2019 ESC/EAS Guidelines for the Management of Dyslipidaemias

Total Page:16

File Type:pdf, Size:1020Kb

2019 ESC/EAS Guidelines for the Management of Dyslipidaemias ESC/EAS GUIDELINES European Heart Journal (2019) 00,1À78 doi:10.1093/eurheartj/ehz455 2019 ESC/EAS Guidelines for the management Downloaded from https://academic.oup.com/eurheartj/advance-article-abstract/doi/10.1093/eurheartj/ehz455/5556353 by guest on 23 September 2019 of dyslipidaemias: lipid modification to reduce cardiovascular risk The Task Force for the management of dyslipidaemias of the European Society of Cardiology (ESC) and European Atherosclerosis Society (EAS) Authors/Task Force Members: Franc¸ois Mach* (Chairperson) (Switzerland), Colin Baigent* (Chairperson) (United Kingdom), Alberico L. Catapano1* (Chairperson) (Italy), Konstantinos C. Koskinas (Switzerland), Manuela Casula1 (Italy), Lina Badimon (Spain), M. John Chapman1 (France), Guy G. De Backer (Belgium), Victoria Delgado (Netherlands), Brian A. Ference (United Kingdom), Ian M. Graham (Ireland), Alison Halliday (United Kingdom), Ulf Landmesser (Germany), Borislava Mihaylova (United Kingdom), Terje R. Pedersen (Norway), Gabriele Riccardi1 (Italy), Dimitrios J. Richter (Greece), Marc S. Sabatine (United States of America), Marja-Riitta Taskinen1 (Finland), Lale Tokgozoglu1 (Turkey), Olov Wiklund1 (Sweden) The three chairpersons contributed equally to the document. *Corresponding authors: Franc¸ois Mach, Cardiology Department, Geneva University Hospital, 4 Gabrielle-Perret-Gentil, 1211 Geneva, Switzerland. Tel: þ41 223 727 192, Fax: þ41 223 727 229, Email: [email protected]. Colin Baigent, Nuffield Department of Population Health, University of Oxford, Richard Doll Building, Roosevelt Drive, Oxford OX3 7LF, United Kingdom. Tel: þ44 1865 743 741, Fax: þ44 1865 743 985, Email: [email protected]. Alberico L. Catapano, Department of Pharmacological and Biomolecular Sciences, University of Milan, Via Balzaretti, 9, 20133 Milan, and Multimedica IRCCS, Milan, Italy. Tel: þ39 02 5031 8401, Fax: þ39 02 5031 8386, Email: [email protected]. ESC Committee for Practice Guidelines (CPG), National Cardiac Societies document reviewers and Author/Task Force Member affiliations: listed in the Appendix. 1Representing the EAS. ESC entities having participated in the development of this document: Associations: Acute Cardiovascular Care Association (ACCA), Association of Cardiovascular Nursing & Allied Professions (ACNAP), European Association of Cardiovascular Imaging (EACVI), European Association of Preventive Cardiology (EAPC), European Association of Percutaneous Cardiovascular Interventions (EAPCI). Councils: Council for Cardiology Practice, Council on Hypertension, Council on Stroke. Working Groups: Aorta and Peripheral Vascular Diseases, Atherosclerosis and Vascular Biology, Cardiovascular Pharmacotherapy, e-Cardiology, Thrombosis. The content of these European Society of Cardiology (ESC) Guidelines has been published for personal and educational use only. No commercial use is authorized. No part of the ESC Guidelines may be translated or reproduced in any form without written permission from the ESC. Permission can be obtained upon submission of a written request to Oxford University Press, the publisher of the European Heart Journal and the party authorized to handle such permissions on behalf of the ESC (journals.permissions@oxfordjour- nals.org). Disclaimer. The ESC/EAS Guidelines represent the views of the ESC and EAS, and were produced after careful consideration of the scientific and medical knowledge, and the evidence available at the time of their publication. The ESC and EAS is not responsible in the event of any contradiction, discrepancy, and/or ambiguity between the ESC/EAS Guidelines and any other official recommendations or guidelines issued by the relevant public health authorities, in particular in relation to good use of healthcare or therapeutic strategies. Health professionals are encouraged to take the ESC/EAS Guidelines fully into account when exercising their clinical judgment, as well as in the determination and the implementation of preventive, diagnostic, or therapeutic medical strategies; however, the ESC/EAS Guidelines do not override, in any way whatsoever, the individual responsibil- ity of health professionals to make appropriate and accurate decisions in consideration of each patient’s health condition and in consultation with that patient and, where appro- priate and/or necessary, the patient’s caregiver. Nor do the ESC/EAS Guidelines exempt health professionals from taking into full and careful consideration the relevant official updated recommendations or guidelines issued by the competent public health authorities, in order to manage each patient’s case in light of the scientifically accepted data pur- suant to their respective ethical and professional obligations. It is also the health professional’s responsibility to verify the applicable rules and regulations relating to drugs and medical devices at the time of prescription. VC The European Society of Cardiology and the European Atherosclerosis Association 2019. All rights reserved. For permissions please email: [email protected]. 