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Review Article

治疗纯合子家族性高胆固醇血症的选择:洛米他派的作用

卷 27, 期 23, 2020

页: [3773 - 3783] 页: 11

弟呕挨: 10.2174/0929867326666190121120735

价格: $65

摘要

背景:洛米他派(美国Juxtapid®和欧洲Lojuxta®)是第一个开发的微粒体甘油三酯转移蛋白(MTP)的抑制剂，被批准为治疗纯合子家族性高胆固醇血症(HoFH)的新药物。它通过直接地或者选择性地与MTP结合，从而减少含脂蛋白的apo-B在肝脏和肠道中的合成与分泌。 目的:本文旨在总结洛米他派在治疗HoFH中的最新研究进展。 结果: 洛米他派的有效性和安全性已经在几个试验中进行了评估，并已显示其降低血浆中低密度脂蛋白胆固醇(LDL-C)水平平均超过50%。最常见的副作用是胃肠道和肝脏事件，但洛米他派通常表现出良好的耐受性和令人满意的患者依从性。最近，在欧洲，为了评估洛米他派长期的安全性和有效性，建立了the LOWER registry (ClinicalTrials.gov Identifier: NCT02135705)，以便从实际生活临床实践中获取用洛米他派 治疗患有HoFH患者的信息。此外，洛米他派降低甘油三酯水平的观察结果表明，可以考虑将洛米他派 用于治疗严重高甘油三酯血症患者——这些患者反复发作胰腺炎且对常规治疗反应不佳。 结论: 洛米他派是一种治疗HoFH的创新和有效的药物。长期安全数据、儿童和孕妇HoFH患者的治疗以及严重高甘油三酯血症的治疗仍需进一步研究。

关键词: 纯合子家族性高胆固醇血症，洛米他派，LOWER Registry，甘油三酯转移蛋白(MTP)抑制剂，MTP SNPs，常染色体显性高胆固醇血症(ADH)

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[1]
Sjouke, B.; Hovingh, G.K.; Kastelein, J.J.P.; Stefanutti, C. Homozygous autosomal dominant hypercholesterolaemia: prevalence, diagnosis, and current and future treatment perspectives. Curr. Opin. Lipidol., 2015, 26(3), 200-209.
[http://dx.doi.org/10.1097/MOL.0000000000000179] [PMID: 25950706]
[2]
Hartgers, M.L.; Ray, K.K.; Hovingh, G.K. New approaches in detection and treatment of familial hypercholesterolemia. Curr. Cardiol. Rep., 2015, 17(12), 109.
[http://dx.doi.org/10.1007/s11886-015-0665-x] [PMID: 26482752]
[3]
Patel, R.S.; Scopelliti, E.M.; Savelloni, J. Therapeutic management of familial hypercholesterolemia: current and emerging drug therapies. Pharmacotherapy, 2015, 35(12), 1189-1203.
[http://dx.doi.org/10.1002/phar.1672] [PMID: 26684558]
[4]
Stefanutti, C.; Morozzi, C.; Di Giacomo, S.; Sovrano, B.; Mesce, D.; Grossi, A. Management of homozygous familial hypercholesterolemia in real-world clinical practice: A report of 7 Italian patients treated in Rome with lomitapide and lipoprotein apheresis. J. Clin. Lipidol., 2016, 10(4), 782-789.
[http://dx.doi.org/10.1016/j.jacl.2016.02.009] [PMID: 27578108]
[5]
Pisciotta, L.; Priore Oliva, C.; Pes, G.M.; Di Scala, L.; Bellocchio, A.; Fresa, R.; Cantafora, A.; Arca, M.; Calandra, S.; Bertolini, S. Autosomal recessive hypercholesterolemia (ARH) and homozygous familial hypercholesterolemia (FH): a phenotypic comparison. Atherosclerosis, 2006, 188(2), 398-405.
