GH, and ISS not treated with GH. Specific Aim 3 will define specific parent and youth characteristics that modify the relationship of height to QoL and self-esteem for those two years. The research proposed in this application will address a critical gap in our understanding of how patient and parent characteristics alter the potential impact of being short, and transform the currently held, traditional disease model of short stature into a richer framework that includes parent characteristics, parenting styles and youth characteristics as well as height. The proposed work will thereby lay the foundation for future definitive, long-term, randomized controlled trials testing the efficacy of GH or other interventions for short stature that include QoL and psychosocial adjustment as primary outcomes, and enable the field and clinical care to move forward in an evidence-based, value-driven way.