Endocrine Abstracts

Background: X-linked hypophosphataemia (XLH) is a genetic condition that causes significant skeletal deformities and is associated with lifelong disability and pain. In October 2018, the NHS in England recommended burosumab, an anti-FGF23 antibody, for treating XLH with radiographic evidence of bone disease in children aged 1 year and over, and in young people with growing bones. The clinical and cost effectiveness of burosumab for treating adults with XLH is ...

Introduction: Long-acting growth hormone (LaGH) therapy has emerged as a newer treatment option for children, with the potential to improve adherence and compliance. Clinical trials have shown LaGH formulations to be effective and safe in children with GH deficiency (GHD). Somatrogon (Ngenla) is licensed for use in children with GHD over than 3 years of age.Objective: We report the real-world experience of using once wee...

Background: Pubertal progression is inhibited in central precocious puberty with the use of gonadotropin releasing hormone (GnRH) analogues. They are usually given every 10 to 12 weeks via an intramuscular depot, but more recently, a 6-monthly preparation has become available for clinical use.Aim: The aim of this project was to evaluate the efficacy and acceptability of 6-monthly triptorelin, a GnRH analogue, at a tertiary children’s hospital.<p...