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Review

Anti-PCSK9 antibodies for the treatment of heterozygous familial hypercholesterolemia: patient selection and perspectives

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Pages 343-351 | Published online: 04 Sep 2017

References

  • HopkinsPNTothPPBallantyneCMRaderDJNational Lipid Association Expert Panel on Familial HypercholesterolemiaFamilial hypercholesterolemias: prevalence, genetics, diagnosis and screening recommendations from the National Lipid Association Expert Panel on Familial HypercholesterolemiaJ Clin Lipidol20115Suppl 3S9S1721600530
  • SharifiMFutemaMNairDHumphriesSEGenetic architecture of familial hypercholesterolaemiaCurr Cardiol Rep20171954428405938
  • NordestgaardBGChapmanMJHumphriesSEEuropean Atherosclerosis Society Consensus PanelFamilial hypercholesterolaemia is underdiagnosed and undertreated in the general population: guidance for clinicians to prevent coronary heart disease: consensus statement of the European Atherosclerosis SocietyEur Heart J201334453478349023956253
  • De BackerGBesselingJChapmanJEUROASPIRE InvestigatorsPrevalence and management of familial hypercholesterolaemia in coronary patients: an analysis of EUROASPIRE IV, a study of the European Society of CardiologyAtherosclerosis2015241116917525997074
  • CuchelMBruckertEGinsbergHNEuropean Atherosclerosis Society Consensus Panel on Familial HypercholesterolaemiaHomozygous 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 SocietyEur Heart J201435322146215725053660
  • DefescheJCLansbergPJUmans-EckenhausenMAKasteleinJJAdvanced method for the identification of patients with inherited hypercholesterolemiaSemin Vasc Med200441596515199434
  • WilliamsRRHuntSCSchumacherMCDiagnosing heterozygous familial hypercholesterolemia using new practical criteria validated by molecular geneticsAm J Cardiol19937221711768328379
  • Risk of fatal coronary heart disease in familial hypercholesterolaemiaScientific Steering Committee on behalf of the Simon Broome Register GroupBMJ199130368078938961933004
  • CatapanoALGrahamIDe BackerGAuthors/Task Force MembersAdditional Contributor2016 ESC/EAS Guidelines for the Management of DyslipidaemiasEur Heart J201637392999305827567407
  • McCrindleBWOseLMaraisADEfficacy and safety of atorvastatin in children and adolescents with familial hypercholesterolemia or severe hyperlipidemia: a multicenter, randomized, placebo-controlled trialJ Pediatr20031431748012915827
  • WiegmanAHuttenBAde GrootEEfficacy and safety of statin therapy in children with familial hypercholesterolemia: a randomized controlled trialJAMA2004292333133715265847
  • VersmissenJOosterveerDMYazdanpanahMEfficacy of statins in familial hypercholesterolaemia: a long term cohort studyBMJ2008337a242319001495
  • RaalFJPappuASIllingworthDRInhibition of cholesterol synthesis by atorvastatin in homozygous familial hypercholesterolaemiaAtherosclerosis2000150242142810856535
  • HagemenasFCPappuASIllingworthDRThe effects of simvastatin on plasma lipoproteins and cholesterol homeostasis in patients with heterozygous familial hypercholesterolaemiaEur J Clin Invest19902021501572112479
  • HovinghGKDavidsonMHKasteleinJJO’ConnorAMDiagnosis and treatment of familial hypercholesterolaemiaEur Heart J2013341396297123416791
  • NorataGDBallantyneCMCatapanoALNew therapeutic principles in dyslipidaemia: focus on LDL and Lp(a) lowering drugsEur Heart J201334241783178923509227
  • PisciottaLFasanoTBellocchioAEffect of ezetimibe coadministered with statins in genotype-confirmed heterozygous FH patientsAtherosclerosis20071942e116e12217140581
  • KasteleinJJAkdimFStroesESSimvastatin with or without ezetimibe in familial hypercholesterolemiaN Engl J Med2008358141431144318376000
  • LagaceTAPCSK9 and LDLR degradation: regulatory mechanisms in circulation and in cellsCurr Opin Lipidol201425538739325110901
  • NorataGDTavoriHPirilloAFazioSCatapanoALBiology of pro-protein convertase subtilisin kexin 9: beyond low-density lipoprotein cholesterol loweringCardiovasc Res2016112142944227496869