[http://dx.doi.org/10.1016/j.atherosclerosis.2005.11.016] [PMID: 16343504]
[6]
Nordestgaard, B.G.; Chapman, M.J.; Humphries, S.E.; Ginsberg, H.N.; Masana, L.; Descamps, O.S.; Wiklund, O.; Hegele, R.A.; Raal, F.J.; Defesche, J.C.; Wiegman, A.; Santos, R.D.; Watts, G.F.; Parhofer, K.G.; Hovingh, G.K.; Kovanen, P.T.; Boileau, C.; Averna, M.; Borén, J.; Bruckert, E.; Catapano, A.L.; Kuivenhoven, J.A.; Pajukanta, P.; Ray, K.; Stalenhoef, A.F.; Stroes, E.; Taskinen, M.R.; Tybjærg-Hansen, A. European Atherosclerosis Society Consensus Panel. Familial hypercholesterolaemia is underdiagnosed and undertreated in the general population: guidance for clinicians to prevent coronary heart disease: consensus statement of the European Atherosclerosis Society. Eur. Heart J., 2013, 34(45), 3478-90a.
[http://dx.doi.org/10.1093/eurheartj/eht273] [PMID: 23956253]
[7]
Cuchel, M.; Bruckert, E.; Ginsberg, H.N.; Raal, F.J.; Santos, R.D.; Hegele, R.A.; Kuivenhoven, J.A.; Nordestgaard, B.G.; Descamps, O.S.; Steinhagen-Thiessen, E.; Tybjærg-Hansen, A.; Watts, G.F.; Averna, M.; Boileau, C.; Borén, J.; Catapano, A.L.; Defesche, J.C.; Hovingh, G.K.; Humphries, S.E.; Kovanen, P.T.; Masana, L.; Pajukanta, P.; Parhofer, K.G.; Ray, K.K.; Stalenhoef, A.F.; Stroes, E.; Taskinen, M.R.; Wiegman, A.; Wiklund, O.; Chapman, M.J. European Atherosclerosis Society Consensus Panel on Familial Hypercholesterolaemia. 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(32), 2146-2157.
[http://dx.doi.org/10.1093/eurheartj/ehu274] [PMID: 25053660]
[8]
Sjouke, B.; Kusters, D.M.; Kindt, I.; Besseling, J.; Defesche, J.C.; Sijbrands, E.J.; Roeters van Lennep, J.E.; Stalenhoef, A.F.; Wiegman, A.; de Graaf, J.; Fouchier, S.W.; Kastelein, J.J.; Hovingh, G.K. Homozygous autosomal dominant hypercholesterolaemia in the Netherlands: prevalence, genotype-phenotype relationship, and clinical outcome. Eur. Heart J., 2015, 36(9), 560-565.
[http://dx.doi.org/10.1093/eurheartj/ehu058] [PMID: 24585268]
[9]
France, M. Homozygous familial hypercholesterolaemia: update on management. Paediatr. Int. Child Health, 2016, 36(4), 243-247.
[http://dx.doi.org/10.1080/20469047.2016.1246640] [PMID: 27967828]
[10]
Bertolini, S.; Pisciotta, L.; Rabacchi, C.; Cefalù, A.B.; Noto, D.; Fasano, T.; Signori, A.; Fresa, R.; Averna, M.; Calandra, S. Spectrum of mutations and phenotypic expression in patients with autosomal dominant hypercholesterolemia identified in Italy. Atherosclerosis, 2013, 227(2), 342-348.
[http://dx.doi.org/10.1016/j.atherosclerosis.2013.01.007] [PMID: 23375686]
[11]
Blom, D.J.; Fayad, Z.A.; Kastelein, J.J.; Larrey, D.; Makris, L.; Schwamlein, C.; Bloeden, L.; Underberg, J. LOWER investigators. LOWER, a registry of lomitapide-treated patients with homozygous familial hypercholesterolemia: Rationale and design. J. Clin. Lipidol., 2016, 10(2), 273-282.
[http://dx.doi.org/10.1016/j.jacl.2015.11.011] [PMID: 27055957]
[12]
Panno, M.D.; Cefalù, A.B.; Averna, M.R. Lomitapide: a novel drug for homozygous familial hypercholesterolemia. Clin. Lipidol., 2014, 9, 19-32.
[http://dx.doi.org/10.2217/clp.13.74]
[13]
Davis, K.A.; Miyares, M.A. Lomitapide: A novel agent for the treatment of homozygous familial hypercholesterolemia. Am. J. Health Syst. Pharm., 2014, 71(12), 1001-1008.
