Randomized, double-blind, placebo-controlled pilot trial of megestrol acetate in malnourished children with cystic fibrosis

Background: Undernutrition is common in patients with cystic fibrosis (CF). Nutritional rehabilitation has been shown to improve linear growth, pulmon ary function, well-being, and resistance to infection in this population. T he purpose of this study was to determine whether the administration of meg estrol acetate (MA) induces weight gain in malnourished patients with CE an d to assess the composition of weight gain. Methods: In a randomized, placebo-controlled, double-blind, crossover study , 12 children with CF received MA (10 mg/kg/d) or placebo for 12 weeks, fol lowed by a 12-week washout period, then the alternative treatment. Anthropo metrics, caloric intake, and clinical assessment were obtained every 6 week s; pulmonary function tests, biochemistry, hematology, cortisol, growth hor mone, insulin, C-peptide, insulin-like growth factor-1, insulin-like growth factor binding protein-3, and dual-energy x-ray absorptiometry scans were obtained every 12 weeks. Results: Six children did not complete the study, three for reasons unrelat ed to the study, two because they developed diabetes while receiving MA, an d one who had glucose intolerance while receiving the placebo. Average weig ht gain was 3.05 kg in the MA group and 0.3 kg in the placebo group. The ch ange in weight z score was +0.76 in the MA group and -0.05 in the placebo g roup. The change in height z score was -0.06 in the MA group and t0.06 in t he placebo group. Lean body mass and body fat increased by 1507 g and 1192 g respectively in the MA group. Pulmonary function tests improved in the MA group; serum cortisol levels decreased. Side effects included glucosuria, insomnia, hyperactivity, and irritability. Conclusions: Weight, body fat, and lean body mass increased and pulmonary f unction improved in the children with CF given MA. Adrenal suppression, glu cose intolerance, and diabetes are side effects.