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Randomized Controlled Trial
. 2018 May 1;25(5):422-429.
doi: 10.5551/jat.42242. Epub 2017 Nov 29.

Efficacy and Safety of Pitavastatin in Children and Adolescents with Familial Hypercholesterolemia in Japan and Europe

Mariko Harada-Shiba  1 , John J P Kastelein  2 , G Kees Hovingh  2 , Kausik K Ray  3 , Akira Ohtake  4 , Osamu Arisaka  5 , Takao Ohta  6 , Tomoo Okada  7 , Hideki Suganami  8 , Albert Wiegman  9
Affiliations
Randomized Controlled Trial

Efficacy and Safety of Pitavastatin in Children and Adolescents with Familial Hypercholesterolemia in Japan and Europe

Mariko Harada-Shiba et al. J Atheroscler Thromb. .

Abstract

Aim: Children with Familial Hypercholesterolemia (FH) are widely prescribed statins, and it has been suggested that the effects of statins differ among ethnicities. We compared the efficacy and safety of pitavastatin in children and adolescents with FH in clinical trials conducted in Japan and Europe.

Methods: Low-density lipoprotein cholesterol (LDL-C) reductions, adjusted for confounding factors, and safety were compared between the studies in Japan and Europe. In the Japanese study, 14 males with heterozygous FH, aged 11.8±1.6 years, were randomized to 52-week double-blind treatment with 1 or 2 mg/day pitavastatin. In the European study, 106 children and adolescents with high risk hyperlipidemia (103 heterozygous FH), aged 10.6±2.9 years, were randomized to 12-week double-blind treatment with 1, 2 or 4 mg/day pitavastatin or placebo; 84 of these patients and 29 new patients participated in a 52-week open-label extension study.

Results: Age, body weight and baseline LDL-C were identified as factors influencing LDL-C reduction. There were no significant differences in the adjusted mean percentage reduction in LDL-C in Japanese and European children by pitavastatin (24.5% and 23.6%, respectively at 1 mg/day and 33.5% and 30.8%, respectively at 2 mg/day). Pitavastatin was well tolerated without any difference in the frequency or nature of adverse events between the treatment groups, or between the studies.

Conclusion: There were no significant differences between the efficacy or safety of pitavastatin in Japanese and European children and adolescents with FH, suggesting no relevant ethnic differences in the safety or efficacy of pitavastatin.

Keywords: Children; Ethnic difference; Familial hypercholesterolemia; Low-density lipoprotein cholesterol; Pitavastatin.

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Conflict of interest statement

M.H.-S. reports personal fees from Kowa, during the conduct of the study, grants from Astellas, Kaneka Medics, MSD, Takeda, personal fees from Astellas Amgen, Astellas, Sanofi, Astrazeneca, JCR Pharm, Daiichi-Sankyo, MSD, Phizer, Torii, Shionogi, outside the submitted work. J.J.P.K. reports personal fees from Kowa, during the conduct of the study, personal fees from Affiris, Akarna, Amgen, CSL, Behring, Staten Biotech, Regeneron, Madrigal, The Medicines Company, Esperion, Gemphire, Akcea, Ionis, outside the submitted work. G.K.H. reports grants from Netherlands Organisation for Scientific Research, grants from Cardiovascular Research Initiative, grants from European Union, personal fees and non-financial support from AMGEN, personal fees and non-financial support from Aegerion, personal fees from Pfizer, personal fees and non-financial support from AstraZeneca, personal fees and non-financial support from Sanofi, personal fees from Regeneron, personal fees from Kowa, personal fees from IONIS, personal fees from Cerenis, non-financial support from Synageva, outside the submitted work. K.K.R. reports grants from Sanofi, Regeneron, Pfizer, Amgen, MSD, personal fees from Sanofi, Amgen, Regeneron, Lilly, Medicines Company, AstraZeneca, Pfizer, Kowa, Algorithm, IONIS, Esperion, Novo Nordisk, Takeda, Boehringer Ingelheim, Resverlogix, Abbvie, outside the submitted work. A.O. has nothing to disclose. O.A. has nothing to disclose. T.O. has nothing to disclose. T.O. has nothing to disclose. H.S. is an employee of Kowa. A.W. has nothing to disclose.

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