{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,23]],"date-time":"2026-08-23T17:47:22Z","timestamp":1787507242102,"version":"build-2736575974"},"reference-count":29,"publisher":"Japan Atherosclerosis Society","issue":"4","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["J Atheroscler Thromb","JAT"],"published-print":{"date-parts":[[2017]]},"DOI":"10.5551\/jat.38216","type":"journal-article","created":{"date-parts":[[2017,2,1]],"date-time":"2017-02-01T17:05:06Z","timestamp":1485968706000},"page":"402-411","source":"Crossref","is-referenced-by-count":54,"title":["Efficacy and Safety of Lomitapide in Japanese Patients with Homozygous Familial Hypercholesterolemia"],"prefix":"10.5551","volume":"24","author":[{"given":"Mariko","family":"Harada-Shiba","sequence":"first","affiliation":[{"name":"National Cerebral and Cardiovascular Research Center"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Katsunori","family":"Ikewaki","sequence":"additional","affiliation":[{"name":"National Defense Medical College"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Atsushi","family":"Nohara","sequence":"additional","affiliation":[{"name":"Kanazawa University Graduate School of Medical Sciences"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yoshihiko","family":"Otsubo","sequence":"additional","affiliation":[{"name":"Shin-Koga Clinic, Medical Group Tenjin-kai"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Koji","family":"Yanagi","sequence":"additional","affiliation":[{"name":"Kenporen Osaka Central Hospital"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Masayuki","family":"Yoshida","sequence":"additional","affiliation":[{"name":"Tokyo Medical and Dental University"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Qing","family":"Chang","sequence":"additional","affiliation":[{"name":"Aegerion Pharmaceuticals, Inc."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pamela","family":"Foulds","sequence":"additional","affiliation":[{"name":"Aegerion Pharmaceuticals, Inc."}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"3489","reference":[{"key":"1","doi-asserted-by":"crossref","unstructured":"1) Cuchel M, Bruckert E, Ginsberg HN, Raal FJ, Santos RD, Hegele RA, Kuivenhoven JA, Nordestgaard BG, Descamps OS, Steinhagen-Thiessen E, Tybjaerg-Hansen A, Watts GF, Averna M, Boileau C, Boren J, Catapano AL, Defesche JC, Hovingh GK, Humphries SE, Kovanen PT, Masana L, Pajukanta P, Parhofer KG, Ray KK, Stalenhoef AF, Stroes E, Taskinen MR, Wiegman A, Wiklund O, Chapman MJ: Homozygous familial hypercholesterolaemia: new insights and guidance for clinicians to improve detection and clinical management. A position paper from the Consensus Panel on Familial Hypercholesterolaemia of the European Atherosclerosis Society. Eur Heart J, 2014; 35: 2146-2157","DOI":"10.1093\/eurheartj\/ehu274"},{"key":"2","doi-asserted-by":"crossref","unstructured":"2) Beigel R, Beigel Y: Homozygous familial hypercholesterolemia: long term clinical course and plasma exchange therapy for two individual patients and review of the literature. J Clin Apher, 2009; 24: 219-224","DOI":"10.1002\/jca.20215"},{"key":"3","doi-asserted-by":"crossref","unstructured":"3) Hobbs HH, Brown MS, Goldstein JL: Molecular genetics of the LDL receptor gene in familial hypercholesterolemia. Hum Mutat, 1992; 1: 445-466","DOI":"10.1002\/humu.1380010602"},{"key":"4","doi-asserted-by":"crossref","unstructured":"4) Kolansky DM, Cuchel M, Clark BJ, Paridon S, McCrindle BW, Wiegers SE, Araujo L, Vohra Y, Defesche JC, Wilson JM, Rader DJ: Longitudinal evaluation and assessment of cardiovascular disease in patients with homozygous familial hypercholesterolemia. Am J Cardiol, 2008; 102: 1438-1443","DOI":"10.1016\/j.amjcard.2008.07.035"},{"key":"5","doi-asserted-by":"crossref","unstructured":"5) Gidding SS, Ann Champagne M, de Ferranti SD, Defesche J, Ito MK, Knowles JW, McCrindle B, Raal F, Rader D, Santos RD, Lopes-Virella M, Watts GF, Wierzbicki AS, and on behalf of the American Heart Association Atherosclerosis, Hypertension, and Obesity in the Young Committee of the Council on Cardiovascular Disease in the Young, Council on Cardiovascular