ASSESSMENT OF GROWTH HORMONE USE IN SHORT CHILDREN

矮小儿童生长激素使用的评估

• 批准号: 2857444
• 负责人: LEONA CUTTLER
• 金额: $ 30.34万
• 依托单位: CASE WESTERN RESERVE UNIVERSITY
• 依托单位国家: 美国
• 财政年份: 1992
• 资助国家: 美国
• 起止时间: 1992-09-01 至 2000-12-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/2857444
• 关键词: attitude; body height; child (0-11); child physical development; clinical research; epidemiology; health care cost /financing; hormone therapy; human subject; human therapy evaluation; outcomes research; parents; patient care personnel attitude; somatotropin

This proposal focuses on the use of biosynthetic human growth hormone (GH) in short children. Children who are deficient in endogenous GH fail to grow at a normal rate, and replacement of the deficient hormone may prevent severe dwarfism. Biosynthetic GH, derived by recombinant DNA technology and approved by the FDA (1985), was initially targeted for use in children with GH deficiency. However, the current use of GH is broadening because 1) the criteria for defining GH deficiency have become controversial, 2) evidence suggests that GH increases growth in many non-GH deficient short children, and 3) there are no generally accepted guidelines for the use of GH. In the absence of clear guidelines, the potential number of candidates for GH ranges from fewer than 20,000 if strict criteria for classical GH deficiency are applied to over 1,900,000 with a cost of $27 billion if indications are liberalized to include children who fall below the-fifth percentile for height. Given this uncertainty, the use of GH will be influenced not only by data on its medical outcome (i.e. its effect on height of short children) but also by the perceptions of its value among physicians, families of short children, and third party payers. Little is known about these areas. The specific aims of this project are, therefore, to determine the number of candidates for GH therapy, the attitudes of parents towards short stature and their preferences regarding height augmentation therapy, the opinions and practices of physicians regarding short stature and GH treatment, and the policies of thirdparty payers towards GH therapy. This will be accomplished by multiple methods of data collection - including the use of existing data sources to estimate the number of potential U.S. candidates for GH, detailed interviews and preference analysis with parents of short children, a national survey of physicians, and a national study of third party payers. These studies will determine patterns of GH use under current circumstances, and Will lead to a model for projecting the future demand for GH under different circumstances. Through the results of this study, we will be able to project the consequences of changes in cost, insurance coverage, efficacy data, and route of administration for GH on future patterns of GH utilization. These results are needed for the generation of rational policy guidelines for the use of GH. In addition, the approach developed-is likely to be widely applicable to other situations in which a) treatments may be expanded beyond the initially targeted population, b) the distinction between disease therapy and cosmetic therapy becomes blurred, and c) the- implementation of a treatment is influenced by consumer preference as well as by physician practice.

该提案的重点是生物合成的人类生长激素的使用。 (Gh)简称儿童。内源性生长激素缺乏的儿童失败 以正常的速度生长，替代缺乏的荷尔蒙可能 预防严重侏儒症。重组DNA衍生的生物合成生长激素 技术并经FDA批准(1985)，最初的目标是使用 在生长激素缺乏的儿童中。然而，GH目前的使用是 扩大范围是因为1)定义生长激素缺乏的标准已经成为 有争议的证据表明，生长激素能促进许多人的生长 非生长激素缺乏的矮小儿童，以及3)没有普遍接受的 生长激素的使用指南。在缺乏明确指导方针的情况下， GH的潜在候选人数量从少于20,000人不等，如果 经典生长激素缺乏症的严格标准适用于190万人以上 如果将适应症自由化以包括 身高低于第五个百分位数的儿童。鉴于此， 不确定性，GH的使用将不仅受到其数据的影响 医疗结果(即其对矮小儿童身高的影响)，但也由 医生、家庭对其价值的认知 儿童和第三方付款人。人们对这些地区知之甚少。 因此，该项目的具体目标是确定 在生长激素治疗的候选人中，父母对Short的态度 身高和他们对增高治疗的偏好 内科医生对矮小和生长激素的看法和做法 治疗，以及第三方付款人对生长激素治疗的政策。 这将通过多种数据收集方法来实现- 包括使用现有数据源来估计 GH的潜在美国候选人、详细面试和偏好 对矮小儿童的父母的分析，一项对医生的全国性调查， 以及一项关于第三方付款人的全国性研究。这些研究将确定 在当前情况下的生长激素使用模式，并将导致一个模型 以预测在不同情况下对生长激素的未来需求。 通过这项研究的结果，我们将能够预测 成本、保险覆盖范围、疗效数据和 生长激素给药途径对未来生长激素利用模式的影响。 这些结果是生成Rational政策指导方针所必需的 用于生长激素的使用。此外，开发的方法-很可能是 广泛适用于a)处理可能 超出了最初的目标人群，b)区别 疾病治疗和美容治疗之间的关系变得模糊，以及c) 治疗的实施受到消费者偏好的影响，如 医生的实践也是如此。