A Treatment/Control Group Study of Growth Hormone Treatment

Introduction: This is a report on the first year of a 2-year study measuring the behavioral impact of growth hormone replacement therapy (GHRT) in a population of persons with Prader-Willi syndrome (PWS). A psycho-social burden associated with short statute previously has been identified in a non-PWS population. In addition to short stature and morbid obesity, this syndrome is characterized by a deteriorating neurobehavioral/neu-rocognitive picture that intensifies in quantity and severity over time. Thus, both positive behavioral change and behavioral deterioration were examined. Methods: A 2-year controlled study with control group cross-over in the second year was utilized. Fifty-four consecutive children with genetically confirmed PWS were enrolled and after a 6-month growth assessment were randomized into a 60:40 treatment:control ratio. Treatment consisted of nutropin (Genentech) 1 mg/m2/day. A modified Offord Survey Diagnostic Instrument was used to monitor behavior at 6-month intervals. Family stress was monitored with the Family Inventory of Life Events, and marital functioning was measured with the Snyder Marital Satisfaction Inventory. Results: No differences were found between the treatment and control groups or within each group across time for attentional symptoms, anxiety, depression, violence, and psychotic symptoms. Within the treatment group, a significant reduction was reported for both depressive and obsessive symptoms, as well as a reduction in skin-picking. No behavioral deterioration was reported. Conclusions: We conclude that in addition to the previously detailed improvements in physical parameters for these children, behavioral improvement including a lack of predictable behavioral deterioration during the treatment period is a strong argument for the use of GHRT for persons with this difficult syndrome.▪ The Endocrinologist 2000; 10: 31S-37S