Improving Clinical Status and Quality of Life in Cystic Fibrosis Patients with Growth Hormone Treatment

Cystic Fibrosis (CF) is a common genetic disorder in America that can be life-threatening. Previous research indicates that a man-made hormone, known as human recombinant growth hormone (GH), can enhance growth, weight gain, muscle mass, and lung function in CF patients. This study aims to delve deeper into the benefits of GH use in CF, including whether patients with better initial weight and lung function gain more from the treatment, if GH improves the patient's quality of life, and if the positive effects persist after stopping GH. The study hypothesizes that GH treatment will improve CF patients' health and quality of life, regardless of their initial weight or lung function, and that these benefits will last for at least a year after treatment ends. The study involves 40 prepubertal children from five CF centers across the United States. Participants are randomly assigned to receive GH treatment during either the first or second year. The GH is administered at a dose of 0.3mg/kg/wk. Every three months, the study measures height, height growth rate, weight, and weight growth rate. Every six months, it evaluates muscle mass using DEXA, lung function, including respiratory muscle strength, and quality of life using a CF-specific questionnaire. After a year, participants switch to the other treatment group. This 24-month study allows for a statistical comparison of results between treated and non-treated subjects from multiple centers, and assesses if the benefits persist in the group that received GH during the first year.