2 ESC/EAS Guidelines Document Reviewers: Christian Mueller (ESC Review Coordinator) (Switzerland), Heinz Drexel (EAS Review Coordinator) (Austria), Victor Aboyans (France), Alberto Corsini1 (Italy), Wolfram Doehner (Germany), Michel Farnier (France), Bruna Gigante (Sweden), Meral Kayikcioglu1 (Turkey), Goran Krstacic (Croatia), Ekaterini Lambrinou (Cyprus), Basil S. Lewis (Israel), Josep Masip (Spain), Philippe Moulin1 (France), Steffen Petersen (United Kingdom), Anna Sonia Petronio (Italy), 1 1 Massimo Francesco Piepoli (Italy), Xavier Pinto (Spain), Lorenz Raber€ (Switzerland), Kausik K. Ray Downloaded from https://academic.oup.com/eurheartj/advance-article-abstract/doi/10.1093/eurheartj/ehz455/5556353 by guest on 23 September 2019 (United Kingdom), Zeljko Reiner1 (Croatia), Walter F. Riesen (Switzerland), Marco Roffi (Switzerland), Jean-Paul Schmid (Switzerland), Evgeny Shlyakhto (Russian Federation), Iain A. Simpson (United Kingdom), Erik Stroes1 (Netherlands), Isabella Sudano (Switzerland), Alexandros D. Tselepis1 (Greece), Margus Viigimaa1 (Estonia), Cecile Vindis (France), Alexander Vonbank (Austria), Michal Vrablik1 (Czech Republic), Mislav Vrsalovic (Croatia), Jose´ Luis Zamorano Gomez (Spain), Jean-Philippe Collet (ESC CPG Supervisor) (France) The disclosure forms of all experts involved in the development of these Guidelines are available on the ESC website www.escardio.org/guidelines For the Supplementary Data which include background information and detailed discussion of the data that have provided the basis for the Guidelines see https://academic.oup.com/eurheartj/article-lookup/doi/ 10.1093/eurheartj/ehz455#supplementary-data ................................................................................................................................................................................................... Keywords Guidelines • dyslipidaemias • cholesterol • triglycerides • low-density lipoproteins • high-density lipopro- teins • apolipoprotein B • lipoprotein(a) • lipoprotein remnants • total cardiovascular risk • treatment (lifestyle) • treatment (drugs) • treatment (adherence) • very low-density lipoproteins • familial hypercholesterolaemia . 5.3.4 Lipoprotein(a) and risk of atherosclerosis . 19 Table of contents . 5.4 Laboratory measurement of lipids and lipoproteins . 19 Abbreviationsandacronyms........................................ 4 . 5.4.1Lipoproteinmeasurement ............................... 19 . 1Preamble......................................................... 6 . 5.4.2Lipidmeasurements..................................... 20 2Introduction...................................................... 8 . 5.4.3Fastingornon-fasting? ................................... 20 . 2.1Whatisnewinthe2019Guidelines? .......................... 8 . 5.5 Recommendations for measuring lipids and lipoproteins to 3Whatiscardiovasculardiseaseprevention? ........................ 8 . estimate risk of atherosclerotic cardiovascular disease . 20 . 3.1Definitionandrationale....................................... 8 . 6Treatmenttargetsandgoals...................................... 21 . 3.2 Development of the Joint Task Force Guidelines for the . 7 Lifestyle modifications to improve the plasma lipid profile . 22 managementofdyslipidaemias.................................... 8 . 7.1 Influence of lifestyle on total cholesterol and low-density . 4Totalcardiovascularrisk .......................................... 8 . lipoproteincholesterollevels.................................... 24 4.1Totalcardiovascularriskestimation ........................... 8 . 7.2Influenceoflifestyleontriglyceridelevels ..................... 24 . 4.1.1 Rationale for assessing total cardiovascular disease risk . 11 . 7.3 Influence of lifestyle on high-density lipoprotein cholesterol 4.1.2 How to use the risk estimation charts . 14 . levels........................................................... 25 . 4.2Risklevels................................................... 15 . 7.4 Lifestyle recommendations to improve the plasma lipid 4.2.1 Role of non-invasive cardiovascular imaging . profile.......................................................... 25 . techniques in the assessment of total cardiovascular . 7.4.1Bodyweightandphysicalactivity......................... 25 diseaserisk................................................... 16 . 7.4.2Dietaryfat .............................................. 25 . 4.2.2Risk-basedinterventionstrategies.......................
