Recombinant Human Growth Hormone in the Treatment of Patients With Cystic Fibrosis

Abstract

CONTEXT: Recombinant human growth hormone (rhGH) improves growth in patients with growth hormone deficiency or idiopathic short stature. Its role in patients with cystic fibrosis (CF) is unclear. OBJECTIVE:To review the effectiveness of rhGH in the treatment of patients with CF. METHODS:Medline and the Cochrane Central Register of Controlled Trials were searched from the earliest date through April 2010. Randomized controlled trials, observational studies, systematic reviews/ meta-analyses, or case reports were included if rhGH therapy was administered to patients with CF and data on prespecified harms, intermediate outcomes, or final health outcomes were reported. When applicable, end points were pooled by using a random-effects model. The overall body of evidence was graded for each outcome as insufficient, low, moderate, or high. RESULTS:Ten unique controlled trials (n ϭ 312) and 8 observational studies (n ϭ 58) were included. On quantitative synthesis of controlled trials, several markers of pulmonary function, anthropometrics, and bone mineralization were significantly improved versus control. Results of single-arm observational studies for the aforementioned outcomes were generally supportive of findings in clinical trials. There is insufficient evidence to determine the effect of rhGH on intravenous antibiotic use during therapy, pulmonary exacerbations, healthrelated quality-of-life, bone consequences, or total mortality, but moderate evidence suggests that rhGH therapy reduces the rate of hospitalization versus control.CONCLUSIONS: rhGH improved almost all intermediate measures of pulmonary function, height, and weight in patients with CF. Improvements in bone mineral content are also promising. However, with the exception of hospitalizations, the benefits on final health outcomes cannot be directly determined at this time.

A multi‐center controlled trial of growth hormone treatment in children with cystic fibrosis

“…Those data were similar to the composite data from the other nine controlled and eight observational studies of rhGH in CF included in their analysis. 21 Our reported improvements in growth and weight did not persist beyond the 12-month treatment period. The decline in height velocity is consistent with the catch-down growth described following discontinuation of rhGH in growing children.…”

“…In fact, better linear growth may be associated with better lung growth (higher lung volumes) and better lung function, independent of the beneficial effect of weight gain. 21 …”

“…15 Previous studies of rhGH in CF individuals demonstrated improvements in growth velocity, weight gain, indices of metabolism, and clinical status. 16–21 However, these studies had small sample sizes or short duration of therapy that warranted further investigation in a controlled clinical trial. Further, due to concerns about precipitating CF-related diabetes, more data on rhGH-related development of glucose intolerance and diabetes were needed.…”

Hormônio de crescimento em crianças e adolescentes com fibrose cística

“…Os autores concluíram que a utilização do rhGH melhorou o peso, a estatura, a massa óssea e quase todas as medidas de função pulmonar, além de diminuir o número de internações hospitalares. No entanto, com a exceção das internações, os benefícios nos desfechos finais à saúde não podem ser diretamente determinados até este momento devido ao pouco tempo de uso do rhGH (34).…”

3.19 Nutrition in Cystic Fibrosis

“…The efficacy of growth hormone therapy in CF has recently been reviewed [19] . While growth parameters and pulmonary function seem to improve in treated patients, the overall benefits to health cannot be determined from the moderate evidence available.…”