GROWTH HORMONE USE IN ADOLESCENTS AND ADULTS WITH CYSTIC FIBROSIS

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。所列机构为中心机构，不一定为研究者机构。囊性纤维化（CF）是美国最常见的致命遗传性疾病，每2500名新生儿中就有1名受到影响。临床上以肺部感染、胰腺外分泌和内分泌功能不全为特征。许多CF患者营养不良，营养不良显然与临床结果恶化有关。因此，改善营养和生长对于CF患者的成功治疗和最终生存至关重要。美国国家囊性纤维化基金会（National Cystic Fibrosis Foundation）的数据显示，34%的成年CF患者体重低于理想体重的85%，37%的患者体重指数低于19%。临床稳定和急性CF患者的蛋白质catenin率升高。蛋白质负平衡可能导致CF的发病率和死亡率。瘦体重与一秒用力呼气量（FEV 1）和急性肺恶化住院次数相关。 我们将对12例GH患者进行为期一年的治疗。在开始GH治疗前，患者将接受为期6个月的研究，不接受任何治疗。每例患者将通过比较治疗数据与非治疗数据作为其自身对照。研究的主要终点是确定GH对体重和瘦体重的影响。次要终点是记录以下方面的改善：1）全身蛋白质转换，2）肝脏葡萄糖生成，3）骨矿物质密度，4）肺功能状态和5）生活质量。将监测不良事件，如葡萄糖耐受不良。我们假设生长激素（GH）的合成代谢作用将通过改善瘦体重、骨质减少、肌肉力量、肺功能和生活质量来改善CF成人的临床状况。

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. Cystic fibrosis (CF) is the most common lethal genetic disorder in America, affecting 1 in 2500 births. Clinically the disease is characterized by frequent pulmonary infections and pancreatic exocrine and endocrine insufficiency. Many CF patients are malnourished and malnutrition has been clearly linked to worsened clinical outcome. Therefore improved nutrition and growth are of paramount importance in the successful treatment and ultimate survival of patients with CF. The national Cystic Fibrosis Foundation has documented that 34% of adults with CF are less that 85% of ideal body weight and 37% have a body mass index less than 19%. Both clinically stable and acutely ill CF patients have elevated rates of protein catabolism. Negative protein balance may contribute to morbidity and mortality in CF. Lean body mass has been correlated with forced expiratory volume in one second (FEV1) and with the number of hospitalizations from acute pulmonary exacerbation. We will treat 12 patients with GH for one year. Prior to starting GH, patients will be studied for 6 months without any treatment. Each patient will serve as his/her own control by comparing treatment data to non-treatment data. The primary endpoint of the study is to determine the effect of GH on body weight and lean body mass. The secondary endpoints are to document improvement in: 1) whole body protein turnover, 2) hepatic glucose production, 3) bone mineral density, 4) pulmonary function status, and 5) quality of life. Adverse events, such as glucose intolerance, will be monitored. We hypothesize that the anabolic effects of growth hormone (GH) will improve the clinical status of adults with CF by improving lean body mass, osteopenia, muscle strength, pulmonary function, and quality of life.