Systemic sclerosis-associated pulmonary hypertension: why disease-specific composite endpoints are needed
Identifieur interne : 006030 ( Main/Exploration ); précédent : 006029; suivant : 006031Systemic sclerosis-associated pulmonary hypertension: why disease-specific composite endpoints are needed
Auteurs : Christopher P. Denton [Royaume-Uni] ; Jerome Avouac [France] ; Frank Behrens [Allemagne] ; Daniel E. Furst [États-Unis] ; Ivan Foeldvari [Allemagne] ; Marc Humbert [France] ; Doerte Huscher [Allemagne] ; Otylia Kowal-Bielecka [Pologne] ; Marco Matucci-Cerinic [Italie] ; Peter Nash [Australie] ; Christian F. Opitz [Allemagne] ; David Pittrow [Allemagne] ; Lewis J. Rubin [États-Unis] ; James R. Seibold [États-Unis] ; Oliver Distler [Suisse]Source :
- Arthritis Research & Therapy [ 1478-6354 ] ; 2011.
Abstract
Pulmonary arterial hypertension (PAH) is a serious complication of systemic sclerosis (SSc). In clinical trials PAH-SSc has been grouped with other forms, including idiopathic PAH. The primary endpoint for most pivotal studies was improvement in exercise capacity. However, composite clinical endpoints that better reflect long-term outcome may be more meaningful. We discuss potential endpoints and consider why the same measures may not be appropriate for both idiopathic PAH and PAH-SSc due to inherent differences in clinical outcome and management strategies of these two forms of PAH. Failure to take this into account may compromise progress in managing PAH in SSc.
Url:
DOI: 10.1186/ar3346
PubMed: 21699746
PubMed Central: 3218892
Affiliations:
- Allemagne, Australie, France, Italie, Pologne, Royaume-Uni, Suisse, États-Unis
- Berlin, Californie, Connecticut, District de Darmstadt, District de Dresde, Hesse (Land), Saxe (Land), Île-de-France
- Berlin, Dresde, Francfort-sur-le-Main, Orsay
- Université Paris-Sud
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Denton</name><affiliation wicri:level="1"><nlm:aff id="I1">Centre for Rheumatology, Royal Free Campus, University College Medical School, London, NW3 2PF, UK</nlm:aff><country xml:lang="fr">Royaume-Uni</country><wicri:regionArea>Centre for Rheumatology, Royal Free Campus, University College Medical School, London, NW3 2PF</wicri:regionArea><wicri:noRegion>NW3 2PF</wicri:noRegion></affiliation></author><author><name sortKey="Avouac, Jerome" sort="Avouac, Jerome" uniqKey="Avouac J" first="Jerome" last="Avouac">Jerome Avouac</name><affiliation wicri:level="1"><nlm:aff id="I2">Rheumatology A Department, RDU, Paris, 75006, France</nlm:aff><country xml:lang="fr">France</country><wicri:regionArea>Rheumatology A Department, RDU, Paris, 75006</wicri:regionArea><wicri:noRegion>75006</wicri:noRegion><wicri:noRegion>75006</wicri:noRegion></affiliation></author><author><name sortKey="Behrens, Frank" sort="Behrens, Frank" uniqKey="Behrens F" first="Frank" last="Behrens">Frank Behrens</name><affiliation wicri:level="3"><nlm:aff id="I3">German Rheumatism Research Centre, 10117 Berlin, Germany</nlm:aff><country xml:lang="fr">Allemagne</country><wicri:regionArea>German Rheumatism Research Centre, 10117 Berlin</wicri:regionArea><placeName><region type="land" nuts="3">Berlin</region><settlement type="city">Berlin</settlement></placeName></affiliation></author><author><name sortKey="Furst, Daniel E" sort="Furst, Daniel E" uniqKey="Furst D" first="Daniel E" last="Furst">Daniel E. Furst</name><affiliation wicri:level="2"><nlm:aff id="I4">Division of Rheumatology, Department of Medicine, David Geffen School at UCLA, Los Angeles, CA 90095, USA</nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>Division of Rheumatology, Department of Medicine, David Geffen School at UCLA, Los Angeles, CA 90095</wicri:regionArea><placeName><region type="state">Californie</region></placeName></affiliation></author><author><name sortKey="Foeldvari, Ivan" sort="Foeldvari, Ivan" uniqKey="Foeldvari I" first="Ivan" last="Foeldvari">Ivan Foeldvari</name><affiliation wicri:level="1"><nlm:aff id="I5">Pediatric Rheumatology Clinic, General Hospital Eilbek, 22081 Germany</nlm:aff><country xml:lang="fr">Allemagne</country><wicri:regionArea>Pediatric Rheumatology Clinic, General Hospital Eilbek</wicri:regionArea><wicri:noRegion>General Hospital Eilbek</wicri:noRegion><wicri:noRegion>General