ESPE Yearbook of Paediatric Endocrinology

Cochrane Database Syst Rev. 2021 Aug 23;8(8):CD008901.Division of Molecular Genetics and Department of Pediatrics, Columbia University Medical Center, New York, NY, USA. vvt2114@cumc.columbia.edu. PMID: 34424546

Brief Summary: This systematic review evaluated the efficacy and safety of rhGH therapy in improving lung function, quality of life and clinical status of children and young adults (aged up to 23 years) with cystic fibrosis (CF). Eight randomized and quasi-randomized controlled trials, collectively comprising 291 patients, were included. Short-term rhGH treatment (duration ranging from 6 to 12 months) improved anthropometric parameters (height, weight and lean body mass), with no dose-dependent effect. There was no robust evidence that rhGH treatment improved lung function, muscle strength, or quality of life. rhGH therapy increased fasting blood glucose levels, although without crossing the threshold for diagnosis of diabetes.

Cystic fibrosis (CF) is a multi-system monogenic autosomal recessive disease, caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene which encodes the CFTR protein that regulates the transport of electrolytes such as chloride and bicarbonate (1). This chronic disease severely affects lungs, digestive system and pancreas. A further hallmark of CF is poor ponderal and linear growth, closely related to lung function, nutritional status and overall health status. Multiple factors are implicated in growth impairment of CF patients. Inflammation, malnutrition and glucocorticoid treatment affect GH/IGF1 axis function (2). rhGH therapy has been proposed as a therapeutic option but the evidence supporting this use of rhGH is weak. According to a previous systematic review, short-term rhGH treatment improved height, weight, growth rate, bone mineral content and pulmonary function without adverse effects such as diabetes (3).

The current review suggests that rhGH therapy may be effective in improving anthropometric parameters such as height, weight and body mass index in children with CF whereas no consistent benefit was observed in pulmonary function, quality of life and clinical status. Though no increased incidence of diabetes was found, the observed significant rise in glucose levels raises concern about long-term detrimental effect on glucose homeostasis in patients predisposed to CF-related diabetes.

References: 1. Ratjen F, Döring G. Cystic fibrosis. Lancet. 2003;361(9358):681–9. 2. Le TN, Anabtawi A, Putman MS, Tangpricha V, Stalvey MS. Growth failure and treatment in cystic fibrosis. J Cyst Fibros. 2019;18 Suppl 2(Suppl 2):S82–s7. 3. Amorim PG, Mendes Tde B, Oliveira LS, Guerra-Júnior G, Ribeiro JD. [Growth hormone in children and adolescents with cystic fibrosis]. Arq Bras Endocrinol Metabol. 2011;55(9):671–6.