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

ABSTRACT

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.