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A cross-national investigation of cardiovascular survival in homozygous familial hypercholesterolemia: The Sino-Roman Study

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收录情况： ◇ SCIE

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机构： [1]Extracorporeal Therapeutic Techniques Unit, Lipid Clinic and Atherosclerosis Prevention Centre, Immunohematology and Transfusion Medicine, ‘‘Umberto I’’ [2]Department of Molecular Medicine, ‘‘Sapienza’’ University of Rome, Rome, Italy [3]School of Medicine, Faculty of Health and Medical Sciences, University of Western Australia, Perth, Western Australia, Australia [4]Department of Atherosclerosis, Beijing Anzhen Hospital, Capital Medical University, Beijing, China [5]Key Laboratory of Genome Sciences and Information, Beijing Institute of Genomics, Chinese Academy of Sciences, Beijing, China [6]Lipid Disorders Clinic, Cardiometabolic Service, Department of Cardiology, Royal Perth Hospital, Perth, Western Australia, Australia [7]and Beijing Institute of Heart, Lung and Blood Vessel Diseases, Beijing Anzhen Hospital, Capital Medical University, Beijing, China

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关键词： China Familial hypercholesterolemia Homozygous Italy Lipoprotein apheresis Novel therapies

摘要：

Background: Homozygous familial hypercholesterolemia (hoFH) is a rare inherited disorder characterized by extreme elevation of low-density lipoprotein (LDL) cholesterol, accelerated coronary artery disease, and premature death. Aggressive LDL-lowering therapies are important for survival, but these are not available worldwide. Objective: The aim of the study was to compare and contrast cardiovascular outcomes and mortality of hoFH patients in 2 countries with disparate use of lipoprotein apheresis (LA) and modern therapies for lowering LDL cholesterol. Methods: A retrospective study was undertaken comparing cardiovascular disease (CVD)-free survival and mortality in 44 hoFH patients who were treated with statins but not LA, from a center in Beijing, China, and 18 hoFH patients who were treated with LA and novel therapies from an early age, from a center in Rome, Italy. Results: CVD-free survival and survival were significantly reduced in Chinese patients compared with the Italian patients after 30 years of follow-up (log-rank P <.01). In a pooled analysis, cardiovascular survival was significantly increased with earlier age at treatment, longer duration of treatment, and lower on-treatment LDL cholesterol concentrations (P <.05). In addition, the probability of a CVD event and death were increased in patients that carried a null mutation in the LDLR or had elevated lipoprotein(a). Conclusions: We show that coronary artery disease outcomes in patients with hoFH can be significantly improved with earlier and potent LDL cholesterol lowering with pharmacotherapies and LA. This has major implications for countries, such as China, where the models of care for hoFH remains underdeveloped. © 2019 National Lipid Association

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出版当年[2018]版：

大类 | 2 区

医学

小类 | 1 区药学

最新[2023]版：

大类 | 3 区医学

小类 | 3 区药学

JCR分区：

出版当年[2017]版：

Q1 PHARMACOLOGY & PHARMACY

最新[2023]版：

Q2 PHARMACOLOGY & PHARMACY

影响因子： 3.6 最新[2023版] 3.8 最新五年平均 3.58 出版当年[2017版] 3.934 出版当年五年平均 5.812 出版前一年[2016版] 3.581 出版后一年[2018版]

第一作者：

第一作者机构： [1]Extracorporeal Therapeutic Techniques Unit, Lipid Clinic and Atherosclerosis Prevention Centre, Immunohematology and Transfusion Medicine, ‘‘Umberto I’’ [2]Department of Molecular Medicine, ‘‘Sapienza’’ University of Rome, Rome, Italy

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通讯作者：

通讯机构： [3]School of Medicine, Faculty of Health and Medical Sciences, University of Western Australia, Perth, Western Australia, Australia [4]Department of Atherosclerosis, Beijing Anzhen Hospital, Capital Medical University, Beijing, China [6]Lipid Disorders Clinic, Cardiometabolic Service, Department of Cardiology, Royal Perth Hospital, Perth, Western Australia, Australia [7]and Beijing Institute of Heart, Lung and Blood Vessel Diseases, Beijing Anzhen Hospital, Capital Medical University, Beijing, China [*1]School of Medicine, University of Western Australia, GPO Box X2213, Perth, Western Australia 6847, Australia. [*2]Beijing Anzhen Hospital, Capital Medical University, No.2 Anzhen Road, Chaoyang District, Beijing 100029, China.

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A cross-national investigation of cardiovascular survival in homozygous familial hypercholesterolemia: The Sino-Roman Study

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