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.
Identifieur interne : 000873 ( Main/Exploration ); précédent : 000872; suivant : 000874Long-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.
Auteurs : Frederick J. Raal [Afrique du Sud] ; G Kees Hovingh [Pays-Bas] ; Dirk Blom [Afrique du Sud] ; Raul D. Santos [Brésil] ; Mariko Harada-Shiba [Japon] ; Eric Bruckert [France] ; Patrick Couture [Canada] ; Handrean Soran [Royaume-Uni] ; Gerald F. Watts [Australie] ; Christopher Kurtz [États-Unis] ; Narimon Honarpour [États-Unis] ; Lihua Tang [États-Unis] ; Sree Kasichayanula [États-Unis] ; Scott M. Wasserman [États-Unis] ; Evan A. Stein [États-Unis]Source :
- The lancet. Diabetes & endocrinology [ 2213-8595 ] ; 2017.
Abstract
Homozygous familial hypercholesterolaemia is a genetic disorder characterised by substantially raised LDL cholesterol, reduced LDL receptor function, xanthomas, and cardiovascular disease before age 20 years. Conventional therapy is with statins, ezetimibe, and apheresis. We aimed to assess the long-term safety and efficacy of the proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitor evolocumab in a subset of patients with homozygous familial hypercholesterolaemia enrolled in an open-label, non-randomised phase 3 trial.
DOI: 10.1016/S2213-8587(17)30044-X
PubMed: 28215937
Affiliations:
- Afrique du Sud, Australie, Brésil, Canada, France, Japon, Pays-Bas, Royaume-Uni, États-Unis
- Angleterre, Californie, Grand Manchester, Hollande-Septentrionale, Ohio, État de São Paulo, Île-de-France
- Amsterdam, Manchester, Paris, São Paulo
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Le document en format XML
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Raal</name><affiliation wicri:level="1"><nlm:affiliation>Carbohydrate and Lipid Metabolism Research Unit, Faculty of Health Sciences, University of Witwatersrand, Johannesburg, South Africa.</nlm:affiliation><country xml:lang="fr">Afrique du Sud</country><wicri:regionArea>Carbohydrate and Lipid Metabolism Research Unit, Faculty of Health Sciences, University of Witwatersrand, Johannesburg</wicri:regionArea><wicri:noRegion>Johannesburg</wicri:noRegion></affiliation></author><author><name sortKey="Hovingh, G Kees" sort="Hovingh, G Kees" uniqKey="Hovingh G" first="G Kees" last="Hovingh">G Kees Hovingh</name><affiliation wicri:level="3"><nlm:affiliation>Department of Vascular Medicine, Academisch Medisch Centrum, Amsterdam, Netherlands.</nlm:affiliation><country xml:lang="fr">Pays-Bas</country><wicri:regionArea>Department of Vascular Medicine, Academisch Medisch Centrum, Amsterdam</wicri:regionArea><placeName><settlement type="city">Amsterdam</settlement><region nuts="2" type="province">Hollande-Septentrionale</region></placeName></affiliation></author><author><name sortKey="Blom, Dirk" sort="Blom, Dirk" uniqKey="Blom D" first="Dirk" last="Blom">Dirk Blom</name><affiliation wicri:level="1"><nlm:affiliation>Division of Lipidology, Department of Medicine, University of Cape Town, Cape Town, South Africa.</nlm:affiliation><country xml:lang="fr">Afrique du Sud</country><wicri:regionArea>Division of Lipidology, Department of Medicine, University of Cape Town, Cape Town</wicri:regionArea><wicri:noRegion>Cape Town</wicri:noRegion></affiliation></author><author><name sortKey="Santos, Raul D" sort="Santos, Raul D" uniqKey="Santos R" first="Raul D" last="Santos">Raul D. Santos</name><affiliation wicri:level="3"><nlm:affiliation>Lipid Clinic Heart Institute (InCor), University of Sao Paulo Medical School Hospital and Preventive Medicine Center and Cardiology Program, Hospital Israelita Albert Einstein, Sao Paulo, Brazil.