and Stroke Nursing, Council on Functional Genomics and Translational Biology, and Council on Lifestyle and Cardiometabolic Health: The agenda for familial hypercholesterolemia: a scientific statement from the American Heart Association. Circulation, 2015; 132: 2167-2192","DOI":"10.1161\/CIR.0000000000000297"},{"key":"6","doi-asserted-by":"crossref","unstructured":"6) Teramoto T, Sasaki J, Ishibashi S, Birou S, Daida H, Dohi S, Egusa G, Hiro T, Hirobe K, Iida M, Kihara S, Kinoshita M, Maruyama C, Ohta T, Okamura T, Yamashita S, Yokode M, Yokote K, Harada-Shiba M, Arai H, Bujo H, Nohara A, Ohta T, Oikawa S, Okada T, Wakatsuki A: Familial hypercholesterolemia. J Atheroscler Thromb, 2014; 21: 6-10","DOI":"10.5551\/jat.20040"},{"key":"7","doi-asserted-by":"crossref","unstructured":"7) Harada-Shiba M, Arai H, Oikawa S, Ohta T, Okada T, Okamura T, Nohara A, Bujo H, Yokote K, Wakatsuki A, Ishibashi S, Yamashita S: Guidelines for the management of familial hypercholesterolemia. J Atheroscler Thromb, 2012; 19: 1043-1060","DOI":"10.5551\/jat.14621"},{"key":"8","doi-asserted-by":"crossref","unstructured":"8) Zhou M, Zhao D: Familial hypercholesterolemia in Asian populations. Journal of atherosclerosis and thrombosis, 2016; 23: 539-549","DOI":"10.5551\/jat.34405"},{"key":"9","doi-asserted-by":"crossref","unstructured":"9) Watts GF, Ding PY, George P, Hagger MS, Hu M, Lin J, Khoo KL, Marais AD, Miida T, Nawawi HM, Pang J, Park JE, Gonzalez-Santos LB, Su TC, Truong TH, Santos RD, Soran H, Yamashita S, Tomlinson B: Translational research for improving the care of familial hypercholesterolemia: the \u201cten countries study\u201d and beyond. J Atheroscler Thromb, 2016; 23: 891-900","DOI":"10.5551\/jat.35949"},{"key":"10","doi-asserted-by":"crossref","unstructured":"10) Pang J, Lansberg PJ, Watts GF: International developments in the care of familial hypercholesterolemia: where now and where to next. J Atheroscler Thromb, 2016; 23: 505-519","DOI":"10.5551\/jat.34108"},{"key":"11","doi-asserted-by":"crossref","unstructured":"11) Hegele RA: Monogenic dyslipidemias: window on determinants of plasma lipoprotein metabolism. Am J Hum Genet, 2001; 69: 1161-1177","DOI":"10.1086\/324647"},{"key":"12","unstructured":"12) Marais AD: Familial hypercholesterolaemia. Clin Biochem Rev, 2004; 25: 49-68"},{"key":"13","doi-asserted-by":"crossref","unstructured":"13) Harada-Shiba M, Takagi A, Miyamoto Y, Tsushima M, Ikeda Y, Yokoyama S, Yamamoto A: Clinical features and genetic analysis of autosomal recessive hypercholesterolemia. J Clin Endocrinol Metab, 2003; 88: 2541-2547","DOI":"10.1210\/jc.2002-021487"},{"key":"14","doi-asserted-by":"crossref","unstructured":"14) Gagne C, Gaudet D, Bruckert E, Ezetimibe Study Group: Efficacy and safety of ezetimibe coadministered with atorvastatin or simvastatin in patients with homozygous familial hypercholesterolemia. Circulation, 2002; 105: 2469-2475","DOI":"10.1161\/01.CIR.0000018744.58460.62"},{"key":"15","doi-asserted-by":"crossref","unstructured":"15) Yamamoto A, Harada-Shiba M, Endo M, Kusakabe N, Tanioka T, Kato H, Shoji T: The effect of ezetimibe on serum lipids and lipoproteins in patients with homozygous familial hypercholesterolemia undergoing LDL-apheresis therapy. Atherosclerosis, 2006; 186: 126-131","DOI":"10.1016\/j.atherosclerosis.2005.06.039"},{"key":"16","doi-asserted-by":"crossref","unstructured":"16) Raal FJ, Honarpour N, Blom DJ, Hovingh GK, Xu F, Scott R, Wasserman SM, Stein EA and TESLA Investigators: Inhibition of PCSK9 with evolocumab in homozygous familial hypercholesterolaemia (TESLA Part B): a randomised, double-blind, placebo-controlled trial. Lancet, 2015; 385: 341-350","DOI":"10.1016\/S0140-6736(14)61374-X"},{"key":"17","doi-asserted-by":"crossref","unstructured":"17) Thompson GR: LDL apheresis. Atherosclerosis, 2003; 167: 1-13","DOI":"10.1016\/S0021-9150(02)00251-4"},{"key":"18","doi-asserted-by":"crossref","unstructured":"18) Vella A, Pineda AA, O\u2019Brien T: Low-density lipoprotein apheresis for the treatment of refractory hyperlipidemia. Mayo Clin Proc, 2001; 76: 1039-1046","DOI":"10.4065\/76.10.1039"},{"key":"19","doi-asserted-by":"crossref","unstructured":"19) Makino