The role of rosuvastatin in the treatment of pediatric homozygous familial hypercholesterolemia

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Introduction: Homozygous familial hypercholesterolemia (HoFH) is a rare disorder characterized by severely increased cholesterol levels that lead to an extremely high risk of premature cardiovascular disease (CVD). Aggressive lipid-lowering therapy should be started at an early age with highly potent statins. Rosuvastatin is such a highly efficacious statin with a favorable safety profile that might be advantageous in pediatric HoFH. Areas covered: The authors conducted a PubMed search for rosuvastatin papers and papers on statin use in HoFH published in English. This review describes the pharmacology, safety and efficacy of rosuvastatin and discusses its use in pediatric HoFH. Expert opinion: To date, there is very little evidence on the efficacy and safety of rosuvastatin in pediatric HoFH. Sufficient clinical evidence has proven the lipid-lowering capacity and subsequent CVD prevention of rosuvastatin in hypercholesterolemic adults. Furthermore, clinical studies in children heterozygous familial hypercholesterolemia aged 6 years and older revealed no untoward safety concerns. Rosuvastatin is, therefore, a promising agent in the treatment of pediatric HoFH
Original languageEnglish
Pages (from-to)1325-1330
JournalExpert opinion on orphan drugs
Issue number12
Publication statusPublished - 2014

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