Recommended publications
  • (AAV1-LPLS447X) Gene Therapy for Lipoprotein Lipase Deficiency
    Gene Therapy (2013) 20, 361–369 & 2013 Macmillan Publishers Limited All rights reserved 0969-7128/13 www.nature.com/gt ORIGINAL ARTICLE Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPLS447X) gene therapy for lipoprotein lipase deficiency: an open-label trial D Gaudet1,2,JMe´ thot1,2,SDe´ry1, D Brisson1,2, C Essiembre1, G Tremblay1, K Tremblay1,2, J de Wal3, J Twisk3, N van den Bulk3, V Sier-Ferreira3 and S van Deventer3 We describe the 2-year follow-up of an open-label trial (CT-AMT-011–01) of AAV1-LPLS447X gene therapy for lipoprotein lipase (LPL) deficiency (LPLD), an orphan disease associated with chylomicronemia, severe hypertriglyceridemia, metabolic complications and potentially life-threatening pancreatitis. The LPLS447X gene variant, in an adeno-associated viral vector of serotype 1 (alipogene tiparvovec), was administered to 14 adult LPLD patients with a prior history of pancreatitis. Primary objectives were to assess the long-term safety of alipogene tiparvovec and achieve a X40% reduction in fasting median plasma triglyceride (TG) at 3–12 weeks compared with baseline. Cohorts 1 (n ¼ 2) and 2 (n ¼ 4) received 3 Â 1011 gc kg À 1, and cohort 3 (n ¼ 8) received 1 Â 1012 gc kg À 1. Cohorts 2 and 3 also received immunosuppressants from the time of alipogene tiparvovec administration and continued for 12 weeks. Alipogene tiparvovec was well tolerated, without emerging safety concerns for 2 years. Half of the patients demonstrated a X40% reduction in fasting TG between 3 and 12 weeks. TG subsequently returned to baseline, although sustained LPLS447X expression and long-term changes in TG-rich lipoprotein characteristics were noted independently of the effect on fasting plasma TG.
    [Show full text]
  • Classification of Medicinal Drugs and Driving: Co-Ordination and Synthesis Report
    Project No. TREN-05-FP6TR-S07.61320-518404-DRUID DRUID Driving under the Influence of Drugs, Alcohol and Medicines Integrated Project 1.6. Sustainable Development, Global Change and Ecosystem 1.6.2: Sustainable Surface Transport 6th Framework Programme Deliverable 4.4.1 Classification of medicinal drugs and driving: Co-ordination and synthesis report. Due date of deliverable: 21.07.2011 Actual submission date: 21.07.2011 Revision date: 21.07.2011 Start date of project: 15.10.2006 Duration: 48 months Organisation name of lead contractor for this deliverable: UVA Revision 0.0 Project co-funded by the European Commission within the Sixth Framework Programme (2002-2006) Dissemination Level PU Public PP Restricted to other programme participants (including the Commission x Services) RE Restricted to a group specified by the consortium (including the Commission Services) CO Confidential, only for members of the consortium (including the Commission Services) DRUID 6th Framework Programme Deliverable D.4.4.1 Classification of medicinal drugs and driving: Co-ordination and synthesis report. Page 1 of 243 Classification of medicinal drugs and driving: Co-ordination and synthesis report. Authors Trinidad Gómez-Talegón, Inmaculada Fierro, M. Carmen Del Río, F. Javier Álvarez (UVa, University of Valladolid, Spain) Partners - Silvia Ravera, Susana Monteiro, Han de Gier (RUGPha, University of Groningen, the Netherlands) - Gertrude Van der Linden, Sara-Ann Legrand, Kristof Pil, Alain Verstraete (UGent, Ghent University, Belgium) - Michel Mallaret, Charles Mercier-Guyon, Isabelle Mercier-Guyon (UGren, University of Grenoble, Centre Regional de Pharmacovigilance, France) - Katerina Touliou (CERT-HIT, Centre for Research and Technology Hellas, Greece) - Michael Hei βing (BASt, Bundesanstalt für Straßenwesen, Germany).
