title: Recombinant growth hormone therapy for cystic fibrosis in children and young adults.
abstract: BACKGROUND
  Cystic fibrosis is an inherited condition causing disease most noticeably in the lungs, digestive tract and pancreas. People with cystic fibrosis often have malnutrition and growth delay. Adequate nutritional supplementation does not improve growth optimally and hence an anabolic agent, recombinant growth hormone, has been proposed as a potential intervention.
  
  
  OBJECTIVE
  To evaluate the effectiveness and safety of recombinant human growth hormone therapy in improving lung function, quality of life and clinical status of children and young adults with cystic fibrosis.
  
  
  METHODS
  We searched the Cochrane Cystic Fibrosis and Genetic Disorders Group's Trials Register comprising references identified from comprehensive electronic database searches and handsearches of relevant journals and abstract books of conference proceedings. Date of latest search: 15 May 2013.We conducted a search of relevant endocrine journals and proceedings of the Endocrinology Society meetings using Scopus and Proceedings First. Date of latest search: 15 March 2012.
  
  
  METHODS
  Randomised and quasi-randomised controlled trials of all preparations of recombinant growth hormone compared to either no treatment, or placebo, or each other at any dose (high-dose and low-dose) or route and for any duration, in children or young adults aged up to 25 years diagnosed with cystic fibrosis (by sweat test or genetic testing).
  
  
  METHODS
  Two authors independently screened papers, extracted trial details and assessed their risk of bias.
  
  
  RESULTS
  Four controlled trials were included in this review (with 161 participants in total), each with an unclear risk of bias. Analysis of data obtained from these trials shows improvement in height for all comparisons, but improvements in weight and lean tissue mass were only reported in the comparison of standard dose recombinant growth hormone versus no treatment. There is moderate improvement in one parameter of pulmonary function tests, functional vital capacity (per cent predicted) when comparing standard dose recombinant growth hormone and no treatment. Little evidence was found for improvement in quality of life. An improvement in fasting blood glucose levels was reported when comparing rhGH to placebo only. Exercise capacity improved in participants receiving standard dose recombinant growth hormone versus no treatment, but not for any other comparison. There is insufficient evidence to conclude any changes in hospitalisations, antibiotic use or significant adverse effects.
  
  
  CONCLUSIONS
  Recombinant growth hormone therapy is effective in improving the intermediate outcomes in height, weight and lean tissue mass when compared with no treatment. One measure of pulmonary function test showed moderate improvement. No significant changes in quality of life, clinical status or side-effects were observed in this review. Long-term, well-designed randomised controlled trials of recombinant growth hormone therapy in patients with cystic fibrosis are required prior to evaluation of human growth hormone treatment for routine use in patients.
authors.affiliation: Haverstraw Pediatrics, Haverstraw, NY, USA. vidhuthaker@gmail.com.
authors.forename: Vidhu
authors.initials: V
authors.lastname: Thaker
authors.forename: Alexandra L
authors.initials: AL
authors.lastname: Haagensen
authors.forename: Ben
authors.initials: B
authors.lastname: Carter
authors.forename: Zbys
authors.initials: Z
authors.lastname: Fedorowicz
authors.forename: Brian W
authors.initials: BW
authors.lastname: Houston
mesh_terms: D000293:Adolescent; D001786:Blood Glucose / Q000378:metabolism; D001827:Body Height / Q000187:drug effects*; D015992:Body Mass Index; D001835:Body Weight / Q000187:drug effects / Q000502:physiology; D002648:Child; D003550:Cystic Fibrosis / Q000150:complications / Q000188:drug therapy*; D017079:Exercise Tolerance / Q000187:drug effects / Q000502:physiology; D005215:Fasting / Q000097:blood; D019382:Human Growth Hormone / Q000627:therapeutic use*; D006801:Humans; D011788:Quality of Life; D016032:Randomized Controlled Trials as Topic; D011994:Recombinant Proteins / Q000627:therapeutic use; D014797:Vital Capacity / Q000187:drug effects / Q000502:physiology; D055815:Young Adult
chemical_list: D001786:Blood Glucose; D011994:Recombinant Proteins; D019382:Human Growth Hormone