ESPE Yearbook of Paediatric Endocrinology

J Clin Endocrinol Metab, 2022. 107(5): p. 1357-1367. PMID: 34964458Brief Summary: This multicenter, randomized, controlled, phase 2 study compared the effects of once-weekly long-acting growth hormone (GH) somapacitan versus daily GH administered to GH-deficient (GHD) children over a period of 156 weeks. Efficacy estimates were height velocity (HV; cm/y) at year 3 and changes from baseline to end for height SDS, HV SDS, IGF-I SDS, and insulin-li...

Eur J Endocrinol 2021;185:K7–K11 doi: 10.1530/EJE-21-0312Brief Summary: Steroid profiling of 10 placentas of healthy full-term neonates (five male and five female) using liquid chromatography-tandem mass spectrometry revealed the presence of C11-oxy C19 steroids (androgens) in the fetal-placental unit at term – pointing to alternative androgen pathways in this unit.<p cla...

Brief summary: This is the first multi-national phase 2 dose finding trial aimed at investigating efficacy and safety of the long acting GH (Somapacitan) with daily GH in short children born SGA. 62 GH treatment-naïve short children born SGA were enrolled in the study. Efficacy was valuated with estimated mean annualized height velocity (HV) after 26 weeks of follow-up. Somapacitan showed similar efficacy of daily rhGH. The study will be extended for further 4 years in or...

To read the full abstract: J Clin Endocrinol Metab ;105(4):e1847–e1861.The REAL 3 multicenter randomized, controlled, double-blind phase 2 study evaluated the efficacy and safety of different doses of once-weekly Somapacitan, compared to conventional once-daily GH in 59 prepubertal GHD children. The treatment period lasted 26 weeks and an additional extension to 52 weeks. At week 26, height velocity (HV) was 7.8, 10.9 and 13.1 cm/year, resp...

Brief summary: This multicenter, randomized, controlled, phase 3 study compared the effects of long-acting GH (Somapacitan; 0.16 mg/kg/wk) with daily GH (Norditropin; 0.034 mg/kg/d), in GHD children. The trial was conducted over 52 weeks, followed by an ongoing 3-year single-group extension period. Similar efficacy and safety for somapacitan compared to daily GH was demonstrated over 52 weeks of treatment.Long-acting growth hormone (LAGH) formulations ha...

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...

To read the full abstract: Horm Res Paediatr. 2019;92:1–14In March 2019, 46 international experts from 14 countries across 5 continents attended a 3-day workshop organized by the Growth Hormone Research Society (GRS) and produced this perspective on the diagnosis, management and therapy in children with short stature. In this context, this expert panel tackled almost all aspects related to the management of children with short stature, prov...

n.karavitaki@bham.ac.uk Eur J Endocrinol. 2022; 186: P35-P52. PMID: 35319491.Brief Summary: An international panel of experts produced this consensus statement after critical review of the existing evidence on the safety of GH replacement in cancer and intracranial tumour survivors and in patients with increased cancer risk. Current evidence does not support an association betwe...