    [Show full text]
  • 2019 ESC/EAS Guidelines for the Management of Dyslipidaemias
    Atherosclerosis 290 (2019) 140–205 Contents lists available at ScienceDirect Atherosclerosis journal homepage: www.elsevier.com/locate/atherosclerosis 2019 ESC/EAS guidelines for the management of dyslipidaemias: Lipid ☆ T modification to reduce cardiovascular risk ∗ ∗∗ Authors/Task Force Members (François Macha, ,2, Colin Baigentb, ,2, ∗∗∗ Alberico L. Catapanoc, ,1,2, Konstantinos C. Koskinasd, Manuela Casulae,f,1, Lina Badimong, M. John Chapmanh,i,cm,1, Guy G. De Backerj, Victoria Delgadok, Brian A. Ferencel, Ian M. Grahamm, Alison Hallidayn, Ulf Landmessero,p,q, Borislava Mihaylovar,s, Terje R. Pedersent, Gabriele Riccardiu,1, Dimitrios J. Richterv, Marc S. Sabatinew, Marja-Riitta Taskinenx,1, Lale Tokgozogluy,1, Olov Wiklundz), ESC National Cardiac Societies (Djamaleddine Nibouchean, Parounak H. Zelveianao, Peter Siostrzonekap, Ruslan Najafovaq, Philippe van de Bornear, Belma Pojskicas, Arman Postadzhiyanat, Lambros Kyprisau, Jindřich Špinarav, Mogens Lytken Larsenaw, Hesham Salah Eldinax, Margus Viigimaaay, Timo E. Strandbergaz, Jean Ferrièresba, Rusudan Agladzebb, Ulrich Laufsbc, Loukianos Rallidisbd, László Bajnokbe, Thorbjörn Gudjónssonbf, Vincent Maherbg, Yaakov Henkinbh, Michele Massimo Guliziabi, Aisulu Mussagaliyevabj, Gani Bajraktaribk, Alina Kerimkulovabl, Gustavs Latkovskisbm, Omar Hamouibn, Rimvydas Slapikasbo, Laurent Visserbp, Philip Dinglibq, Victoria Ivanovbr, Aneta Boskovicbs, Mbarek Nazzibt, Frank Visserenbu, Irena Mitevskabv, Kjetil Retterstølbw, Piotr Jankowskibx, Ricardo Fontes-Carvalhoby, Dan Gaitabz, Marat Ezhovca,
    [Show full text]
  • Small Dense Low-Density Lipoprotein: Biomarker Or Potential Drug Target?
