Efficacy and safety of pitavastatin in Japanese male children with familial hypercholesterolemia

Aim: the purpose of this study was to evaluate the efficacy and safety of livalo® tablets (pitavastatin) in japanese male children with heterozygous familial hypercholesterolemia (fh). methods: a multicenter,randomized,double-blind,parallel study was conducted in 14 male children 10-15 years of age with heterozygous fh. pitavastatin (1 mg/day or 2 mg/day) was administered orally for 52 weeks. the primary endpoint was the percent change in the ldl-cholesterol (ldl-c) concentrations from baseline to endpoint (repeated measures ancova at weeks 8 and 12). secondary endpoints included the percentage of patients who achieved the target ldl-c concentration and percent changes in the levels of lipoprotein and lipid parameters at the visit performed at 52 weeks. results: the percent change in ldl-c from baseline (mean 258 mg/dl for all patients) to the endpoint was -27.3% (95%ci; -34.0,-20.5) and -34.3% (95%ci; -41.0,-27.5) in the patients receiving 1 mg and 2 mg of pitavastatin,respectively. stable reductions in the total cholesterol (tc),non-hdl cholesterol (non-hdl-c),apolipoprotein b (apo-b) and ldl-c levels and non-hdl-c/ hdl-c and apo-b/apo-a1 ratios were observed up to 52 weeks in both groups. one patient in each dose group (14%) reached the treatment target level of 130 mg/dl. adverse events were observed in seven (100%) patients receiving 1 mg and five (71%) patients receiving 2 mg of pitavastatin,although none were considered related to the study treatment. one patient in the 1 mg group reported a musculoskeletal ae; however,it was attributed to recent excessive exercise. conclusions: pitavastatin significantly reduced the ldl-c levels and was well tolerated when administered at usual adult doses in 14 male children 10-15 years of age with heterozygous fh. pitavastatin is a promising therapeutic agent for pediatric dyslipidemia with few safety concerns. © 2016,japan atherosclerosis society. all rights reserved.