[http://dx.doi.org/10.2146/ajhp130592] [PMID: 24865757]
[14]
Rader, D.J.; Kastelein, J.J. Lomitapide and mipomersen: two first-in-class drugs for reducing low-density lipoprotein cholesterol in patients with homozygous familial hypercholesterolemia. Circulation, 2014, 129(9), 1022-1032.
[http://dx.doi.org/10.1161/circulationaha.113.001292] [PMID: 24589695]
[15]
Averna, M.; Cefalù, A.B.; Stefanutti, C.; Di Giacomo, S.; Sirtori, C.R.; Vigna, G. Individual analysis of patients with HoFH participating in a phase 3 trial with lomitapide: The Italian cohort. Nutr. Metab. Cardiovasc. Dis., 2016, 26(1), 36-44.
[http://dx.doi.org/10.1016/j.numecd.2015.11.001] [PMID: 26723464]
[16]
Farnier, M.; Bruckert, E. Severe familial hypercholesterolaemia: current and future management. Arch. Cardiovasc. Dis., 2012, 105(12), 656-665.
[http://dx.doi.org/10.1016/j.acvd.2012.05.011] [PMID: 23199621]
[17]
Sanchez, A.P.; Cunard, R.; Ward, D.M. The selective therapeutic apheresis procedures. J. Clin. Apher., 2013, 28(1), 20-29.
[http://dx.doi.org/10.1002/jca.21265] [PMID: 23420592]
[18]
Thompson, G.R.; Miller, J.P.; Breslow, J.L. Improved survival of patients with homozygous familial hypercholesterolaemia treated with plasma exchange. Br. Med. J. (Clin. Res. Ed.), 1985, 291(6510), 1671-1673.
[http://dx.doi.org/10.1136/bmj.291.6510.1671] [PMID: 3935235]
[19]
Catapano, A.L.; Graham, I.; De Backer, G.; Wiklund, O.; Chapman, M.J.; Drexel, H.; Hoes, A.W.; Jennings, C.S.; Landmesser, U.; Pedersen, T.R.; Reiner, Ž.; Riccardi, G.; Taskinen, M.R.; Tokgozoglu, L.; Verschuren, W.M.M.; Vlachopoulos, C.; Wood, D.A.; Zamorano, J.L.; Cooney, M.T. ESC Scientific Document Group. 2016 ESC/EAS guidelines for the management of dyslipidaemias. Eur. Heart J., 2016, 37(39), 2999-3058.
[http://dx.doi.org/10.1093/eurheartj/ehw272] [PMID: 27567407]
[20]
Robinson, J.G. Management of familial hypercholesterolemia: a review of the recommendations from the national lipid association expert panel on familial hypercholesterolemia. J. Manag. Care Pharm., 2013, 19(2), 139-149.
[http://dx.doi.org/10.18553/jmcp.2013.19.2.139] [PMID: 23461430]
[21]
Watts, G.F.; Gidding, S.; Wierzbicki, A.S.; Toth, P.P.; Alonso, R.; Brown, W.V.; Bruckert, E.; Defesche, J.; Lin, K.K.; Livingston, M.; Mata, P.; Parhofer, K.G.; Raal, F.J.; Santos, R.D.; Sijbrands, E.J.; Simpson, W.G.; Sullivan, D.R.; Susekov, A.V.; Tomlinson, B.; Wiegman, A.; Yamashita, S.; Kastelein, J.J.; International, F.H. Integrated guidance on the care of familial hypercholesterolemia from the International FH Foundation. J. Clin. Lipidol., 2014, 8(2), 148-172.
[http://dx.doi.org/10.1016/j.jacl.2014.01.002] [PMID: 24636175]
[22]
Maron, D.J.; Fazio, S.; Linton, M.F. Current perspectives on statins. Circulation, 2000, 101(2), 207-213.
[http://dx.doi.org/10.1161/01.CIR.101.2.207] [PMID: 10637210]
[23]
Thompson, G.R.; Barbir, M.; Davies, D.; Dobral, P.; Gesinde, M.; Livingston, M.; Mandry, P.; Marais, A.D.; Matthews, S.; Neuwirth, C.; Pottle, A.; le Roux, C.; Scullard, D.; Tyler, C.; Watkins, S. Efficacy criteria and cholesterol targets for LDL apheresis. Atherosclerosis, 2010, 208(2), 317-321.