    Published online: 02.10.2019 THIEME 92 SmallReview Dense Article Low-Density Lipoprotein Samanta Small Dense Low-Density Lipoprotein: Biomarker or Potential Drug Target? Basabdatta Samanta1 1Department of Biochemistry, Burdwan Medical College, West Address for correspondence Basabdatta Samanta, MBBS, MD, Bengal, India DNB, Department of Biochemistry, Burdwan Medical College, Burdwan 713104, West Bengal, India (e-mail: [email protected]). Ann Natl Acad Med Sci (India) 2019;55:92–97 Abstract Ischemic heart disease is currently an epidemic affecting individuals worldwide. Increased incidence along with earlier onset of disease has led to the constant search for biomarkers that will help in earlier identification and treatment of at risk individuals. Small dense low-density lipoprotein (sdLDL) is the atherogenic subtype of low-densi- ty lipoprotein (LDL). It is smaller in size and higher in density in comparison to other LDL subtypes. Higher levels of sdLDL have been found to be associated with increased incidence of ischemic heart disease and adverse outcomes. Properties including decreased resistance to oxidative stress and prolonged residence time in the circula- tion account for its increased atherogenic potential. Hence intervention approaches targeting sdLDL directly in at risk individuals may be beneficial. Genetic, lifestyle, and environmental factors affect sdLDL levels. But the main deter- mining factor is the level of triglycerides (TGs). Higher TG levels are associated with higher levels of very low density lipoprotein (VLDL) 1 and sdLDL. Various drugs Keywords have been used for targeting sdLDL with varying outcomes; drugs tried out include ► ischemic heart disease statins, fibrates, niacin, cholesterol ester transfer protein inhibitors and sodium-glu- ► small dense low - cose co-transporter-2 inhibitors.
    [Show full text]
  • Regulation of Pharmaceutical Prices: Evidence from a Reference Price Reform in Denmark
    A Service of Leibniz-Informationszentrum econstor Wirtschaft Leibniz Information Centre Make Your Publications Visible. zbw for Economics Kaiser, Ulrich; Mendez, Susan J.; Rønde, Thomas Working Paper Regulation of pharmaceutical prices: Evidence from a reference price reform in Denmark ZEW Discussion Papers, No. 10-062 Provided in Cooperation with: ZEW - Leibniz Centre for European Economic Research Suggested Citation: Kaiser, Ulrich; Mendez, Susan J.; Rønde, Thomas (2010) : Regulation of pharmaceutical prices: Evidence from a reference price reform in Denmark, ZEW Discussion Papers, No. 10-062, Zentrum für Europäische Wirtschaftsforschung (ZEW), Mannheim This Version is available at: http://hdl.handle.net/10419/41440 Standard-Nutzungsbedingungen: Terms of use: Die Dokumente auf EconStor dürfen zu eigenen wissenschaftlichen Documents in EconStor may be saved and copied for your Zwecken und zum Privatgebrauch gespeichert und kopiert werden. personal and scholarly purposes. Sie dürfen die Dokumente nicht für öffentliche oder kommerzielle You are not to copy documents for public or commercial Zwecke vervielfältigen, öffentlich ausstellen, öffentlich zugänglich purposes, to exhibit the documents publicly, to make them machen, vertreiben oder anderweitig nutzen. publicly available on the internet, or to distribute or otherwise use the documents in public. Sofern die Verfasser die Dokumente unter Open-Content-Lizenzen (insbesondere CC-Lizenzen) zur Verfügung gestellt haben sollten, If the documents have been made available under an Open gelten abweichend von diesen Nutzungsbedingungen die in der dort Content Licence (especially Creative Commons Licences), you genannten Lizenz gewährten Nutzungsrechte. may exercise further usage rights as specified in the indicated licence. www.econstor.eu Dis cus si on Paper No. 10-062 Regulation of Pharmaceutical Prices: Evidence from a Reference Price Reform in Denmark Ulrich Kaiser, Susan J.
