Prader–Willi syndrome and growth hormone treatment in children and adults

Abstract

Prader–Willi syndrome (PWS) is a complex multisystem genetic disorder, including severe neonatal hypotonia, early onset of hyperphagia and development of morbid obesity, short stature, hypogonadism, learning disabilities, behavioral problems and psychiatric phenotypes. PWS patients’ body composition resembles that of individuals with growth hormone deficiency, including short stature and reduced lean body mass with concomitant increased fat mass. Previous studies suggest that growth hormone (GH) therapy may be beneficial in children and adults with PWS. While short-term benefits of treatment with GH have been shown, whether these beneficial effects are dose dependent and persist or wane with prolonged therapy remains uncertain in adults. This review focuses on the benefits, safety and adverse effects of GH treatment in children and adults.