engleski

Effect of growth hormone therapy in children with Prader-Willi syndrome - our first experiences

Prader-Willi syndrome (PWS) is the most common cause of morbid obesity in childhood. It is the consequence of the lack of expression of genes on the paternally inherited 15q11.2-q13 region. Hyperphagia, obesity, short stature, psychomotor retardation and deterioration of behavior predominate in clinical presentation. Recombinant human growth hormone (rhGH) therapy, along with restriction of caloric intake, has become the mainstay in the management of PWS patients. Anthropometric parameters (height, body mass index (BMI)), therapy eff ect on carbohydrate and lipid metabolism, and occurrence of side eff ects were monitored in four children with PWS treated with rhGH for more than 2 years at doses of up to 1 mg/m2/day. During the follow-up, the height standard deviation score (SDS) increased in comparison with baseline values, and after more than 2 years of treatment with rhGH it was within the reference range for the general children population. BMI SDS decreasedafter the first year of treatment, but thereafter increased again ; still, the level of BMI SDS was much better in comparison with most children with PWS of the same age and gender. RhGH therapy had no negative eff ect on glucose and lipid metabolism, nor caused any other adverse eff ect. Therapy including a customized diet for PWS, along with rhGH therapy, provided a satisfactory growth rate and prevented development of morbid obesity without side effects. This treatment approach would ensure transition of a greater number of PWS patients into adult care, where the multidisciplinary approach in care should be continued.

Prader-Willi syndrome ; Obesity, morbid ; Human growth hormone – therapeutic use ; Paternal

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