    [Show full text]
  • LAI Expert Consensus Statement
    st st 8 Supplement to Journal of The Association of Physicians of India ■ Published on 1 of Every Month 1 November, 2020 Lipid Association of India Expert Consensus Statement on Management of Dyslipidemia in Indians 2020: Part III Expert Consensus Panel: Raman Puri, Chairman1, Vimal Mehta, Co-Chair2, SS Iyengar, Co-Chair3, SN Narasingan, Co-Chair4, P Barton Duell5, GB Sattur6, Krishnaswami Vijayaraghavan7, JC Mohan8, SK Wangnoo9, Jamshed Dalal10, D Prabhakar11, Rajeev Agarwal12, Manish Bansal13, Jamal Yusuf2, Saibal Mukhopadhyay2, Sadanand Shetty14, Prabhash Chand Manoria15, Avishkar Sabharwal16, Akshayaya Pradhan17, Rahul Mehrotra18, Sundeep Mishra19, Sonika Puri20, A Muruganathan21, Abdul Hamid Zargar22, Rashida Melinkari Patanwala23, Soumitra Kumar24, Neil Bardoloi25, KK Pareek26, Aditya Kapoor27, Ashu Rastogi28, Devaki R Nair29, Altamash Shaikh30, Chandra Mani Adhikari31, Muhammad Shoaib Momen Majumder32, Dheeraj Kapoor33, Madhur Yadav34, MR Mubarak35, AK Pancholia36, Rakesh Kumar Sahay37, Rashmi Nanda38, Nathan D Wong39 Introduction proposed by Lipid Association of India regarding various interventions based (LAI),8 identification and application on scientific evidence. The section on ndia is in the middle of an epidemic of lipid markers like lipoprotein (a) low LDL-C levels sums the evidence to Iof atherosclerotic cardiovascular [Lp(a)] and apolipoprotein B (apo the present date and gives justification disease (ASCVD) which is showing B) for risk stratification and control for lower proposed LDL-C goals. Since no signs of abating.1,2
    [Show full text]
  • The Personalized Medicine Report
    THE PERSONALIZED MEDICINE REPORT 2017 · Opportunity, Challenges, and the Future The Personalized Medicine Coalition gratefully acknowledges graduate students at Manchester University in North Manchester, Indiana, and at the University of Florida, who updated the appendix of this report under the guidance of David Kisor, Pharm.D., Director, Pharmacogenomics Education, Manchester University, and Stephan Schmidt, Ph.D., Associate Director, Pharmaceutics, University of Florida. The Coalition also acknowledges the contributions of its many members who offered insights and suggestions for the content in the report. CONTENTS INTRODUCTION 5 THE OPPORTUNITY 7 Benefits 9 Scientific Advancement 17 THE CHALLENGES 27 Regulatory Policy 29 Coverage and Payment Policy 35 Clinical Adoption 39 Health Information Technology 45 THE FUTURE 49 Conclusion 51 REFERENCES 53 APPENDIX 57 Selected Personalized Medicine Drugs and Relevant Biomarkers 57 HISTORICAL PRECEDENT For more than two millennia, medicine has maintained its aspiration of being personalized. In ancient times, Hippocrates combined an assessment of the four humors — blood, phlegm, yellow bile, and black bile — to determine the best course of treatment for each patient. Today, the sequence of the four chemical building blocks that comprise DNA, coupled with telltale proteins in the blood, enable more accurate medical predictions. The Personalized Medicine Report 5 INTRODUCTION When it comes to medicine, one size does not fit all. Treatments that help some patients are ineffective for others (Figure 1),1 and the same medicine may cause side effects in only certain patients. Yet, bound by the constructs of traditional disease, and, at the same time, increase the care delivery models, many of today’s doctors still efficiency of the health care system by improving prescribe therapies based on population averages.
    [Show full text]
  • 2019 ESC/EAS Guidelines for the Management of Dyslipidaemias
    Atherosclerosis 290 (2019) 140–205 Contents lists available at ScienceDirect Atherosclerosis journal homepage: www.elsevier.com/locate/atherosclerosis 2019 ESC/EAS guidelines for the management of dyslipidaemias: Lipid ☆ T modification to reduce cardiovascular risk ∗ ∗∗ Authors/Task Force Members (François Macha, ,2, Colin Baigentb, ,2, ∗∗∗ Alberico L. Catapanoc, ,1,2, Konstantinos C. Koskinad, Manuela Casulae,f,1, Lina Badimong, M. John Chapmanh,i,cm,1, Guy G. De Backerj, Victoria Delgadok, Brian A. Ferencel, Ian M. Grahamm, Alison Hallidayn, Ulf Landmessero,p,q, Borislava Mihaylovar,s, Terje R. Pedersent, Gabriele Riccardiu,1, Dimitrios J. Richterv, Marc S. Sabatinew, Marja-Riitta Taskinenx,1, Lale Tokgozogluy,1, Olov Wiklundz), ESC Committee for Practice Guidelines (CPG) (Stephan Windeckeraa, Victor Aboyansab, Colin Baigentac, Jean-Philippe Colletab, Veronica Deanab, Victoria Delgadoad, Donna Fitzsimonsac, Chris P. Galeac, Diederick Grobbeead, Sigrun Halvorsenae, Gerhard Hindricksaf, Bernard Iungab, Peter Jüniag, Hugo A. Katusaf, Ulf Landmesseraf, Christophe Leclercqab, Maddalena Lettinoah, Basil S. Lewisai, Bela Merkelyaj, Christian Muelleraa, Steffen Petersenac, Anna Sonia Petronioah, Dimitrios J. Richterak, Marco Roffiaa, Evgeny Shlyakhtoal, Iain A. Simpsonac, Miguel Sousa-Uvaam, Rhian M. Touyzac), ESC National Cardiac Societies (Djamaleddine Nibouchean, Parounak H. Zelveianao, Peter Siostrzonekap, Ruslan Najafovaq, Philippe van de Bornear, Belma Pojskicas, Arman Postadzhiyanat, Lambros Kyprisau, Jindřich Špinarav, Mogens Lytken
