hrp0095fc10.2 | GH and IGFs | ESPE2022

The first-year growth response to once-weekly growth hormone (GH) treatment can be predicted from the pre-treatment blood transcriptome in children with GH deficiency (GHD)

Garner Terence , Clayton Peter , Murray Philip , Bagci Ekaterine , Højby Michael , Stevens Adam

Growth response to daily GH treatment can be predicted using pre-treatment gene expression profiles.1 Once-weekly GH treatment potentially reduces the burden of daily injections2 and thus may be a major advancement in care for patients with GHD, vs standard, daily GH treatment. Here we investigate the prediction of first-year growth response based on pre-treatment blood transcriptome in children with GHD undergoing treatment with daily or once-weekly GH. ...

hrp0095p1-305 | Growth and Syndromes | ESPE2022

Once-Weekly Somapacitan vs Daily Growth Hormone in Children Born Small for Gestational Age: 1-year Results from a Randomised Phase 2 Trial

Juul Anders , Backeljauw Philippe , Bakhtadze Bagci Ekaterine , Højby Michael , Kawai Masanobu , Juul Kildemoes Rasmus , Linglart Agnès , Zuckerman-Levin Nehama , Horikawa Reiko

Treatment of short stature in children born small for gestational age (SGA) requires daily growth hormone (GH) injections that can be burdensome for patients and caregivers. Once-weekly somapacitan is a long-acting GH in phase 3 development for replacement therapy in children with GH deficiency. We report the 52-week results of the first phase 2, multinational, randomised, open-label, controlled, dose-finding trial (NCT03878446) investigating efficacy and safety of somapacitan...