Growth hormone treatment improves growth and clinical status in prepubertal children with cystic fibrosis: results of a multicenter randomized controlled trial.

Context: This multicenter, randomized, controlled, crossover trial of prepubertal children with cystic fibrosis (CF) tests the hypotheses that recombinant human GH (rhGH) treatment 1) improves height, weight, lean mass, and bone content irrespective of baseline measures; 2) improves clinical status and quality of life; and 3) has continued effect after cessation after 1 yr of treatment.

Methods: Sixty-one prepubertal subjects (
Results: Groups were similar at baseline and prepubertal during the entire study. After 1 yr, GH showed significantly greater gain in height, weight, lean mass, and bone mineral content. Gain in height was similar regardless of baseline. There were fewer hospitalizations in the rhGH-treated group and improvement in CF quality of life questionnaires measures of weight and body image. There was no difference in pulmonary function between groups. Results were similar in those treated with rhGH the second year. After cessation of rhGH treatment, there was sustained effect for increased height and weight velocity, as well as accrual of bone mineral.

Conclusion: rhGH therapy improves height and weight, decreases the number of hospitalizations, and improves quality of life in prepubertal children with CF. These effects are sustained after rhGH is discontinued.