Growth hormone improves clinical status in prepubertal children with cystic fibrosis: Results of a randomized controlled trial

Objectives: We conducted a 1-year randomized controlled trial to test the h ypothesis that growth hormone (GH) improves the clinical status of children with cystic Fibrosis. Study design: Nineteen prepubertal children were randomized to control (Non TX, n = 9) or to daily injections of CH (0.3 mg/kg/wk) (GHTX, n = 10) for 1 year. Every 3 months height, weight, and lean tissue mass were measured. C aloric intake, resting energy expenditure, pulmonary function, and respirat ory muscle strength were measured every, 6 months, as were total number of hospitalizations and courses of outpatient intravenous antibiotics. Results: The GHTX group had significantly greater height, height velocity ( NonTX = 3.8 +/- 1.4 cm/y, GHTX = 8.1 +/- 2.4 cm/y; P = .002), weight, weigh t velocity (NonTX = 2.1 +/- 0.9 kg/y, GHTX 4.5 +/- 1.1 kg/y; P = .004), and change in lean tissue mass (NonTX = 2.1 +/- 1.6 kg, GHTX = 4.7 +/- 1.7 kg; P = .01) analyzed by the Student/test. The GHTX group had significant impr ovement in delta forced vital capacity compared with the year before study, and respiratory muscle strength improved. The number of hospitalizations a nd outpatient intravenous antibiotic courses significantly decreased in the GHTX group but did not change in the NonTX group. No subject had developme nt of cystic fibrosis-related diabetes. Conclusions: Results of the first randomized controlled trial of GH treatme nt in cystic fibrosis indicate that CH improves growth and clinical status.