  • CohenJCBoerwinkleEMosleyTHJrHobbsHHSequence variations in PCSK9, low LDL, and protection against coronary heart diseaseN Engl J Med2006354121264127216554528
  • CohenJPertsemlidisAKotowskiIKGrahamRGarciaCKHobbsHHLow LDL cholesterol in individuals of African descent resulting from frequent nonsense mutations in PCSK9Nat Genet200537216116515654334
  • KathiresanSA PCSK9 missense variant associated with a reduced risk of early-onset myocardial infarctionN Engl J Med2008358212299230018499582
  • WernerCHoffmannMMWinklerKBohmMLaufsURisk prediction with proprotein convertase subtilisin/kexin type 9 (PCSK9) in patients with stable coronary disease on statin treatmentVascul Pharmacol20146229410224685817
  • JeongHJLeeHSKimKSKimYKYoonDParkSWSterol-dependent regulation of proprotein convertase subtilisin/kexin type 9 expression by sterol-regulatory element binding protein-2J Lipid Res200849239940917921436
  • NorataGDTibollaGCatapanoALPCSK9 inhibition for the treatment of hypercholesterolemia: promises and emerging challengesVascul Pharmacol201462210311124924410
  • CareskeyHEDavisRAAlbornWETrouttJSCaoGKonradRJAtorvastatin increases human serum levels of proprotein convertase subtilisin/kexin type 9J Lipid Res200849239439818033751
  • AwanZSeidahNGMacFadyenJGRosuvastatin, proprotein convertase subtilisin/kexin type 9 concentrations, and LDL cholesterol response: the JUPITER trialClin Chem201258118318922065156
  • WelderGZinehIPacanowskiMATrouttJSCaoGKonradRJHigh-dose atorvastatin causes a rapid sustained increase in human serum PCSK9 and disrupts its correlation with LDL cholesterolJ Lipid Res20105192714272120525997
  • TibollaGNorataGDArtaliRMeneghettiFCatapanoALPro-protein convertase subtilisin/kexin type 9 (PCSK9): from structure-function relation to therapeutic inhibitionNutr Metab Cardiovasc Dis2011211183584321943799
  • RayKKLandmesserULeiterLAInclisiran in patients at high cardiovascular risk with elevated LDL cholesterolN Engl J Med2017376151430144028306389
  • KasteleinJJNissenSERaderDJSafety and efficacy of LY30,15,014, a monoclonal antibody to proprotein convertase subtilisin/kexin type 9 (PCSK9): a randomized, placebo-controlled Phase 2 studyEur Heart J201637171360136926757788
  • RidkerPMTardifJCAmarencoPSPIRE InvestigatorsLipid-reduction variability and antidrug-antibody formation with bococizumabN Engl J Med2017376161517152628304227
  • NichollsSJPuriRAndersonTEffect of evolocumab on progression of coronary disease in statin-treated patients: the GLAGOV randomized clinical trialJAMA2016316222373238427846344
  • SabatineMSGiuglianoRPKeechACFOURIER Steering Committee and InvestigatorsEvolocumab and clinical outcomes in patients with cardiovascular diseaseN Engl J Med2017376181713172228304224
  • RidkerPMRevkinJAmarencoPSPIRE Cardiovascular Outcome InvestigatorsCardiovascular efficacy and safety of bococizumab in high-risk patientsN Engl J Med2017376161527153928304242
  • SteinEAMellisSYancopoulosGDEffect of a monoclonal antibody to PCSK9 on LDL cholesterolN Engl J Med2012366121108111822435370
  • SteinEAGipeDBergeronJEffect of a monoclonal antibody to PCSK9, REGN727/SAR2,36,553, to reduce low-density lipoprotein cholesterol in patients with heterozygous familial hypercholesterolaemia on stable statin dose with or without ezetimibe therapy: a phase 2 randomised controlled trialLancet20123809836293622633824
  • KasteleinJJGinsbergHNLangsletGODYSSEY FH I and FH II: 78 week results with alirocumab treatment in 735 patients with heterozygous familial hypercholesterolaemiaEur Heart J201536432996300326330422
  • TeramotoTKobayashiMTasakiHEfficacy and Safety of Alirocumab in Japanese Patients With Heterozygous Familial Hypercholesterolemia or at High Cardiovascular Risk With Hypercholesterolemia Not Adequately Controlled With Statins- ODYSSEY JAPAN Randomized Controlled TrialCirc J20168091980198727452202
  • GinsbergHNRaderDJRaalFJEfficacy and Safety of Alirocumab in Patients with Heterozygous Familial Hypercholesterolemia and LDL-C of 160 mg/dl or HigherCardiovasc Drugs Ther201630547348327618825
  • MoriartyPMParhoferKGBabirakSPAlirocumab in patients with heterozygous familial hypercholesterolaemia undergoing lipoprotein apheresis: the ODYSSEY ESCAPE trialEur Heart J201637483588359527572070
