Early diagnosis and optimal clinical care can improve outcomes for people with PWS, and one proven intervention is growth hormone therapy. This treatment was approved by the US Food and Drug Administration for children with PWS in June 2000. Since then, several benefits have been reported, and growth hormone therapy for PWS has become an important step after an initial diagnosis.
What is growth hormone therapy?
Human growth hormone is produced naturally by the pituitary gland in the brain. It is released periodically throughout the day and particularly in the first few hours of sleep. The hormone spurs growth in children and adolescents through the action of insulin like growth factor-1 (IGF-1), and also helps regulate body composition, fluid balance, fat and carbohydrate metabolism, and heart function.
If the pituitary gland does not produce enough growth hormone, or if the growth hormone is not effectively used by the body, signs of growth hormone deficiency – like what is seen in people with PWS – can appear. Symptoms of growth hormone deficiency can include short stature, increased fat mass, decreased muscle mass, decreased stamina and low mood. Fortunately, growth hormone deficiency can be treated.
With growth hormone therapy for PWS, a synthetic growth hormone is injected in the body to compensate for insufficient production and release of growth hormone from the pituitary gland.
In PWS, growth hormone is effective not only in increasing height, but also in:
- Decreasing body fat
- Increasing muscle mass
- Improving weight distribution
- Increasing stamina
- Improving bone health
In addition to these positive effects on growth and body composition, growth hormone therapy produces positive effects on development, behavior, and quality of life in those with PWS.
The use of the treatment is widespread in PWS. As reported in the Global PWS Registry, which has more than 2,000 registered participants diagnosed with PWS, 91% of respondents say their participant is using or has used growth hormone. Of those participants who have used growth hormone, 71% began before age 2, and 93% perceive they have received benefit from the drug.