Endocrine Abstracts

Efficacy of rhGH for treatment of pediatric GH deficiency (PGHD) may depend on several variables at treatment initiation (age, body weight, height velocity (HV), IGF-I SDS, skeletal maturation, GHmax), but may be compromised by poor adherence to required daily injections. Somavaratan is a novel, long-acting rhGH fusion protein that demonstrated clinically meaningful improvements in HV and IGF-I concentration in PGHD patients. Over 200 pre-pubertal GHD subjects have enrolled in...

Requirement for daily rhGH injections is a treatment burden that can compromise adherence and efficacy in patients with GHD. Somavaratan is a novel long-acting rhGH fusion protein in clinical development for pediatric and adult GHD. In a multicenter, randomized Phase 1b/2a study, somavaratan significantly improved height velocity (HV) and IGF-I in pre-pubertal children with GHD. Preliminary efficacy and safety in subjects who have completed 3 years of somavaratan treatment are...

Background: Survivors with Fontan circulation suffer from chronic systemic hypoperfusion resulting in end-organ injury. Little is known about the effects of these hemodynamic perturbations on bone. We hypothesized that chronic Fontan circulation (>5 years after surgery) would be associated with bone deficits.Methods: Peripheral quantitative computed tomography (pQCT) was performed on 10 Fontan patients (seven males, 11.8±1.7 years) and 11 health...

Introduction: Growth hormone (GH) replacement therapy is safe and confers significant benefits in body composition, bone health, cardiometabolic risk factors, and quality of life in adults with GH deficiency. However, longer term safety and efficacy data are limited by the lack of consistency in data collection between studies. The study aim was to develop a simple global register by standardising and developing a minimum data set (MDS) to facilitate international collection o...