</nlm:affiliation><country xml:lang="fr">Brésil</country><wicri:regionArea>Lipid Clinic Heart Institute (InCor), University of Sao Paulo Medical School Hospital and Preventive Medicine Center and Cardiology Program, Hospital Israelita Albert Einstein, Sao Paulo</wicri:regionArea><placeName><settlement type="city">São Paulo</settlement><region type="state">État de São Paulo</region></placeName></affiliation></author><author><name sortKey="Harada Shiba, Mariko" sort="Harada Shiba, Mariko" uniqKey="Harada Shiba M" first="Mariko" last="Harada-Shiba">Mariko Harada-Shiba</name><affiliation wicri:level="1"><nlm:affiliation>Department of Molecular Innovation in Lipidology, National Cerebral and Cardiovascular Center Research Institute, Osaka, Japan.</nlm:affiliation><country xml:lang="fr">Japon</country><wicri:regionArea>Department of Molecular Innovation in Lipidology, National Cerebral and Cardiovascular Center Research Institute, Osaka</wicri:regionArea><wicri:noRegion>Osaka</wicri:noRegion></affiliation></author><author><name sortKey="Bruckert, Eric" sort="Bruckert, Eric" uniqKey="Bruckert E" first="Eric" last="Bruckert">Eric Bruckert</name><affiliation wicri:level="3"><nlm:affiliation>Hôspital Pitié Salpêtrière, Assistance-Publique Hôpitaux de Paris, Paris, France.</nlm:affiliation><country xml:lang="fr">France</country><wicri:regionArea>Hôspital Pitié Salpêtrière, Assistance-Publique Hôpitaux de Paris, Paris</wicri:regionArea><placeName><region type="region">Île-de-France</region><region type="old region">Île-de-France</region><settlement type="city">Paris</settlement></placeName></affiliation></author><author><name sortKey="Couture, Patrick" sort="Couture, Patrick" uniqKey="Couture P" first="Patrick" last="Couture">Patrick Couture</name><affiliation wicri:level="1"><nlm:affiliation>Centre Hospitalier Universitaire de Québec, Quebec City, QC, Canada.</nlm:affiliation><country xml:lang="fr">Canada</country><wicri:regionArea>Centre Hospitalier Universitaire de Québec, Quebec City, QC</wicri:regionArea><wicri:noRegion>QC</wicri:noRegion></affiliation></author><author><name sortKey="Soran, Handrean" sort="Soran, Handrean" uniqKey="Soran H" first="Handrean" last="Soran">Handrean Soran</name><affiliation wicri:level="3"><nlm:affiliation>Cardiovascular Trials Unit, Central Manchester University Hospitals NHS Foundation Trust, Manchester, UK.</nlm:affiliation><country xml:lang="fr">Royaume-Uni</country><wicri:regionArea>Cardiovascular Trials Unit, Central Manchester University Hospitals NHS Foundation Trust, Manchester</wicri:regionArea><placeName><settlement type="city">Manchester</settlement><region type="country">Angleterre</region><region type="région" nuts="1">Grand Manchester</region></placeName></affiliation></author><author><name sortKey="Watts, Gerald F" sort="Watts, Gerald F" uniqKey="Watts G" first="Gerald F" last="Watts">Gerald F. Watts</name><affiliation wicri:level="1"><nlm:affiliation>Lipid Disorders Clinic, Department of Cardiology, Royal Perth Hospital, School of Medicine and Pharmacology, University of Western Australia, Perth, WA, Australia.</nlm:affiliation><country xml:lang="fr">Australie</country><wicri:regionArea>Lipid Disorders Clinic, Department of Cardiology, Royal Perth Hospital, School of Medicine and Pharmacology, University of Western Australia, Perth, WA</wicri:regionArea><wicri:noRegion>WA</wicri:noRegion></affiliation></author><author><name sortKey="Kurtz, Christopher" sort="Kurtz, Christopher" uniqKey="Kurtz C" first="Christopher" last="Kurtz">Christopher Kurtz</name><affiliation wicri:level="2"><nlm:affiliation>Amgen, Thousand Oaks, CA, USA.</nlm:affiliation><country xml:lang="fr">États-Unis</country><wicri:regionArea>Amgen, Thousand Oaks, CA</wicri:regionArea><placeName><region type="state">Californie</region></placeName></affiliation></author><author><name sortKey="Honarpour, Narimon" sort="Honarpour, Narimon" uniqKey="Honarpour N" first="Narimon" last="Honarpour">Narimon Honarpour</name><affiliation wicri:level="2"><nlm:affiliation>Amgen, Thousand Oaks, CA, USA.