    [Show full text]
  • Long-Term Retrospective Analysis of Gene Therapy with Alipogene Tiparvovec and Its Effect on Lipoprotein Lipase Deficiency-Induced Pancreatitis
    RESEARCH ARTICLE Long-Term Retrospective Analysis of Gene Therapy with Alipogene Tiparvovec and Its Effect on Lipoprotein Lipase Deficiency-Induced Pancreatitis Daniel Gaudet,1,* Erik S. Stroes,2 Julie Me´ thot,1 Diane Brisson,1 Karine Tremblay,1 Sophie J. Bernelot Moens,2 Giorgio Iotti,3 Irene Rastelletti,3 Diego Ardigo,3 Deyanira Corzo,4 Christian Meyer,4 Marc Andersen,4 Philippe Ruszniewski,5 Mark Deakin,6 and Marco J. Bruno7 1Ecogene-21 Clinical and Translational Research Center and Lipidology Unit, Community Genetic Medicine Centre, Department of Medicine, Universite´ de Montreal, Montreal, Canada; 2Academic Medical Center, Amsterdam, The Netherlands; 3Chiesi Farmaceutici, Parma, Italy; 4uniQure B.V., Amsterdam, The Netherlands; 5Beaujon Hospital, Denis Diderot University, Paris, France; 6University Hospital of North Midlands, Stoke-on-Trent, United Kingdom; 7Erasmus Medical Centre, Rotterdam, The Netherlands. Alipogene tiparvovec (Glybera) is a gene therapy product approved in Europe under the ‘‘exceptional cir- cumstances’’ pathway as a treatment for lipoprotein lipase deficiency (LPLD), a rare genetic disease re- sulting in chylomicronemia and a concomitantly increased risk of acute and recurrent pancreatitis, with potentially lethal outcome. This retrospective study analyzed the frequency and severity of pancreatitis in 19 patients with LPLD up to 6 years after a single treatment with alipogene tiparvovec. An independent adjudication board of three pancreas experts, blinded to patient identification and to pre- or post-gene therapy period, performed a retrospective review of data extracted from the patients’ medical records and categorized LPLD-related acute abdominal pain events requiring hospital visits and/or hospitalizations based on the adapted 2012 Atlanta diagnostic criteria for pancreatitis.
    [Show full text]
  • Small Dense Low-Density Lipoprotein: Biomarker Or Potential Drug Target?
    Published online: 2019-10-02 THIEME 92 SmallReview Dense Article Low-Density Lipoprotein Samanta Small Dense Low-Density Lipoprotein: Biomarker or Potential Drug Target? Basabdatta Samanta1 1Department of Biochemistry, Burdwan Medical College, West Address for correspondence Basabdatta Samanta, MBBS, MD, Bengal, India DNB, Department of Biochemistry, Burdwan Medical College, Burdwan 713104, West Bengal, India (e-mail: [email protected]). Ann Natl Acad Med Sci (India) 2019;55:92–97 Abstract Ischemic heart disease is currently an epidemic affecting individuals worldwide. Increased incidence along with earlier onset of disease has led to the constant search for biomarkers that will help in earlier identification and treatment of at risk individuals. Small dense low-density lipoprotein (sdLDL) is the atherogenic subtype of low-densi- ty lipoprotein (LDL). It is smaller in size and higher in density in comparison to other LDL subtypes. Higher levels of sdLDL have been found to be associated with increased incidence of ischemic heart disease and adverse outcomes. Properties including decreased resistance to oxidative stress and prolonged residence time in the circula- tion account for its increased atherogenic potential. Hence intervention approaches targeting sdLDL directly in at risk individuals may be beneficial. Genetic, lifestyle, and environmental factors affect sdLDL levels. But the main deter- mining factor is the level of triglycerides (TGs). Higher TG levels are associated with higher levels of very low density lipoprotein (VLDL) 1 and sdLDL. Various drugs Keywords have been used for targeting sdLDL with varying outcomes; drugs tried out include ► ischemic heart disease statins, fibrates, niacin, cholesterol ester transfer protein inhibitors and sodium-glu- ► small dense low - cose co-transporter-2 inhibitors.