Hospital Eilbek</wicri:noRegion><wicri:noRegion>General Hospital Eilbek</wicri:noRegion></affiliation></author><author><name sortKey="Humbert, Marc" sort="Humbert, Marc" uniqKey="Humbert M" first="Marc" last="Humbert">Marc Humbert</name><affiliation wicri:level="4"><nlm:aff id="I6">Service de Pneumologie et Reanimation Respiratoire, Centre des Maladies Vasculaires Pulmonaires, Hopital Antoine-Beclere, Université Paris-Sud, Clamart, 92140, France</nlm:aff><country xml:lang="fr">France</country><wicri:regionArea>Service de Pneumologie et Reanimation Respiratoire, Centre des Maladies Vasculaires Pulmonaires, Hopital Antoine-Beclere, Université Paris-Sud, Clamart, 92140</wicri:regionArea><wicri:noRegion>92140</wicri:noRegion><orgName type="university">Université Paris-Sud</orgName><placeName><settlement type="city">Orsay</settlement><region type="region" nuts="2">Île-de-France</region></placeName></affiliation></author><author><name sortKey="Huscher, Doerte" sort="Huscher, Doerte" uniqKey="Huscher D" first="Doerte" last="Huscher">Doerte Huscher</name><affiliation wicri:level="3"><nlm:aff id="I7">Division of Rheumatology/ZAFES, JW Goethe University, 60325 Frankfurt, Germany</nlm:aff><country xml:lang="fr">Allemagne</country><wicri:regionArea>Division of Rheumatology/ZAFES, JW Goethe University, 60325 Frankfurt</wicri:regionArea><placeName><region type="land" nuts="1">Hesse (Land)</region><region type="district" nuts="2">District de Darmstadt</region><settlement type="city">Francfort-sur-le-Main</settlement></placeName></affiliation></author><author><name sortKey="Kowal Bielecka, Otylia" sort="Kowal Bielecka, Otylia" uniqKey="Kowal Bielecka O" first="Otylia" last="Kowal-Bielecka">Otylia Kowal-Bielecka</name><affiliation wicri:level="1"><nlm:aff id="I8">Department of Rheumatology and Internal Medicine, Medical University of Bialystok, 15-089, Poland</nlm:aff><country xml:lang="fr">Pologne</country><wicri:regionArea>Department of Rheumatology and Internal Medicine, Medical University of Bialystok, 15-089</wicri:regionArea><wicri:noRegion>15-089</wicri:noRegion></affiliation></author><author><name sortKey="Matucci Cerinic, Marco" sort="Matucci Cerinic, Marco" uniqKey="Matucci Cerinic M" first="Marco" last="Matucci-Cerinic">Marco Matucci-Cerinic</name><affiliation wicri:level="1"><nlm:aff id="I9">Department of Medicine, Division of Rheumatology, Denothe Center, University of Florence, 50134, Italy</nlm:aff><country xml:lang="fr">Italie</country><wicri:regionArea>Department of Medicine, Division of Rheumatology, Denothe Center, University of Florence, 50134</wicri:regionArea><wicri:noRegion>50134</wicri:noRegion></affiliation></author><author><name sortKey="Nash, Peter" sort="Nash, Peter" uniqKey="Nash P" first="Peter" last="Nash">Peter Nash</name><affiliation wicri:level="1"><nlm:aff id="I10">Rheumatology Research Unit, Cotton Tree, Sunshine Coast, Queensland, 4558, Australia</nlm:aff><country xml:lang="fr">Australie</country><wicri:regionArea>Rheumatology Research Unit, Cotton Tree, Sunshine Coast, Queensland, 4558</wicri:regionArea><wicri:noRegion>4558</wicri:noRegion></affiliation></author><author><name sortKey="Opitz, Christian F" sort="Opitz, Christian F" uniqKey="Opitz C" first="Christian F" last="Opitz">Christian F. Opitz</name><affiliation wicri:level="3"><nlm:aff id="I11">Klinik für Innere Medizin, DRK-Kliniken Berlin Köpenick, 12559 Berlin, Germany</nlm:aff><country xml:lang="fr">Allemagne</country><wicri:regionArea>Klinik für Innere Medizin, DRK-Kliniken Berlin Köpenick, 12559 Berlin</wicri:regionArea><placeName><region type="land" nuts="3">Berlin</region><settlement type="city">Berlin</settlement></placeName></affiliation></author><author><name sortKey="Pittrow, David" sort="Pittrow, David" uniqKey="Pittrow D" first="David" last="Pittrow">David Pittrow</name><affiliation wicri:level="3"><nlm:aff id="I12">Institute for Clinical Pharmacology, Medical Faculty, Technical University, 01187 Dresden, Germany</nlm:aff><country xml:lang="fr">Allemagne</country><wicri:regionArea>Institute for Clinical Pharmacology, Medical Faculty, Technical University, 01187 Dresden</wicri:regionArea><placeName><region type="land" nuts="1">Saxe (Land)</region><region type="district" nuts="2">District