Early growth hormone treatment supports physical development beyond height alone. Research has found benefits in body composition, muscle strength, mobility, and motor development.
Another study by de Lind van Wijngaarden and colleagues followed 55 children in the Netherlands over four years of growth hormone therapy. Children had increases in height similar to their peers, along with improvements in body composition, including decreased body fat and more stable lean body mass.
Other Reported Benefits
- Respiratory function
- Physical performance
- Levels of resting energy expenditure
- Cholesterol levels
- Bone mineral density
- Head circumference
Cognitive Improvements
A study by Dykens and colleagues found that children with PWS who received growth hormone had higher verbal and overall IQ scores, along with stronger communication and daily living skills, than children who had not received treatment.
Children who started growth hormone before 12 months of age also had higher IQ scores than those who started between ages 1 and 5.
Another long-term study found that children who started growth hormone earlier in infancy had better adaptive skills over time, including skills used in everyday life.
Early Development and Motor Skills
A study by Donze and colleagues followed 63 infants and toddlers with PWS during three years of growth hormone treatment. Early developmental and motor skills improved over that time, helping narrow the developmental gap with same-age peers. Children who started growth hormone treatment at a younger age showed greater gains.
Growth hormone therapy has a well-established safety profile in PWS, but regular monitoring is an important part of treatment. Healthcare providers watch for changes in sleep and breathing, blood sugar, IGF-1 levels, thyroid function, and scoliosis, among other factors.
Sleep and Breathing
Sleep-disordered breathing, including sleep apnea, is common in people with PWS. A sleep study is generally recommended before starting growth hormone therapy and again after treatment begins.
One study looked at changes in sleep in the six weeks following initiation of growth hormone therapy. Most patients had less apnea, but about one-third had worsening of sleep disturbances generally associated with enlarged tonsils and upper respiratory infections.
Any new or worsening snoring, pauses in breathing, or other changes in sleep should be discussed with the care team.
Blood Sugar and Metabolism
Growth hormone can affect how the body uses insulin and glucose. Blood sugar should be monitored during treatment, particularly in people who have obesity or other risk factors for diabetes. Regular monitoring can help identify and address changes early.
Scoliosis
Scoliosis is common in people with PWS, whether or not they receive growth hormone. Research has not found that growth hormone increases the risk of scoliosis in people with PWS, but the spine should be monitored as children grow so changes can be identified and treated when needed.
Does Growth Hormone Increase Mortality Risk?
Research has not found that growth hormone treatment itself increases mortality risk in PWS. People with PWS do have a higher risk of serious health complications and premature death than the general population, but long-term research suggests that mortality is more closely linked to other health conditions. This reinforces the importance of careful screening and ongoing monitoring during treatment.
When Growth Hormone May Not Be Appropriate
There are factors that should exclude growth hormone therapy as a therapeutic option for some individuals. These may include:
- Severe obesity
- Uncontrolled diabetes mellitus
- Untreated severe obstructive sleep apnea
- Active cancer
- Active psychosis
A PWS-experienced endocrinologist can help determine whether growth hormone is appropriate and whether any health concerns should be addressed before treatment begins or resumes.
Possible Side Effects
Growth hormone therapy is generally well tolerated, but side effects can occur. These may include:
- Swelling caused by fluid retention
- Joint pain
- Headache
- New or worsening sleep-disordered breathing
- Changes in blood sugar or insulin sensitivity
- Changes in thyroid hormone levels
New hip or knee pain, limping or other changes in walking, or severe or persistent headaches with vision changes should be reported to the care team promptly, as these can be signs of less common complications.
Ongoing Monitoring
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Regular follow-up helps make sure growth hormone therapy remains safe and effective. Depending on age and individual needs, monitoring may include:
- Height, weight, growth, and body composition
- IGF-1 levels
- Blood sugar and diabetes risk
- Thyroid function
- Sleep and breathing
- Scoliosis
- Puberty and bone health
- Cholesterol and liver health, when appropriate
- Changes in behavior or mental health
The timing and type of monitoring will vary based on age, health history, and response to treatment. The growth hormone dose may be adjusted over time based on these results and the individual’s response to treatment.
Clinical Guidelines
International consensus guidelines provide recommendations for starting, monitoring, and managing growth hormone therapy in PWS.
