ISSN 1662-4009 (online)

ey0018.4-13 | New Paradigms | ESPEYB18

4.13 A Genome-wide pharmacogenetic study of growth hormone responsiveness

A Dauber , Y Meng , L Audi , S Vedantam , B Weaver , A Carrascosa , K Albertsson-Wikland , M Ranke , A Jorge , J Cara , MP Wajnrajch , A Lindberg , C Camacho-Hübner , JN Hirschhorn

J Clin Endocrinol Metab. 2020;105:3203–3214. doi: 10.1210/clinem/dgaa443. PMID: 32652002The authors performed a large genome-wide association study (GWAS) to assess the role of common genetic variants in the response to GH therapy. A total of 614 children treated with GH were included: 276 with idiopathic GHD, 297 with ISS, and 41 born SGA. The findings implicate some novel mechanisms...

ey0020.2-8 | Long-Acting Growth Hormone (LAGH) | ESPEYB20

2.8. Efficacy and safety of weekly somatrogon vs daily somatropin in children with growth hormone deficiency: A phase 3 study

CL Deal , J Steelman , E Vlachopapadopoulou , R Stawerska , LA Silverman , M Phillip , HS Kim , C Ko , O Malievskiy , JF Cara , CL Roland , CT Taylor , SR Valluri , MP Wajnrajch , A Pastrak , BS Miller

Brief summary: This 12-months randomized, controlled, phase 3 study compared the efficacy and safety of once-weekly Somatrogon 0.66 mg/kg/week with once-daily somatropin in prepubertal children with GHD. The efficacy of once-weekly Somatrogon was noninferior to once-daily somatropin, with similar safety and tolerability profiles.Somatrogon (MOD-4023) is a long-acting rhGH recently approved by the European Medicines Agency for the treatment of children wi...

ey0020.2-2 | Important for Clinical Practice | ESPEYB20

2.2. Safety and efficacy of pediatric growth hormone therapy: results from the full KIGS cohort

M Maghnie , MB Ranke , ME Geffner , E Vlachopapadopoulou , L Ibanez , M Carlsson , W Cutfield , R Rooman , R Gomez , MP Wajnrajch , A Linglart , R Stawerska , PE Clayton , F Darendeliler , ACS Hokken-Koelega , R Horikawa , T Tanaka , HG Dorr , K Albertsson-Wikland , M Polak , A Grimberg

Brief summary: This study reports data from the full Kabi/Pfizer International Growth Database (KIGS), a large international database including 83803 children treated with rhGH for GHD and non-GHD conditions. The data confirm that rhGH is effective in increasing short-term height gain and adult height in various conditions and validate rhGH safety, with no new serious adverse events reported.KIGS (Kabi/Pfizer International Growth Database), first establi...