GROWTH HORMONE USE IN ADOLESCENTS AND ADULTS WITH CYSTIC FIBROSIS

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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.