[http://dx.doi.org/10.1016/j.atherosclerosis.2009.06.010] [PMID: 19589528]
[24]
Mabuchi, H.; Koizumi, J.; Shimizu, M.; Kajinami, K.; Miyamoto, S.; Ueda, K.; Takegoshi, T. Hokuriku-FH-LDL-Apheresis Study Group. Long-term efficacy of low-density lipoprotein apheresis on coronary heart disease in familial hypercholesterolemia. Am. J. Cardiol., 1998, 82(12), 1489-1495.
[http://dx.doi.org/10.1016/S0002-9149(98)00692-4] [PMID: 9874053]
[25]
Stefanutti, C.; Blom, D.J.; Averna, M.R.; Meagher, E.A.; Theron, Hd.; Marais, A.D.; Hegele, R.A.; Sirtori, C.R.; Shah, P.K.; Gaudet, D.; Vigna, G.B.; Sachais, B.S.; Di Giacomo, S.; du Plessis, A.M.; Bloedon, L.T.; Balser, J.; Rader, D.J.; Cuchel, M. 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, single-arm, open-label trial. Atherosclerosis, 2015, 240(2), 408-414.
[http://dx.doi.org/10.1016/j.atherosclerosis.2015.03.014] [PMID: 25897792]
[26]
Thompson, G.R. Managing homozygous familial hypercholesterolaemia from cradle to grave. Atheroscler. Suppl., 2015, 18, 16-20.
[http://dx.doi.org/10.1016/j.atherosclerosissup.2015.02.002] [PMID: 25936299]
[27]
Schuff-Werner, P.; Fenger, S.; Kohlschein, P. Role of lipid apheresis in changing times. Clin. Res. Cardiol. Suppl., 2012, 7, 7-14.
[http://dx.doi.org/10.1007/s11789-012-0049-3] [PMID: 22911176]
[28]
Thompson, G.R. HEART-UK LDL Apheresis Working Group. Recommendations for the use of LDL apheresis. Atherosclerosis, 2008, 198(2), 247-255.
[http://dx.doi.org/10.1016/j.atherosclerosis.2008.02.009] [PMID: 18371971]
[29]
Seidah, N.G.; Benjannet, S.; Wickham, L.; Marcinkiewicz, J.; Jasmin, S.B.; Stifani, S.; Basak, A.; Prat, A.; Chretien, M. The secretory proprotein convertase neural apoptosis-regulated convertase 1 (NARC-1): liver regeneration and neuronal differentiation. Proc. Natl. Acad. Sci. USA, 2003, 100(3), 928-933.
[http://dx.doi.org/10.1073/pnas.0335507100] [PMID: 12552133]
[30]
Fasano, T.; Cefalù, A.B.; Di Leo, E.; Noto, D.; Pollaccia, D.; Bocchi, L.; Valenti, V.; Bonardi, R.; Guardamagna, O.; Averna, M.; Tarugi, P. A novel loss of function mutation of PCSK9 gene in white subjects with low-plasma low-density lipoprotein cholesterol. Arterioscler. Thromb. Vasc. Biol., 2007, 27(3), 677-681.
[http://dx.doi.org/10.1161/01.ATV.0000255311.26383.2f] [PMID: 17170371]
[31]
Cohen, J.C.; Boerwinkle, E.; Mosley, T.H., Jr; Hobbs, H.H. Sequence variations in PCSK9, low LDL, and protection against coronary heart disease. N. Engl. J. Med., 2006, 354(12), 1264-1272.
[http://dx.doi.org/10.1056/NEJMoa054013] [PMID: 16554528]
[32]
Noto, D.; Giammanco, A.; Barbagallo, C.M.; Cefalù, A.B.; Averna, M.R. Anti-PCSK9 treatment: Is ultra-low LDL always good? Cardiovasc. Res., 2018, 114(12), 1595-1604.
