Growth Hormone Use in Cystic Fibrosis - a Multicenter Study
Growth Hormone Use in Cystic Fibrosis - a Multicenter Study
Cystic Fibrosis (CF) is the most common lethal genetic disorder in America. Previous studies
 by our group and others have shown that human recombinant growth hormone (GH) improves height
 velocity, weight velocity, lean body mass (LBM) and pulmonary function. These positive
 results have prompted us to ask further questions regarding GH use in CF including: a) Do
 patients with better baseline body weight and pulmonary function derive more benefit from
 treatment than those with worse weight and pulmonary function?, b) Does GH use improve the
 patient's quality of life?, c) Once GH is discontinued, are the positive effects sustained?
 We hypothesize that GH treatment in CF patients will improve their clinical status and their
 quality of life. We further hypothesize that these effects will be present regardless of
 baseline body weight or pulmonary function, and that positive outcome will be sustained for
 at least one year after GH treatment is discontinued. To test our hypothesis, we will recruit
 40 prepubertal children from five CF centers across the United States (8 per center).
 Patients will be randomly assigned to receive treatment with GH (0.3mg/kg/wk) during either
 the first or the second year. All subjects will be seen every three months. We will evaluate
 the following parameters every three months: 1) height, height velocity and Z-score, 2) body
 weight and weight velocity. Every six months we will measure: 1) lean body mass utilizing
 DEXA, 2) pulmonary function, including measurement of respiratory muscle strength (peak
 inspiratory and peak expiratory pressure), 3) quality of life (QOL), quantitated from QOL
 forms specific for CF ("The Cystic Fibrosis Questionnaire"). After one year of study,
 subjects will "cross-over" to the other treatment arm. This 24 month study will allow us to
 statistically compare outcome measures in 20 treated and 20 nontreated subjects from multiple
 centers, and will allow us to assess sustained effect in the 20 subjects who receive GH
 during the first year, by comparing their results to results obtained during the year post
 treatment.
Inclusion Criteria:

 -  CF patients ages 5-12 who are less than the 25th percentile for age and sex normal
 values for height and/or weight