  • RobinsonJGFarnierMKrempfMODYSSEY LONG TERM InvestigatorsEfficacy and safety of alirocumab in reducing lipids and cardiovascular eventsN Engl J Med2015372161489149925773378
  • KasteleinJJHovinghGKLangsletGEfficacy and safety of the proprotein convertase subtilisin/kexin type 9 monoclonal antibody alirocumab vs placebo in patients with heterozygous familial hypercholesterolemiaJ Clin Lipidol2017111195203.e428391886
  • RaalFScottRSomaratneRLow-density lipoprotein cholesterol-lowering effects of AMG 145, a monoclonal antibody to proprotein convertase subtilisin/kexin type 9 serine protease in patients with heterozygous familial hypercholesterolemia: the Reduction of LDL-C with PCSK9 Inhibition in Heterozygous Familial Hypercholesterolemia Disorder (RUTH-ERFORD) randomized trialCirculation2012126202408241723129602
  • RaalFJSteinEADufourRRUTHERFORD-2 InvestigatorsPCSK9 inhibition with evolocumab (AMG 145) in heterozygous familial hypercholesterolaemia (RUTHERFORD-2): a randomised, double-blind, placebo-controlled trialLancet2015385996533134025282519
  • SteinEAHonarpourNWassermanSMXuFScottRRaalFJEffect of the proprotein convertase subtilisin/kexin 9 monoclonal antibody, AMG 145, in homozygous familial hypercholesterolemiaCirculation2013128192113212024014831
  • RaalFJHonarpourNBlomDJTESLA InvestigatorsInhibition of PCSK9 with evolocumab in homozygous familial hypercholesterolaemia (TESLA Part B): a randomised, double-blind, placebo-controlled trialLancet2015385996534135025282520
  • LambertGChatelaisMPetridesFNormalization of low-density lipoprotein receptor expression in receptor defective homozygous familial hypercholesterolemia by inhibition of PCSK9 with alirocumabJ Am Coll Cardiol201464212299230025456764
  • RaalFJHovinghGKBlomDLong-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 studyLancet Diabetes Endocrinol20175428029028215937
  • CuchelMMeagherEAdu Toit TheronHPhase 3 HoFH Lomitapide Study investigatorsEfficacy and safety of a microsomal triglyceride transfer protein inhibitor in patients with homozygous familial hypercholesterolaemia: a single-arm, open-label, phase 3 studyLancet20133819860404623122768
  • RaalFJSantosRDBlomDJMipomersen, an apolipoprotein B synthesis inhibitor, for lowering of LDL cholesterol concentrations in patients with homozygous familial hypercholesterolaemia: a randomised, double-blind, placebo-controlled trialLancet20103759719998100620227758
  • NordestgaardBGChapmanMJRayKEuropean Atherosclerosis Society Consensus PanelLipoprotein(a) as a cardiovascular risk factor: current statusEur Heart J201031232844285320965889
  • AlonsoRAndresEMataNSAFEHEART InvestigatorsLipoprotein(a) levels in familial hypercholesterolemia: an important predictor of cardiovascular disease independent of the type of LDL receptor mutationJ Am Coll Cardiol201463191982198924632281
  • VongpromekRBosSTen KateGJLipoprotein(a) levels are associated with aortic valve calcification in asymptomatic patients with familial hypercholesterolaemiaJ Intern Med2015278216617325487646
  • RaalFJGiuglianoRPSabatineMSReduction in lipoprotein(a) with PCSK9 monoclonal antibody evolocumab (AMG 145): a pooled analysis of more than 1,300 patients in 4 phase II trialsJ Am Coll Cardiol201463131278128824509273
  • GaudetDKereiakesDJMcKenneyJMEffect of alirocumab, a monoclonal proprotein convertase subtilisin/kexin 9 antibody, on lipoprotein(a) concentrations (a pooled analysis of 150 mg every two weeks dosing from phase 2 trials)Am J Cardiol2014114571171525060413
  • RaalFJGiuglianoRPSabatineMSPCSK9 inhibition-mediated reduction in Lp(a) with evolocumab: an analysis of 10 clinical trials and the LDL receptor’s roleJ Lipid Res20165761086109627102113
  • RobinsonJGRosensonRSFarnierMSafety of Very Low Low-Density Lipoprotein Cholesterol Levels With Alirocumab: Pooled Data From Randomized TrialsJ Am Coll Cardiol201769547148228153102
  • KaziDSMoranAECoxsonPGCost-effectiveness of PCSK9 inhibitor therapy in patients with heterozygous familial hypercholesterolemia or atherosclerotic cardiovascular diseaseJAMA2016316774375327533159