Effects of growth hormone treatment in adults with Prader–Willi syndrome

Abstract

Objective Since limited data exist on adults with Prader-Willi syndrome (PWS) and growth hormone (GH) treatment, we report our experience on the effects of treatment for one year on body composition, physical activity, strength and energy expenditure, diet, general chemistry and endocrine data with quality of life measures. Design We studied 11 adults with PWS (6F:5M; average age = 32 yrs) over a 2 year period with GH treatment during the first year only. Electrolytes, IGF-I, glucose, thyroid, insulin, lipids, body composition, physical activity and strength, diet, energy expenditure and quality of life data were collected and analyzed statistically using linear modeling at baseline, at 12 months following GH therapy and at 24 months after treatment cessation for 12 months. Results Total lean muscle mass was significantly increased (p < 0.05) during GH treatment along with moderate-vigorous physical activity and plasma IGF-I and HDL levels, but returned to near baseline after treatment. Percent body fat decreased during the 12 months of GH treatment but increased after treatment. Conclusions Previously reported beneficial effects of GH treatment in children with PWS were found in our adults regarding body composition, physical activity and plasma HDL and IGF-I levels. Several beneficial effects diminished to near baseline after cessation of GH treatment for 12 months supporting the continuation of treatment in PWS into adulthood and possibly adults not previously treated during childhood.

Growth hormone treatment of Prader–Willi syndrome has long‐term, positive effects on body composition

“…Previously published studies have reported some effect on LDL and HDLcholesterol levels during GH treatment (6,(12)(13)(14)(15)(16)(17)(18)(19). In the present study, four of the patients were obese and, in accordance with other studies, only two patients had slight dyslipidaemia, with the exception of the patient with familiar hypercholesterolaemia.…”

“…The total duration of GH therapy was similar in the two groups, but the duration of GH therapy during adulthood was longer if GH treatment had been initiated during adulthood. This is readily explained by the fact, that the initial studies on the efficacy of GH treatment in adult PWS patients were undertaken on patients who first received treatment as adults (14)(15)(16). The continuation of GH treatment initiated during childhood did not become usual clinical practice until later.…”

“…However, a 24-month study of 15 adults with PWS showed that their body composition, muscle size and quality improved with GH treatment and their muscle strength and exercise tolerance increased independently of their GH status (22). It is generally agreed that patients with PWS have some degree of GH insufficiency, and placebo-controlled studies have all shown the beneficial effects of GH therapy (14,15). A systematic evaluation of previous publications performed by The Growth Hormone Research Society showed a positive incline towards GH treatment in adults with PWS (10).…”

The neurobiology of the Prader-Willi phenotype of fragile X syndrome

“…Most individuals with PWS, but not all, are deficient in GH and studies that were initially focused on improving growth have not only demonstrated this effect, but also improvements in metabolism, body composition, behavior and cognition (38)(39)(40)(41)(42)(43)(44). However, GH therapy on occasion can be associated with significant side effects, such as the stimulation of adenoid tissue leading to obstructive sleep apnea so it should be used carefully in the treatment of PWS.…”

Current status of growth hormone therapy in Prader–Willi syndrome

“…The analyzed studies were divided into studies of 12 month duration and studies of 24-72 months duration and the beneficial effects on body composition were found in all studies. Furthermore, a study of 11 adults with PWS was published recently also reporting beneficial effects on body composition [34].…”