A treatment/control group study of growth hormone treatment: Impact on behavior - A preliminary look

Introduction: This is a report on the first year of a 2-year study measurin g the behavioral impact of growth hormone replacement therapy (GHRT) in a p opulation of persons with Prader-Willi syndrome (PWS), A psyche-social burd en associated with short statute previously has been identified in a non-PW S population. In addition to short stature and morbid obesity, this syndrom e is characterized by a deteriorating neurobehavioral/neurecognitive pictur e 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 we re enrolled and after a 6-month growth assessment were randomized into a 60 :40 treatment:control ratio. Treatment consisted of nutropin (Genentech) 1 mg/m(2)/day. A modified Offord Survey Diagnostic Instrument was used to mon itor behavior at 6-month intervals, Family stress was monitored with the Fa mily Inventory of Life Events, and marital functioning was measured with th e 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 symp toms, Within the treatment group, a significant reduction was reported for both depressive and obsessive symptoms, as well as a reduction in skin-pick ing, No behavioral deterioration was reported. Conclusions: We conclude tha t in addition to the previously detailed improvements in physical parameter s for these children, behavioral improvement including a lack of predictabl e behavioral deterioration during the treatment period is a strong argument for the use of GHRT for persons with this difficult syndrome.