Growth hormone treatment of children with Prader-Willi syndrome affects linear growth and body composition favourably

Abstract

We have compared the growth and the body composition in children with Prader-Willi syndrome (PWS) with and without growth hormone treatment (recombinant GH 0.1 IU/kg/day) after a 1-y period. Twenty-nine prepubertal children with PWS, with mean body mass index (BMI) SDS of 2.2, and 10 (control) healthy obese children with mean BMI SDS of 5.6, underwent 24-h frequent blood sampling. Both PWS and control obese children had low and similar GH levels (0.7 microg/l +/- 0.4SD). Serum IGF-I levels, however, were significantly lower in children with PWS (-1.5SDS +/- 0.8SD vs -0.2SDS +/- 0.8SD). The 29 PWS children were randomized into 2 groups of 15 and 14 subjects for GH treatment and no treatment, respectively. Height velocity increased from -1.9SDS to + 6.0SDS in the treated group (p < 0.001) and decreased from -0.1SDS to -1.4SDS in the control PWS group during the study year. BMI decreased significantly for the treated group (+3.0SDS to +2.0SDS). Relative fat mass decreased significantly, while fat-free mass increased (p < 0.001) for the treated group. No significant changes were noticed in body composition in the control PWS group. In conclusion, the low spontaneous 24-h GH secretion, regardless of body weight, and the exceptional response to growth hormone treatment together with the finding of low IGF-I levels suggest that growth hormone deficiency is a common feature of PWS, as a result of hypothalamic dysfunction. Treatment with growth hormone is beneficial for the majority of PWS children.

Auxological and Endocrine Evolution of 28 Children with Prader-Willi Syndrome: Effect of GH Therapy in 14 Children

“…The effect of GH on both growth velocity and BMI has already been reported in recent studies [13, 14, 15, 16, 17, 18, 19, 20]. In our retrospective study, we have a longer duration of GH therapy, and we could show the sustained benefit of this treatment, although we did perform precise studies using DEXA (dual absorptiometry) of body composition [14]and of fat before and after 6 and 12 months of GH treatment in children with PWS [14].…”

“…Growth retardation leads to a reduced adult height [7, 10, 11, 12], and GH therapy appears to be an important tool in the management of these children, as reported in recent papers, not only for its growth-promoting effects, but also for its effect on body composition and muscle mass [13, 14, 15, 16, 17, 18, 19, 20]. These effects had been reported after short-term treatment, 1–2 years, in prospective studies.…”

Effects of Growth Hormone Therapy on Glucose Metabolism and Insulin Sensitivity Indices in Prepubertal Children with Prader-Willi Syndrome

“…However, it has been demonstrated that in PWS patients GH treatment (GHT) reduces fat mass and increases body free fat mass percentage [4,12,13,14], improves respiratory muscle strength and endurance [15], and increases resting metabolic rate as well as physical strength and agility[14]. Changes in behaviour have also been observed [16] and beneficial effects on growth velocity have been demonstrated in many studies [6,12,13,14]. …”

Early Diagnosis and Multidisciplinary Care Reduce the Hospitalization Time and Duration of Tube Feeding and Prevent Early Obesity in PWS Infants

“…One of the main endocrine issues in patients with PWS is the investigation of GH status, mainly because of their obesity [23, 28, 29]. There was no obesity at this period of life and GH secretion could therefore be studied appropriately.…”

“…GH not only accelerates growth but also improves body composition [19], the level of physical activity, and perhaps cognitive functions [20, 21]. Most of the studies on GH treatment were performed in children older than 3 years [16, 22, 23], with only two recent publications showing an interest in toddlers [20, 24]. …”