GROWTH HORMONE USE IN ADOLESCENTS AND ADULTS WITH CYSTIC FIBROSIS

本子项目是利用由NIH/NCRR资助的中心赠款提供的资源的众多研究子项目之一。子项目和研究者（PI）可能已经从另一个NIH来源获得了主要资金，因此可以在其他CRISP条目中表示。列出的机构是中心的，不一定是研究者的机构。囊性纤维化（CF）是美国最常见的致命性遗传疾病，每2500个新生儿中就有1个患有此病。临床表现为多发肺部感染、胰腺外分泌和内分泌功能不全。许多CF患者营养不良，营养不良显然与恶化的临床结果有关。因此，改善营养和生长对CF患者的成功治疗和最终生存至关重要。国家囊性纤维化基金会记录了34%的CF成人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患者的蛋白质分解代谢率均升高。负蛋白平衡可能导致CF的发病率和死亡率。瘦体重与一秒钟用力呼气量（FEV1）和急性肺恶化住院次数相关。我们将对12名GH患者进行为期一年的治疗。在开始GH之前，患者将在没有任何治疗的情况下进行6个月的研究。每个患者将作为自己的对照，将治疗数据与非治疗数据进行比较。该研究的主要目的是确定生长激素对体重和瘦体重的影响。次要终点是记录改善：1)全身蛋白质周转，2)肝葡萄糖生成，3)骨矿物质密度，4)肺功能状态，5)生活质量。不良事件，如葡萄糖耐受不良，将被监测。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.