Research supports the benefits of growth hormone (GH) therapy for people with PWS, with more than two decades of research and clinical experience providing reassuring evidence of safety. Like any treatment, GH requires appropriate screening and monitoring, particularly for health conditions that are already common in PWS.
GH therapy, when started in infants, significantly improves outcomes
Long-Term Growth Hormone Therapy changes the Natural History of Body Composition and Motor Function in Children with PWS (Carrel et al.) is a 5-year follow-up to a 2004 study of growth hormone in young children. The original study, Carrel et al, 2004, reported improved mobility and body composition in young children who had started GH as infants or toddlers.
5 years later, the investigators compared them to another group of children, matched for age, who did not receive GH. Looking at a battery of tests including height, body fat, “good” and “bad” cholesterol, motor strength and agility, those kids taking GH from a young age came out on top for all measures. Given the magnitude of the benefit (highly significant), and the good safety profile, the authors conclude that GH therapy, started in infants, can meaningfully improve growth, body composition, and development.
GH improves body composition of children
In the publication Efficacy and safety of long-term continuous growth hormone treatment in children with Prader-Willi syndrome, the authors followed 55 children in the Netherlands over 4 years of GH therapy.
Height increased and became similar to peers, and body composition improved, with less body fat and stabilized lean body mass. No deleterious effects were noted.
GH does not worsen scoliosis
Scoliosis is common in PWS, and it may develop during the same years that a child is receiving growth hormone.
An additional study by the same group mentioned above looked specifically at the risk of scoliosis associated with GH therapy in PWS. That study compared children on GH to those who did not receive GH and saw no difference in the rate or severity of scoliosis. They conclude (as others have previously Nagai, 2006) that scoliosis is not a reason to stop or change GH therapy.
GH does not increase the risk of cancer
A large post-marketing safety study followed 50,000+ children who received growth hormone between 1985 and 2006. Only about 1% had PWS; the remainder had growth hormone deficiency due to a variety of medical issues.
A major concern when GH first came into clinical use in children was that it would increase the risk of cancer. This study does not support that concern. Fewer than the expected number of children in this large group developed leukemia (3 cases were observed and 5.6 were expected in a group of this size).
Breathing should be monitored during GH therapy
Breathing problems, including obstructive sleep apnea, are common in PWS, whether or not a person is receiving GH. Because GH may increase the size of tonsils and adenoids, breathing should be evaluated before starting GH and monitored during treatment.
If sleep apnea or other breathing concerns develop, they should be evaluated and treated appropriately. Breathing problems do not automatically mean GH must be stopped.
The bottom line
More than two decades of research and clinical experience support the benefits and general safety of use of GH beginning in infancy and extending through adulthood.
Learn more about the importance of growth hormone therapy for PWS.