[http://dx.doi.org/10.1093/cvr/cvy144] [PMID: 29931148]
[33]
Abifadel, M.; Varret, M.; Rabès, J.P.; Allard, D.; Ouguerram, K.; Devillers, M.; Cruaud, C.; Benjannet, S.; Wickham, L.; Erlich, D.; Derré, A.; Villéger, L.; Farnier, M.; Beucler, I.; Bruckert, E.; Chambaz, J.; Chanu, B.; Lecerf, J.M.; Luc, G.; Moulin, P.; Weissenbach, J.; Prat, A.; Krempf, M.; Junien, C.; Seidah, N.G.; Boileau, C. Mutations in PCSK9 cause autosomal dominant hypercholesterolemia. Nat. Genet., 2003, 34(2), 154-156.
[http://dx.doi.org/10.1038/ng1161] [PMID: 12730697]
[34]
Robinson, J.G.; Farnier, M.; Krempf, M.; Bergeron, J.; Luc, G.; Averna, M.; Stroes, E.S.; Langslet, G.; Raal, F.J.; El Shahawy, M.; Koren, M.J.; Lepor, N.E.; Lorenzato, C.; Pordy, R.; Chaudhari, U.; Kastelein, J.J. ODYSSEY LONG TERM Investigators. Efficacy and safety of alirocumab in reducing lipids and cardiovascular events. N. Engl. J. Med., 2015, 372(16), 1489-1499.
[http://dx.doi.org/10.1056/NEJMoa1501031] [PMID: 25773378]
[35]
Sabatine, M.S.; Giugliano, R.P.; Wiviott, S.D.; Raal, F.J.; Blom, D.J.; Robinson, J.; Ballantyne, C.M.; Somaratne, R.; Legg, J.; Wasserman, S.M.; Scott, R.; Koren, M.J.; Stein, E.A. Open-Label Study of Long-Term Evaluation against LDL Cholesterol (OSLER) Investigators. Efficacy and safety of evolocumab in reducing lipids and cardiovascular events. N. Engl. J. Med., 2015, 372(16), 1500-1509.
[http://dx.doi.org/10.1056/NEJMoa1500858] [PMID: 25773607]
[36]
Stein, E.A.; Honarpour, N.; Wasserman, S.M.; Xu, F.; Scott, R.; Raal, F.J. Effect of the proprotein convertase subtilisin/kexin 9 monoclonal antibody, AMG 145, in homozygous familial hypercholesterolemia. Circulation, 2013, 128(19), 2113-2120.
[http://dx.doi.org/10.1161/circulationaha.113.004678] [PMID: 24014831]
[37]
Raal, F.J.; Hovingh, G.K.; Blom, D.; Santos, R.D.; Harada-Shiba, M.; Bruckert, E.; Couture, P.; Soran, H.; Watts, G.F.; Kurtz, C.; Honarpour, N.; Tang, L.; Kasichayanula, S.; Wasserman, S.M.; Stein, E.A. Long-term treatment with evolocumab added to conventional drug therapy, with or without apheresis, in patients with homozygous familial hypercholesterolaemia: an interim subset analysis of the open-label TAUSSIG study. Lancet Diabetes Endocrinol., 2017, 5(4), 280-290.
[http://dx.doi.org/10.1016/S2213-8587(17)30044-X] [PMID: 28215937]
[38]
Guerin, M. Reverse cholesterol transport in familial hypercholesterolemia. Curr. Opin. Lipidol., 2012, 23(4), 377-385.
[http://dx.doi.org/10.1097/MOL.0b013e328353ef07] [PMID: 22510809]
[39]
Ballantyne, C.M.; Shah, S.; Kher, U.; Hunter, J.A.; Gill, G.G.; Cressman, M.D.; Ashraf, T.B.; Johnson-Levonas, A.O.; Mitchel, Y.B. Lipid-modifying efficacy and tolerability of anacetrapib added to ongoing statin therapy in patients with hypercholesterolemia or Low high-density lipoprotein cholesterol. Am. J. Cardiol., 2017, 119(3), 388-396.
[http://dx.doi.org/10.1016/j.amjcard.2016.10.032] [PMID: 27956003]
[40]
Gouni-Berthold, I.; Berthold, H.K. Mipomersen and lomitapide: Two new drugs for the treatment of homozygous familial hypercholesterolemia. Atheroscler. Suppl., 2015, 18, 28-34.
