Prospective randomised treatment with recombinant human growth hormone in cystic fibrosis

Aim: To evaluate the efficacy and safety of treatment with recombinant growth hormone ( rGH) in patients with cystic fibrosis ( CF). Methods: Twenty patients with CF ( aged 10 - 23 years) were randomised to age and sex matched treatment and control groups. The treatment group received daily subcutaneous injections of 1 IU/ kg/ wk rGH for 12 months. Pulmonary function ( forced expiratory volume in one second ( FEV1) and airway resistance), exercise capacity measured with a bicycle ergometer, body composition ( dual energy x ray absorptiometry), and weight were assessed at the beginning of the study and after 6 and 12 months. Results: rGH treatment did not improve weight and pulmonary function, but lean body mass increased significantly in the treatment group. Exercise capacity increased in the treatment group from 143 ( 16) W ( mean ( SD)) to 164 ( 19) W after 12 months of rGH treatment. Conclusion: Treatment of CF patients with rGH for one year improved the exercise capacity significantly but not pulmonary function. The improved exercise capacity needs confirmation in a larger population before such an expensive treatment is justified.