</nlm:affiliation><country xml:lang="fr">États-Unis</country><wicri:regionArea>Amgen, Thousand Oaks, CA</wicri:regionArea><placeName><region type="state">Californie</region></placeName></affiliation></author><author><name sortKey="Tang, Lihua" sort="Tang, Lihua" uniqKey="Tang L" first="Lihua" last="Tang">Lihua Tang</name><affiliation wicri:level="2"><nlm:affiliation>Amgen, Thousand Oaks, CA, USA.</nlm:affiliation><country xml:lang="fr">États-Unis</country><wicri:regionArea>Amgen, Thousand Oaks, CA</wicri:regionArea><placeName><region type="state">Californie</region></placeName></affiliation></author><author><name sortKey="Kasichayanula, Sree" sort="Kasichayanula, Sree" uniqKey="Kasichayanula S" first="Sree" last="Kasichayanula">Sree Kasichayanula</name><affiliation wicri:level="2"><nlm:affiliation>Amgen, Thousand Oaks, CA, USA.</nlm:affiliation><country xml:lang="fr">États-Unis</country><wicri:regionArea>Amgen, Thousand Oaks, CA</wicri:regionArea><placeName><region type="state">Californie</region></placeName></affiliation></author><author><name sortKey="Wasserman, Scott M" sort="Wasserman, Scott M" uniqKey="Wasserman S" first="Scott M" last="Wasserman">Scott M. Wasserman</name><affiliation wicri:level="2"><nlm:affiliation>Amgen, Thousand Oaks, CA, USA.</nlm:affiliation><country xml:lang="fr">États-Unis</country><wicri:regionArea>Amgen, Thousand Oaks, CA</wicri:regionArea><placeName><region type="state">Californie</region></placeName></affiliation></author><author><name sortKey="Stein, Evan A" sort="Stein, Evan A" uniqKey="Stein E" first="Evan A" last="Stein">Evan A. Stein</name><affiliation wicri:level="2"><nlm:affiliation>Metabolic and Atherosclerosis Research Center, Cincinnati, OH, USA. Electronic address: esteinmrl@aol.com.</nlm:affiliation><country xml:lang="fr">États-Unis</country><wicri:regionArea>Metabolic and Atherosclerosis Research Center, Cincinnati, OH</wicri:regionArea><placeName><region type="state">Ohio</region></placeName></affiliation></author></analytic><series><title level="j">The lancet. Diabetes & endocrinology</title><idno type="eISSN">2213-8595</idno><imprint><date when="2017" type="published">2017</date></imprint></series></biblStruct></sourceDesc></fileDesc><profileDesc><textClass></textClass></profileDesc></teiHeader><front><div type="abstract" xml:lang="en">Homozygous familial hypercholesterolaemia is a genetic disorder characterised by substantially raised LDL cholesterol, reduced LDL receptor function, xanthomas, and cardiovascular disease before age 20 years. Conventional therapy is with statins, ezetimibe, and apheresis. We aimed to assess the long-term safety and efficacy of the proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitor evolocumab in a subset of patients with homozygous familial hypercholesterolaemia enrolled in an open-label, non-randomised phase 3 trial.</div></front></TEI><affiliations><list><country><li>Afrique du Sud</li><li>Australie</li><li>Brésil</li><li>Canada</li><li>France</li><li>Japon</li><li>Pays-Bas</li><li>Royaume-Uni</li><li>États-Unis</li></country><region><li>Angleterre</li><li>Californie</li><li>Grand Manchester</li><li>Hollande-Septentrionale</li><li>Ohio</li><li>État de São Paulo</li><li>Île-de-France</li></region><settlement><li>Amsterdam</li><li>Manchester</li><li>Paris</li><li>São Paulo</li></settlement></list><tree><country name="Afrique du Sud"><noRegion><name sortKey="Raal, Frederick J" sort="Raal, Frederick J" uniqKey="Raal F" first="Frederick J" last="Raal">Frederick J. 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