Growth Hormone Treatment in Children with Prader-Willi Syndrome: Three Years of Longitudinal Data in Prepubertal Children and Adult Height Data from the KIGS Database

Short stature is a common feature of children with Prader-Willi syndrome (PWS) as well as hypotonia, hyperphagia, obesity, hypogonadism, behavioral disturbances and hypothalamic dysfunction. Alterations in the GH/IGF1 axis are common in patients with PWS, GH deficiency occurring in approximately 74% and IGF-1 deficiency in nearly 100% [1]. These patients show reduced spontaneous 24-hour GH secretion, insufficient response to GH stimulation testing, sub-normal levels of IGF-1 and reduced pubertal spurt. GH treatment increases height in PWS children and, besides its positive effects on growth, improves body composition, motor development, energy expenditure, bone mineralization and cardiovascular health. Although GH is widely used in PWS patients, the available evidence on long-term efficacy and safety on a large cohort of GH-treated PWS children are limited.