THERAPY WITH CHOLESTYRAMINE IN CHILDREN : EVALUATION OF SAFETY, TOLERABILITY AND EFFICACY

Aim: Cholestyramine, a bile acid sequestrant, reduces serum LDL-cholesterol (C-LDL) but apparently is poorly tolerated for limited palatability. We aimed to evaluate its safety, tolerability and efficacy in children. Methods: In this retrospective study we reviewed the medical records of all hypercholesterolemic children treated with cholestyramine, followed at our Lipid Center from 1996 to 2009. We selected clinical and laboratory data about compliance, side effects and efficacy. Results: Cholestyramine was prescribed to 66 children (out of a total 480), 4 g every other day for one month and then 4 g/day. Lipid profile and coagulation tests were performed 3 months from the beginning of treatment and every 3-6 months afterwards. Drug was adjusted according to C-LDL levels.12/66 patients (18.1%) never started therapy, 11/66 (16.6%) were on therapy for 3-6 months, 43/66 (65.5%) for > 6 months. Low compliant children were more likely to be obese (p < 0.05). Mean reduction of C-LDL was 23.3% after 3 months of treatment (p < 0.01) and a further 5% after 12 months of treatment. Total and non-HDL cholesterol were also significantly lowered. No gastrointestinal adverse effects were reported. 8,6% of children showed triglyceride values greater than 90th percentile. 18.6% had abnormal coagulation tests (PT, aPTT) in the absence of symptoms. Conclusions: In this cohort of patients cholestyramine exhibited optimal safety and tolerability with a strong lipid-lowering effect. Thus we suggest that cholestyramine should not be overlooked as a therapeutic choice for high-LDL dyslipidemias of childhood.