H, Harada-Shiba M: Long-term effect of low-density lipoprotein apheresis in patients with homozygous familial hypercholesterolemia. Ther Apher Dial, 2003; 7: 397-401","DOI":"10.1046\/j.1526-0968.2003.00073.x"},{"key":"20","doi-asserted-by":"crossref","unstructured":"20) Wetterau JR, Lin MC, Jamil H: Microsomal triglyceride transfer protein. Biochim Biophys Acta, 1997; 1345: 136-150","DOI":"10.1016\/S0005-2760(96)00168-3"},{"key":"21","doi-asserted-by":"crossref","unstructured":"21) Cuchel M, Meagher EA, du Toit Theron H, Blom DJ, Marais AD, Hegele RA, Averna MR, Sirtori CR, Shah PK, Gaudet D, Stefanutti C, Vigna GB, Du Plessis AM, Propert KJ, Sasiela WJ, Bloedon LT, Rader DJ: Efficacy and safety of a microsomal triglyceride transfer protein inhibitor in patients with homozygous familial hypercholesterolaemia: a single-arm, open-label, phase 3 study. Lancet, 2013; 381: 40-46","DOI":"10.1016\/S0140-6736(12)61731-0"},{"key":"22","unstructured":"22) LOJUXTA: (lomitapide) summary of product characteristics. Aegerion Pharmaceuticals Ltd, 2015; April"},{"key":"23","unstructured":"23) JUXTAPID: (lomitapide) prescribing information. Aegerion Pharmaceuticals, Cambridge, MA, 2014; August"},{"key":"24","doi-asserted-by":"crossref","unstructured":"24) Taubel J, Sumeray M, Lorch U, McLean A: Pharmacokinetics and pharmacodynamics of lomitapide in Japanese subjects. J Atheroscler Thromb, 2016; 23: 606-620","DOI":"10.5551\/jat.30452"},{"key":"25","doi-asserted-by":"crossref","unstructured":"25) Raal FJ, Pilcher GJ, Panz VR, van Deventer HE, Brice BC, Blom DJ, Marais AD: Reduction in mortality in subjects with homozygous familial hypercholesterolemia associated with advances in lipid-lowering therapy. Circulation, 2011; 124: 2202-2207","DOI":"10.1161\/CIRCULATIONAHA.111.042523"},{"key":"26","doi-asserted-by":"crossref","unstructured":"26) Stefanutti C, Blom DJ, Averna MR, Meagher EA, Theron H, Marais AD, Hegele RA, Sirtori CR, Shah PK, Gaudet D, Vigna GB, Sachais BS, Di Giacomo S, du Plessis AM, Bloedon LT, Balser J, Rader DJ and Cuchel M, for Phase 3 HoFH Lomitapide Study Investigators: The lipid-lowering effects of lomitapide are unaffected by adjunctive apheresis in patients with homozygous familial hypercholesterolaemia - a post-hoc analysis of a Phase 3, singlearm, open-label trial. Atherosclerosis, 2015; 240: 408-414","DOI":"10.1016\/j.atherosclerosis.2015.03.014"},{"key":"27","doi-asserted-by":"crossref","unstructured":"27) Cuchel M, Bloedon LT, Szapary PO, Kolansky DM, Wolfe ML, Sarkis A, Millar JS, Ikewaki K, Siegelman ES, Gregg RE, Rader DJ: Inhibition of microsomal triglyceride transfer protein in familial hypercholesterolemia. The N Engl J Med, 2007; 356: 148-156","DOI":"10.1056\/NEJMoa061189"},{"key":"28","doi-asserted-by":"crossref","unstructured":"28) Blom DJ, Kastelein JJ, Larrey D, Makris L, Phillips H, Bloeden L, Underberg J: Lomitapide Observational Worldwide Evaluation Registry (LOWER): one-year data. American Heart Association Scientific Sessions, 2015; Abstract 2075","DOI":"10.1161\/circ.132.suppl_3.10818"},{"key":"29","doi-asserted-by":"crossref","unstructured":"29) Kolovou GD, Kolovou V, Papadopoulou A, Watts GF: MTP gene variants and response to lomitapide in patients with homozygous familial hypercholesterolemia. J Atheroscler Thromb, 2016; 23: 878-883","DOI":"10.5551\/jat.34777"}],"container-title":["Journal of Atherosclerosis and Thrombosis"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.jstage.jst.go.jp\/article\/jat\/24\/4\/24_38216\/_pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2024,6,22]],"date-time":"2024-06-22T02:06:14Z","timestamp":1719021974000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.jstage.jst.go.jp\/article\/jat\/24\/4\/24_38216\/_article"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2017]]},"references-count":29,"journal-issue":{"issue":"4","published-print":{"date-parts":[[2017]]}},"URL":"https:\/\/doi.org\/10.5551\/jat.38216","relation":{},"ISSN":["1340-3478","1880-3873"],"issn-type":[{"value":"1340-3478","type":"print"},{"value":"1880-3873","type":"electronic"}],"subject":[],"published":{"date-parts":[[2017]]}}}