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Community child health, public health, and epidemiology
Prospective randomised treatment with recombinant human growth hormone in cystic fibrosis
  1. A Schibler1,
  2. R von der Heiden2,
  3. P Birrer1,
  4. P E Mullis2
  1. 1Paediatric Respiratory Medicine, Department of Paediatrics, University of Bern, CH-3010 Bern, Switzerland
  2. 2Paediatric Endocrinology, Department of Paediatrics, University of Bern, CH-3010 Bern, Switzerland
  1. 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.

  • pulmonary function test
  • exercise test
  • anaerobic threshold
  • oxygen consumption
  • lean body mass
  • 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

https://doi.org/10.1136/adc.88.12.1078

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