Height Outcome of Recombinant Human Growth Hormone Treatment in Achondroplasia Children: A Meta-Analysis

Background/Aims: Although recombinant human growth hormone (rhGH) is not approved to treat short stature of achondroplasia (ACH), some studies suggested growth improvement during short-term rhGH treatment. Methods: A meta-analysis of rhGH therapy efficacy in ACH children was performed. Results: From 12 English-language studies, 558 (54.0% males) rhGH-treated ACH children were enrolled. Administration of rhGH (median dosage 0.21 mg/kg/week; range 0.16-0.42 mg/kg/week) improved height (Ht) from baseline [-5.069 standard deviation score (SDS; 95% CI-5.109 to-5.029); p < 0.0001] to 12 [-4.325 SDS (95% CI-4.363 to-4.287); p < 0.0001] and 24 months [-4.073 SDS (95% CI-4.128 to-4.019); p < 0.0001]. Then, Ht remained approximately constant up to 5 years [-3.941 SDS (95% CI-4.671 to-3.212); p < 0.0001]. Conclusions: In ACH children, rhGH treatment increased Ht from-5.0 to-4.0 SDS during 5 years, but insufficient data are available on both the adult Ht and the changes of body proportions.