[http://dx.doi.org/10.1016/j.atherosclerosissup.2015.02.005] [PMID: 25936301]
[41]
Hussain, M.M.; Rava, P.; Walsh, M.; Rana, M.; Iqbal, J. Multiple functions of microsomal triglyceride transfer protein. Nutr. Metab. (Lond.), 2012, 9, 14.
[http://dx.doi.org/10.1186/1743-7075-9-14] [PMID: 22353470]
[42]
Goldberg, A.C. Emerging low-density lipoprotein therapies: Microsomal triglyceride transfer protein inhibitors. J. Clin. Lipidol., 2013, 7(3)(Suppl.), S16-S20.
[http://dx.doi.org/10.1016/j.jacl.2013.03.003] [PMID: 23642324]
[43]
Gordon, D.A.; Jamil, H. Progress towards understanding the role of microsomal triglyceride transfer protein in apolipoprotein-B lipoprotein assembly. Biochim. Biophys. Acta, 2000, 1486(1), 72-83.
[http://dx.doi.org/10.1016/S1388-1981(00)00049-4] [PMID: 10856714]
[44]
Vuorio, A.; Tikkanen, M.J.; Kovanen, P.T. Inhibition of hepatic microsomal triglyceride transfer protein - a novel therapeutic option for treatment of homozygous familial hypercholesterolemia. Vasc. Health Risk Manag., 2014, 10, 263-270.
[http://dx.doi.org/10.2147/VHRM.S36641] [PMID: 24851052]
[45]
Haghpassand, M.; Wilder, D.; Moberly, J.B. Inhibition of apolipoprotein B and triglyceride secretion in human hepatoma cells (HepG2). J. Lipid Res., 1996, 37(7), 1468-1480.
[PMID: 8827519]
[46]
Cuchel, M.; Meagher, E.A.; du Toit Theron, H.; Blom, D.J.; Marais, A.D.; Hegele, R.A.; Averna, M.R.; Sirtori, C.R.; Shah, P.K.; Gaudet, D.; Stefanutti, C.; Vigna, G.B.; Du Plessis, A.M.; Propert, K.J.; Sasiela, W.J.; Bloedon, L.T.; Rader, D.J. Phase 3 HoFH Lomitapide Study investigators. 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(9860), 40-46.
[http://dx.doi.org/10.1016/S0140-6736(12)61731-0] [PMID: 23122768]
[47]
Blom, D.J.; Averna, M.R.; Meagher, E.A.; du Toit Theron, H.; Sirtori, C.R.; Hegele, R.A.; Shah, P.K.; Gaudet, D.; Stefanutti, C.; Vigna, G.B.; Larrey, D.; Bloedon, L.T.; Foulds, P.; Rader, D.J.; Cuchel, M. Long-term efficacy and safety of the microsomal triglyceride transfer protein inhibitor lomitapide in patients with homozygous familial hypercholesterolemia. Circulation, 2017, 136(3), 332-335.
[http://dx.doi.org/10.1161/circulationaha.117.028208] [PMID: 28716835]
[48]
D’Erasmo, L.; Cefalù, A.B.; Noto, D.; Giammanco, A.; Averna, M.; Pintus, P.; Medde, P.; Vigna, G.B.; Sirtori, C.; Calabresi, L.; Pavanello, C.; Bucci, M.; Sabbà, C.; Suppressa, P.; Natale, F.; Calabrò, P.; Sampietro, T.; Bigazzi, F.; Sbrana, F.; Bonomo, K.; Sileo, F.; Arca, M. Efficacy of lomitapide in the treatment of familial homozygous hypercholesterolemia: results of a real-world clinical experience in Italy. Adv. Ther., 2017, 34(5), 1200-1210.
[http://dx.doi.org/10.1007/s12325-017-0531-x] [PMID: 28432645]
[49]
Harada-Shiba, M.; Ikewaki, K.; Nohara, A.; Otsubo, Y.; Yanagi, K.; Yoshida, M.; Chang, Q.; Foulds, P. Efficacy and safety of lomitapide in japanese patients with homozygous familial hypercholesterolemia. J. Atheroscler. Thromb., 2017, 24(4), 402-411.
