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A multicenter, randomized, double-blind, placebo-controlled trial to evaluate the metabolic and respiratory effects of growth hormone in children with cystic fibrosis.
Pediatrics. 2007 Jun; 119(6):e1230-8.Ped

Abstract

OBJECTIVE

Positive effects of growth hormone therapy on growth, nutritional status, and lung function have been observed in patients with cystic fibrosis, but the current evidence is based on unblinded studies that involved a small number of patients. This trial was designed as a multicenter, randomized, placebo-controlled, double-blind study to assess the efficacy and safety of 2 dosages of growth hormone in cystic fibrosis.

METHODS

Sixty-three dystrophic patients with cystic fibrosis were randomly assigned for 24 weeks to 1 of 3 treatment arms: growth hormone dosage of 0.11 IU/kg body weight per day, growth hormone dosage of 0.21 IU/kg body weight per day, or placebo. The 24-week double-blind period was followed by an open treatment period of 24 weeks. The primary outcome measure was the change in forced expiratory volume in 1 second in percentage predicted from baseline. Secondary outcome measures were changes in height, weight, and exercise tolerance.

RESULTS

Height, growth velocity, and growth factors (insulin-like growth factor 1 and insulin-like growth factor-binding protein 3) increased significantly in both treatment groups, whereas weight gain did not differ between the growth hormone groups and placebo. A trend toward improvement in absolute forced vital capacity was observed in patients who received the higher growth hormone dosage, whereas forced expiratory volume in 1 second did not change significantly with growth hormone treatment. Maximal oxygen uptake during peak exercise increased significantly in treated patients. There were no significant differences in the frequency or severity of adverse effects or in the incidence of abnormalities in glucose metabolism.

CONCLUSIONS

These data suggest that in the group investigated, growth hormone therapy was well tolerated and had positive metabolic effects but did not result in short-term improvement of lung function in patients with cystic fibrosis.

Authors+Show Affiliations

Department of Pediatric Endocrinology and Diabetology, Children's Hospital, Charite, Berlin, Germany.No affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info available

Pub Type(s)

Comparative Study
Journal Article
Multicenter Study
Randomized Controlled Trial
Research Support, Non-U.S. Gov't

Language

eng

PubMed ID

17545356

Citation

Schnabel, Dirk, et al. "A Multicenter, Randomized, Double-blind, Placebo-controlled Trial to Evaluate the Metabolic and Respiratory Effects of Growth Hormone in Children With Cystic Fibrosis." Pediatrics, vol. 119, no. 6, 2007, pp. e1230-8.
Schnabel D, Grasemann C, Staab D, et al. A multicenter, randomized, double-blind, placebo-controlled trial to evaluate the metabolic and respiratory effects of growth hormone in children with cystic fibrosis. Pediatrics. 2007;119(6):e1230-8.
Schnabel, D., Grasemann, C., Staab, D., Wollmann, H., & Ratjen, F. (2007). A multicenter, randomized, double-blind, placebo-controlled trial to evaluate the metabolic and respiratory effects of growth hormone in children with cystic fibrosis. Pediatrics, 119(6), e1230-8.
Schnabel D, et al. A Multicenter, Randomized, Double-blind, Placebo-controlled Trial to Evaluate the Metabolic and Respiratory Effects of Growth Hormone in Children With Cystic Fibrosis. Pediatrics. 2007;119(6):e1230-8. PubMed PMID: 17545356.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - A multicenter, randomized, double-blind, placebo-controlled trial to evaluate the metabolic and respiratory effects of growth hormone in children with cystic fibrosis. AU - Schnabel,Dirk, AU - Grasemann,Corinna, AU - Staab,Doris, AU - Wollmann,Hartmut, AU - Ratjen,Felix, AU - ,, PY - 2007/6/5/pubmed PY - 2007/7/10/medline PY - 2007/6/5/entrez SP - e1230 EP - 8 JF - Pediatrics JO - Pediatrics VL - 119 IS - 6 N2 - OBJECTIVE: Positive effects of growth hormone therapy on growth, nutritional status, and lung function have been observed in patients with cystic fibrosis, but the current evidence is based on unblinded studies that involved a small number of patients. This trial was designed as a multicenter, randomized, placebo-controlled, double-blind study to assess the efficacy and safety of 2 dosages of growth hormone in cystic fibrosis. METHODS: Sixty-three dystrophic patients with cystic fibrosis were randomly assigned for 24 weeks to 1 of 3 treatment arms: growth hormone dosage of 0.11 IU/kg body weight per day, growth hormone dosage of 0.21 IU/kg body weight per day, or placebo. The 24-week double-blind period was followed by an open treatment period of 24 weeks. The primary outcome measure was the change in forced expiratory volume in 1 second in percentage predicted from baseline. Secondary outcome measures were changes in height, weight, and exercise tolerance. RESULTS: Height, growth velocity, and growth factors (insulin-like growth factor 1 and insulin-like growth factor-binding protein 3) increased significantly in both treatment groups, whereas weight gain did not differ between the growth hormone groups and placebo. A trend toward improvement in absolute forced vital capacity was observed in patients who received the higher growth hormone dosage, whereas forced expiratory volume in 1 second did not change significantly with growth hormone treatment. Maximal oxygen uptake during peak exercise increased significantly in treated patients. There were no significant differences in the frequency or severity of adverse effects or in the incidence of abnormalities in glucose metabolism. CONCLUSIONS: These data suggest that in the group investigated, growth hormone therapy was well tolerated and had positive metabolic effects but did not result in short-term improvement of lung function in patients with cystic fibrosis. SN - 1098-4275 UR - https://www.unboundmedicine.com/medline/citation/17545356/A_multicenter_randomized_double_blind_placebo_controlled_trial_to_evaluate_the_metabolic_and_respiratory_effects_of_growth_hormone_in_children_with_cystic_fibrosis_ L2 - http://pediatrics.aappublications.org/cgi/pmidlookup?view=long&pmid=17545356 DB - PRIME DP - Unbound Medicine ER -