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. 2003 Dec;88(12):1078–1081. doi: 10.1136/adc.88.12.1078

Prospective randomised treatment with recombinant human growth hormone in cystic fibrosis

A Schibler 1, R von der Heiden 1, P Birrer 1, P Mullis 1
PMCID: PMC1719415  PMID: 14670773

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.

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Selected References

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