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HGH Treatment for Prader Willi Syndrome

Last Updated on by James Jackson

Prader Willi Syndrome treatment includes Human growth hormone. Prader Willi Syndrome is a condition with no cure; however, ongoing research hopes to eventually find a cure. In the meantime, growth hormone will continue to be used in the treatment of Prader Willi Syndrome.

Prader-Willi syndrome is characterized by hypothalamic dysfunction, which results in hypotonia, hypogonadism, obesity, and behavioural abnormalities. Clinically Prader Willi Syndrome symptoms resemble those of GH deficiency, and include decreased total lean body mass, IGF-I levels, and poor linear growth. There is also a marked reduction in muscle mass, which results in diminished energy expenditure, physical function, and strength. Lifelong morbidities of Prader Willi Syndrome include type 2 diabetes mellitus, osteoporosis, cardiorespiratory failure, and respiratory disorders related to obesity and hypotonia.

Diagnosing Prader Willi Syndrome

A diagnosis of Prader Willi Syndrome (PWS) is generally made by a doctor based on clinical symptoms. If an infant is born with significant hypotonia, which is muscle weakness, Prader Willi Syndrome should be suspected. A blood test can confirm the diagnosis. Methylation analysis is preferred for testing, which will detect >99 percent of all cases, as well as all the major genetic subtypes.

Treating Prader Willi Syndrome

The treatment of Prader Willi Syndrome treats the symptoms of the disorder as they occur. Growth home deficiency occurs in children and a number of adults who are suffering from Prader Willi Syndrome. In a number of studies, HGH has been shown to be beneficial for those diagnosed with Prader Willi Syndrome.

The FDA approved HGH as a treatment for Prader Willi Syndrome in June of 2000. Human growth hormone increases body height, muscle mass, stamina, bone density, and it decreases body fat. Studies have shown that HGH has also had a positive effect on behaviour and development.

While the HGH plays an important role in the treatment of Prader Willi Syndrome there are a number of symptoms that remain a challenge. The biggest obstacle remains the inability to have control over food intake, which means those with Prader Willi Syndrome are not able to live independently. To date, no treatment has occurred that can regulate appetite, and as a result continuous supervision is required to prevent a life threatening overeating condition from developing. There are several anti-obesity drugs in clinical development. Some may benefit Prader Willi Syndrome, and evaluation of these drugs in clinical trials is a priority.

Clinical Research for Treatment of Prader Willi Syndrome With HGH


Dutch Growth and Research Foundation, Rotterdam[1]

Twelve children with documented Prader-Labhart-Willi syndrome were treated with human growth hormone (24 U/m2/week) during 1 year. The children were divided into three groups: group 1: overweight and prepubertal (n=6, age 3.8-7.0 years); group 2: underweight and prepubertal (n=3, age 0.6-4.1 years); group 3: pubertal (n=3, age 9.2-14.6 years). In group 1, height increased from -1.7 SD to -0.6 SD, while weight decreased from 1.1 SD to 0.4 SD, with a dramatic drop in weight for height from 3.8 SD to 1.2 SD. Hand length increased from -1.5 SD to -0.4 SD and foot length from -2.5 SD to -1.4 SD. Body fat, measured by dual X-ray energy absorptiometry, dropped by a third, whereas muscle mass increased by a fourth.

Physical capability (Wingate test) improved considerably. The children were reported to be much more active and capable. In group 2, similar changes were seen, but weight for height increased, probably because muscle mass increase exceeded fat mass decrease. Changes in group 3 were similar as in group 1, even though far less distinct.

CONCLUSION: Growth hormone treatment in Prader-Labhart-Willi syndrome led to dramatic changes: distinct increase in growth velocity, height and muscle mass, as well as an improvement in physical performance. Fat mass and weight for height decreased in the initially overweight children, and weight for height increased in underweight children.

KIGS Pfizer International Growth Database [2]

BACKGROUND: Prader-Willi syndrome (PWS) children have impaired growth, and abnormal body composition. Previous 1-year controlled studies showed improvement of height and body composition during GH-treatment.

OBJECTIVE: To evaluate growth, body composition and body proportions during GH-treatment in a large group of PWS children.

PATIENTS: We performed a randomized controlled GH trial in 91 prepubertal PWS children (42 infants, 49 children, aged 3-14 years). After stratification for age, infants were randomized to GH-treatment (GH-group; 1 mg/m(2)/day; n = 20), or no treatment (control group; n = 22) for 1 year. In the second year all infants were treated with GH. After stratification for BMI, children > 3 years of age were randomized to GH-treatment (GH-group; 1 mg/m(2)/day; n = 27) or no treatment (control group; n = 22) for 2 years. Anthropometric parameters were assessed once in every 3 months. Body composition was measured by Dual Energy X-ray Absorptiometry.

RESULTS: Median (interquartile range, iqr) height SDS increased during 2 years of GH in infants from -2.3 (-2.8 to -0.7) to -0.4 (-1.1-0.0) and in prepubertal children from -2.0 (-3.1 to -1.7) to -0.6 (-1.1 to -0.1). In non-GH-treated children height SDS did not increase. Head circumference completely normalized during 1 and 2 years of GH in infants and children, respectively. Body fat percentage and body proportions improved in GH-treated children, but did not completely normalize. Lean body mass SDS improved compared to the control group. Serum IGF-I increased to levels above the normal range in most GH-treated children.

CONCLUSIONS: Our randomized study shows that GH-treatment in PWS children significantly improves height, BMI, head circumference, body composition and body proportions. PWS children are highly sensitive to GH, suggesting that monitoring of serum IGF-I is indicated.

Please Donate To The Prader Willi Association!

[1] Festen DA, de Lind van Wijngaarden R, van Eekelen M, Otten BJ, Wit JM, Duivenvoorden HJ, Hokken-Koelega AC

Dutch Growth and Research Foundation, Rotterdam, the Netherlands. d.festen@erasmusmc.nl
Clinical Endocrinology [2008, 69(3):443-451]
Type:  Journal Article, Multicenter Study, Randomized Controlled Trial
DOI: 10.1111/j.1365-2265.2008.03228.x

[2] Growth hormone treatment and adverse events in Prader-Willi syndrome: data from KIGS (the Pfizer International Growth Database
Craig ME, Cowell CT, Larsson P, Zipf WB, Reiter EO, Albertsson Wikland K, Ranke MB, Price DA; KIGS International Board.
Clin. Endocrinol. (Oxf) [2006]


1 Response to HGH Treatment for Prader Willi Syndrome

  1. sue says:

    My daughter has PWS, she has been on growth hormone for about 4 yrs, she is just getting bigger in weight, could this suggest it doesn’t suit her, she is 9yrs old, and I really struggle to buy clothes for her

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