A pilot study in the efficacy and safety of gemfibrozil in a pediatric population

Gemfibrozil reduces plasma triglycerides and raises high-density lipoprotein cholesterol (HDL-C) in adults and also reduces the incidence of cardiovascular endpoints in adults. Its efficacy in improving lipid abnormalities has not been evaluated in children.

Our purpose was to investigate whether gemfibrozil would lower triglycerides and raise HDL-C with minimal adverse effects in a pediatric population with metabolic syndrome.

We conducted a retrospective study of 47 pediatric patients with metabolic syndrome who started gemfibrozil treatment, 1200 mg/day because of failure or lack of interest in therapeutic lifestyle changes. Eligibility required patients to be younger than 21 years of age, and have pretreatment and on-treatment fasting lipid profiles. Data recorded included age at start of treatment, blood pressure, body mass index, waist circumference, percent body fat, total plasma cholesterol, HDL, low-density lipoprotein, triglycerides, alanine aminotransferase, and aspartate aminotransferase. Treatment duration was elapsed time between starting gemfibrozil and the last clinic visit.

Average age of the study cohort was 14 years and mean duration on medication was 249 days. Those patients who had pretreatment and on-treatment plasma lipid data (33 of 47) were included in an analysis for efficacy. All 47 patients were monitored for safety. Mean triglycerides decreased by 57%: 426 (standard error of mean = 33.6) to 184 mg/dL (standard error of mean = 19) (P < 0.0001). Mean HDL increased by 20%: 35 to 42 mg/dL (P < 0.001). Body composition changes were insignificant, indicating that the lipid changes can be attributed to gemfibrozil. Two of forty-seven patients reported muscle pain from the drug, one of whom was considered to have had a possible adverse effect from the medication as indicated by muscle pain.

Gemfibrozil significantly lowers triglycerides and raises HDL with reasonable safety in a pediatric population with metabolic syndrome.