title: Efficacy and Safety of Pitavastatin in Japanese Male Children with Familial Hypercholesterolemia.
abstract: OBJECTIVE
  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.
authors.affiliation: Department of Molecular Innovation in Lipidology, National Cerebral and Cardiovascular Center Research Institute.
authors.forename: Mariko
authors.initials: M
authors.lastname: Harada-Shiba
authors.forename: Osamu
authors.initials: O
authors.lastname: Arisaka
authors.forename: Akira
authors.initials: A
authors.lastname: Ohtake
authors.forename: Tomoo
authors.initials: T
authors.lastname: Okada
authors.forename: Hideki
authors.initials: H
authors.lastname: Suganami
mesh_terms: D000284:Administration, Oral; D000293:Adolescent; D000704:Analysis of Variance; D002648:Child; D008078:Cholesterol, LDL / Q000097:blood; D004311:Double-Blind Method; D050171:Dyslipidemias / Q000188:drug therapy; D006579:Heterozygote; D006801:Humans; D019161:Hydroxymethylglutaryl-CoA Reductase Inhibitors / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use; D006938:Hyperlipoproteinemia Type II / Q000188:drug therapy*; D007564:Japan; D008297:Male; D061214:Patient Safety; D011804:Quinolines / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*
chemical_list: D008078:Cholesterol, LDL; D019161:Hydroxymethylglutaryl-CoA Reductase Inhibitors; D011804:Quinolines; C108475:pitavastatin