A 10-year experience using combined lipid-lowering pharmacotherapy in children and adolescents

Background: Current pediatric guidelines for heterozygous familial hypercholesterolemia (HeFH) propose pharmacotherapy (PT) with statins from age 8 to 10 years; however, schemes with absorption inhibitors combined with statins, could be started earlier. The aim of the study was to show the 10-year results of a combined treatment protocol. Methods: Prospective, descriptive and analytical study. Pediatric patients (n = 70; mean age at PT initiation 9.3 years [range, 2-17.5]) with HeFH who required PT between 2005 and 2015 were included. All patients = 10 years, with LDL > 190 mg/dL or > 160 mg/dL with one cardiovascular risk factor (CVRF) or > 130 mg/dL with two or more CVRF; and those patients 5-10 years and with LDL-C > 240 mg/dL or a family history of a cardiovascular event before 40 years, were medicated. After a period on a lipid-lowering diet (LLD), all patients were started on ezetimibe. Patients who did not achieve the treatment goal were given statins. The variables were: age, age at PT initiation, duration of PT, initial LDL-C, mean LDL-C during ezetimibe monodrug therapy, mean LDL-C during combined PT, and percentage of LDL decrease. Results: LDL-C levels were: Baseline: 235 mg/dL +/- 55; after 3 months on ezetimibe: 167 mg/dL +/- 47 (decrease: -27.62%). In 18 patients who did not reach the treatment goal atorvastatin was added and their LDL-C decreased -41.5% (p: 0.02). Overall, mean final LDL-C was 155 mg/dL +/- 30.4 (range, 98-257) and treatment goals were reached in 74% of the patients. No severe side effects were reported. Conclusions: Combined and sequential treatment starting at early ages was shown to be safe and effective over this follow-up period.