[http://dx.doi.org/10.5551/jat.38216] [PMID: 28154305]
[50]
Aegerion Pharmaceuticals Juxtapid Prescribing Information, 2015.Available at:. www.aegerion.com
[51]
Berberich, A.J.; Hegele, R.A. Lomitapide for the treatment of hypercholesterolemia. Expert Opin. Pharmacother., 2017, 18(12), 1261-1268.
[http://dx.doi.org/10.1080/14656566.2017.1340941] [PMID: 28598687]
[52]
Cuchel, M.; Blom, D.J.; Averna, M.R. Clinical experience of lomitapide therapy in patients with homozygous familial hypercholesterolaemia. Atheroscler. Suppl., 2014, 15(2), 33-45.
[http://dx.doi.org/10.1016/j.atherosclerosissup.2014.07.005] [PMID: 25257075]
[53]
Won, J.I.; Zhang, J.; Tecson, K.M.; McCullough, P.A. Balancing low-density lipoprotein cholesterol reduction and hepatotoxicity with lomitapide mesylate and mipomersen in patients with homozygous familial hypercholesterolemia. Rev. Cardiovasc. Med., 2017, 18(1), 21-28.
[PMID: 28509890]
[54]
Sacks, F.M.; Stanesa, M.; Hegele, R.A. Progression to hepatitis and fibrosis secondary to lomitapide use--reply. JAMA Intern. Med., 2014, 174(9), 1522-1523.
[http://dx.doi.org/10.1001/jamainternmed.2014.1528] [PMID: 25178869]
[55]
Brown, W.V.; Bramlet, D.A.; Ross, J.L.; Underberg, J.A. JCL roundtable: Risk evaluation and mitigation strategy. J. Clin. Lipidol., 2016, 10(6), 1288-1296.
[http://dx.doi.org/10.1016/j.jacl.2016.10.007] [PMID: 27919344]
[56]
France, M.; Rees, A.; Datta, D.; Thompson, G.; Capps, N.; Ferns, G.; Ramaswami, U.; Seed, M.; Neely, D.; Cramb, R.; Shoulders, C.; Barbir, M.; Pottle, A.; Eatough, R.; Martin, S.; Bayly, G.; Simpson, B.; Halcox, J.; Edwards, R.; Main, L.; Payne, J.; Soran, H. for HEART UK Medical Scientific and Research Committee. HEART UK statement on the management of homozygous familial hypercholesterolaemia in the United Kingdom. Atherosclerosis, 2016, 255, 128-139.
[http://dx.doi.org/10.1016/j.atherosclerosis.2016.10.017] [PMID: 27839699]
[57]
Roeters van Lennep, J.; Averna, M.; Alonso, R. Treating homozygous familial hypercholesterolemia in a real-world setting: Experiences with lomitapide. J. Clin. Lipidol., 2015, 9(4), 607-617.
[http://dx.doi.org/10.1016/j.jacl.2015.05.001] [PMID: 26228681]
[58]
Leipold, R.; Raal, F.; Ishak, J.; Hovingh, K.; Phillips, H. The effect of lomitapide on cardiovascular outcome measures in homozygous familial hypercholesterolemia: a modeling analysis., Eur. J. Prev. Cardiol,. 2017, 01-8.
[http://dx.doi.org/10.1177/2047487317730473]
[59]
Brahm, A.J.; Hegele, R.A. Lomitapide for the treatment of hypertriglyceridemia. Expert Opin. Investig. Drugs, 2016, 25(12), 1457-1463.
[http://dx.doi.org/10.1080/13543784.2016.1254187] [PMID: 27785928]
[60]
Brahm, A.J.; Hegele, R.A. Lomitapide for the treatment of hypertriglyceridemia. Drug Evaluation, 2016, 25(12), 1457-1463.
[http://dx.doi.org/10.1080/13543784.2016.1254187]
[61]
Kolovou, G.D.; Kolovou, V.; Papadopoulou, A.; Watts, G.F.J. MTP Gene variants and response to lomitapide in patients with homozygous familial hypercholesterolemia. J. Atheroscler. Thromb., 2016, 23(7), 878-883.
[http://dx.doi.org/10.5551/jat.34777] [PMID: 27170061]

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