GH IN CHILDREN WITH SHORT STATURE DUE TO GROWTH HORMONE INSENSITIVITY (GHIS)

该子项目是利用NIH/NCRR资助的中心赠款提供的资源的许多研究子项目之一。子项目和研究者（PI）可能从另一个NIH来源获得主要资金，因此可以在其他CRISP条目中表示。所列机构为中心，不一定是研究者所在机构。胰岛素样生长因子I（IGF-I）是响应垂体生长激素（GH）产生的，是生长的主要调节因子。有些孩子有IGF-I缺乏症，因为他们不能响应自己的生长激素。他们有严重的生长迟缓，对常规治疗无反应。本研究评价了重组人IGF-I（rhIGF-I）治疗生长激素不敏感矮小儿童的安全性和有效性。儿童必须有临床记录的GH不敏感性与血清IGF-I水平低的年龄，正常的GH分泌和证据的GH行动失败，或已开发GH中和抗体在GH治疗。 一旦入组，儿童每天两次皮下注射rhIGF-I，直到他们达到最终身高。主要疗效终点是生长率。至少每6个月对受试者进行一次评估。安全措施包括监测血液化学、肾脏和脾脏生长以及心脏功能。

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. Insulin-like growth factor I (IGF-I) is produced in response to pituitary growth hormone (GH) and is a major regulator of growth. Some children have IGF-I deficiency because they cannot respond to their own GH. They have severe growth retardation unresponsive to conventional therapies. This study evaluates the safety and efficacy of recombinant human IGF-I (rhIGF-I) given to short children with growth hormone insensitivity. Children must have clinically documented GH insensitivity with serum IGF-I levels that are low for age, normal GH secretion and evidence for failure of GH action, or have developed GH-neutralizing antibodies during GH treatment. Once enrolled, children are treated with subcutaneous injection of rhIGF-I twice daily until they reach final height. The primary efficacy endpoint is growth rate. Subjects are evaluated a minimum of every 6 months. Safety measures include monitoring of blood chemistries, kidney and spleen growth, and cardiac function.