Prepubertal children <14 years with cystic fibrosis and growth restriction. Sixty-eight subjects were randomized (control n = 32; rhGH n = 36).
Interventions
daily rhGH (Nutropin AQ) or no treatment (control) for 12 months, followed by a 6-month observation (month 18).
Outcome measures
safety and efficacy. Safety was monitored at each visit, including assessments of glucose tolerance
Main results
Mean height standard deviation score (SDS) in the rhGH group increased by 0.5 +/- 0.4 at 12 months (mean +/- SD, P < 0.001); the control group height SDS remained unchanged. Weight increased by 3.8 +/- 1.8 versus 2.8 +/- 1.5 kg, (mean +/- SD, P = 0.0356) and LBM increased by 3.8 +/- 1.8 versus 2.1 +/- 1.4 kg (P = 0.0002) in the rhGH group versus controls, respectively. Forced vital capacity increased by 325 +/- 319 in the rhGH group compared with 178 +/- 152 ml in controls (mean +/- SD, P = 0.032). Forced expiratory volume in 1 sec improved in both groups with a significant difference between groups after adjustment for baseline severity (LS mean +/- SE: rhGH, 224 +/- 37, vs. controls, 108 +/- 40 ml; P = 0.04). There was no difference between groups in exercise tolerance (6-min walk distance) at 1 year. Changes in glucose tolerance for the two groups were similar over the 12-month study period, with three subjects developing IGT and one CFRD in each group. One rhGH-treated patient developed increased intracranial pressure.
Authors' conclusions
Treatment with rhGH in prepubertal children with CF was effective in promoting growth, weight, LBM, lung volume, and lung flows, and had an acceptable safety profile.