    [Show full text]
  • 2 12/ 35 74Al
    (12) INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization International Bureau (10) International Publication Number (43) International Publication Date 22 March 2012 (22.03.2012) 2 12/ 35 74 Al (51) International Patent Classification: (81) Designated States (unless otherwise indicated, for every A61K 9/16 (2006.01) A61K 9/51 (2006.01) kind of national protection available): AE, AG, AL, AM, A61K 9/14 (2006.01) AO, AT, AU, AZ, BA, BB, BG, BH, BR, BW, BY, BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DK, DM, DO, (21) International Application Number: DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, PCT/EP201 1/065959 HN, HR, HU, ID, IL, IN, IS, JP, KE, KG, KM, KN, KP, (22) International Filing Date: KR, KZ, LA, LC, LK, LR, LS, LT, LU, LY, MA, MD, 14 September 201 1 (14.09.201 1) ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, OM, PE, PG, PH, PL, PT, QA, RO, RS, RU, (25) Filing Language: English RW, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, TH, TJ, (26) Publication Language: English TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW. (30) Priority Data: 61/382,653 14 September 2010 (14.09.2010) US (84) Designated States (unless otherwise indicated, for every kind of regional protection available): ARIPO (BW, GH, (71) Applicant (for all designated States except US): GM, KE, LR, LS, MW, MZ, NA, SD, SL, SZ, TZ, UG, NANOLOGICA AB [SE/SE]; P.O Box 8182, S-104 20 ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, MD, RU, TJ, Stockholm (SE).
    [Show full text]
  • Alipogene Tiparvovec (AMT-011, Glybera) for Lipoprotein Lipase Deficiency
    Alipogene tiparvovec (AMT-011, Glybera) for lipoprotein lipase deficiency February 2009 This technology summary is based on information available at the time of research and a limited literature search. It is not intended to be a definitive statement on the safety, efficacy or effectiveness of the health technology covered and should not be used for commercial purposes. The National Horizon Scanning Centre Research Programme is part of the National Institute for Health Research February 2009 National Horizon Scanning Centre News on emerging technologies in healthcare Alipogene tiparvovec (Glybera) for lipoprotein lipase deficiency Target group • Lipoprotein lipase deficiency - also known as familial chylomicronaemia or hyperlipoproteinaemia type I. Background Lipoprotein lipase deficiency (LPLD) is an inherited metabolic disorder, characterised by abnormally elevated plasma concentrations of chylomicrons and triglycerides. LPLD includes patients classified as having hyperlipoproteinaemia type 1 (also known as familial chylomicronaemia) due to a deficiency of lipoprotein lipase (LPL) and those with a deficiency of apolipoprotein C-II, a lipase activating protein. LPL hydrolyses the triglyceride component of circulating chylomicrons and very low density lipoproteins (VLDL). When LPL activity is reduced, chylomicrons accumulate within the bloodstream and cause symptoms such as: abdominal pain, an enlarged spleen and liver, eruptive xanthomas and potentially lethal pancreatitis. The gene encoding for LPL is located on chromosome 8 and is expressed mainly in skeletal muscle, adipose tissue, and heart muscle. Technology description Alipogene tiparvovec (AMT-011, Glybera) is an adeno-associated viral vector (AAV1) based gene therapy, administered intramuscularly (IM) at multiple-sites in a single session. AAV1 carrying the human variant LPLS447X gene is delivered to skeletal muscle, where it becomes active.
    [Show full text]