Prospective randomised treatment with recombinant human growth hormone in cystic fibrosis
- 1Paediatric Respiratory Medicine, Department of Paediatrics, University of Bern, CH-3010 Bern, Switzerland
- 2Paediatric Endocrinology, Department of Paediatrics, University of Bern, CH-3010 Bern, Switzerland
- Correspondence to:
Dr A Schibler
Pediatric Intensive Care, Mater Children’s Hospital, South Brisbane 4101 QLD, Australia; andreasschiblermater.org.au
Abstract
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.
- AT, anaerobic threshold
- CF, cystic fibrosis
- COPD, chronic obstructive pulmonary disease
- DXA, dual energy x ray absorptiometry
- FEV1, forced expiratory volume in one second
- FVC, forced vital capacity
- LBM, lean body mass
- Raw, respiratory airway resistance
- rGH, recombinant growth hormone
- VCO2, carbon dioxide output
- VO2max, maximal oxygen consumption
- W, watt
