title: A multi-center controlled trial of growth hormone treatment in children with cystic fibrosis.
abstract: OBJECTIVE
  We evaluated safety and efficacy of recombinant human growth hormone (rhGH) for improving growth, lean body mass (LBM), pulmonary function, and exercise tolerance in children with cystic fibrosis (CF) and growth restriction.
  
  
  METHODS
  Multicenter, open-label, controlled clinical trial comparing outcomes in prepubertal children <14 years with CF, randomized in a 1:1 ratio to receive daily rhGH (Nutropin AQ) or no treatment (control) for 12 months, followed by a 6-month observation (month 18). Safety was monitored at each visit, including assessments of glucose tolerance.
  
  
  RESULTS
  Sixty-eight subjects were randomized (control n = 32; rhGH n = 36). 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.
  
  
  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.
authors: University of Massachusetts Medical School, Worcester, Massachusetts 01655, USA. michael.stalvey@umassmed.edu
authors: Michael S
authors: MS
authors: Stalvey
authors: Ran D
authors: RD
authors: Anbar
authors: Michael W
authors: MW
authors: Konstan
authors: Joan R
authors: JR
authors: Jacobs
authors: Bert
authors: B
authors: Bakker
authors: Barbara
authors: B
authors: Lippe
authors: David E
authors: DE
authors: Geller
mesh_terms: D000293:Adolescent; D001823:Body Composition / Q000187:drug effects*; D001827:Body Height / Q000187:drug effects*; D001835:Body Weight / Q000187:drug effects; D002648:Child; D002675:Child, Preschool; D003550:Cystic Fibrosis / Q000188:drug therapy*; D017079:Exercise Tolerance / Q000187:drug effects*; D005260:Female; D005541:Forced Expiratory Volume / Q000187:drug effects; D019382:Human Growth Hormone / Q000627:therapeutic use*; D006801:Humans; D008168:Lung / Q000187:drug effects; D008297:Male; D011994:Recombinant Proteins / Q000627:therapeutic use; D016896:Treatment Outcome
chemical_list: D011994:Recombinant Proteins; D019382:Human Growth Hormone