de Dresde</region><settlement type="city">Dresde</settlement></placeName></affiliation></author><author><name sortKey="Rubin, Lewis J" sort="Rubin, Lewis J" uniqKey="Rubin L" first="Lewis J" last="Rubin">Lewis J. Rubin</name><affiliation wicri:level="2"><nlm:aff id="I13">Division of Pulmonary and Critical Care Medicine, University of California, San Diego School of Medicine, La Jolla, CA 92093-0348 USA</nlm:aff><country xml:lang="fr">États-Unis</country><placeName><region type="state">Californie</region></placeName><wicri:cityArea>Division of Pulmonary and Critical Care Medicine, University of California, San Diego School of Medicine, La Jolla</wicri:cityArea></affiliation></author><author><name sortKey="Seibold, James R" sort="Seibold, James R" uniqKey="Seibold J" first="James R" last="Seibold">James R. Seibold</name><affiliation wicri:level="2"><nlm:aff id="I14">Scleroderma Research Consultants, CT 06001, USA</nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>Scleroderma Research Consultants, CT 06001</wicri:regionArea><placeName><region type="state">Connecticut</region></placeName></affiliation></author><author><name sortKey="Distler, Oliver" sort="Distler, Oliver" uniqKey="Distler O" first="Oliver" last="Distler">Oliver Distler</name><affiliation wicri:level="1"><nlm:aff id="I15">Department of Rheumatology, University Hospital Zurich, CH-8091, Switzerland</nlm:aff><country xml:lang="fr">Suisse</country><wicri:regionArea>Department of Rheumatology, University Hospital Zurich, CH-8091</wicri:regionArea><wicri:noRegion>CH-8091</wicri:noRegion></affiliation></author></analytic><series><title level="j">Arthritis Research & Therapy</title><idno type="ISSN">1478-6354</idno><idno type="eISSN">1478-6362</idno><imprint><date when="2011">2011</date></imprint></series></biblStruct></sourceDesc></fileDesc><profileDesc><textClass></textClass></profileDesc></teiHeader><front><div type="abstract" xml:lang="en"><p>Pulmonary arterial hypertension (PAH) is a serious complication of systemic sclerosis (SSc). In clinical trials PAH-SSc has been grouped with other forms, including idiopathic PAH. The primary endpoint for most pivotal studies was improvement in exercise capacity. However, composite clinical endpoints that better reflect long-term outcome may be more meaningful. We discuss potential endpoints and consider why the same measures may not be appropriate for both idiopathic PAH and PAH-SSc due to inherent differences in clinical outcome and management strategies of these two forms of PAH. Failure to take this into account may compromise progress in managing PAH in SSc.</p></div></front><back><div1 type="bibliography"><listBibl><biblStruct><analytic><author><name sortKey="Simonneau, G" uniqKey="Simonneau G">G Simonneau</name></author><author><name sortKey="Galie, N" uniqKey="Galie N">N Galiè</name></author><author><name sortKey="Rubin, Lj" uniqKey="Rubin L">LJ Rubin</name></author><author><name sortKey="Langleben, D" uniqKey="Langleben D">D Langleben</name></author><author><name sortKey="Seeger, W" uniqKey="Seeger W">W Seeger</name></author><author><name sortKey="Domenighetti, G" uniqKey="Domenighetti G">G Domenighetti</name></author><author><name sortKey="Gibbs, S" uniqKey="Gibbs S">S Gibbs</name></author><author><name sortKey="Lebrec, D" uniqKey="Lebrec D">D Lebrec</name></author><author><name sortKey="Speich, R" uniqKey="Speich R">R Speich</name></author><author><name sortKey="Beghetti, M" uniqKey="Beghetti M">M Beghetti</name></author><author><name sortKey="Rich, S" 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sortKey="Kim, Nh" uniqKey="Kim N">NH Kim</name></author><author><name sortKey="Oudiz, Rj" uniqKey="Oudiz R">RJ Oudiz</name></author><author><name sortKey="Peacock, A" uniqKey="Peacock A">A Peacock</name></author><author><name sortKey="Provencher, S" uniqKey="Provencher S">S Provencher</name></author><author><name sortKey="Sitbon, O" uniqKey="Sitbon O">O Sitbon</name></author><author><name sortKey="Tapson, Vf" uniqKey="Tapson V">VF Tapson</name></author><author><name sortKey="Seeger, W" uniqKey="Seeger W">W Seeger</name></author></analytic></biblStruct></listBibl></div1></back></TEI><affiliations><list><country><li>Allemagne</li><li>Australie</li><li>France</li><li>Italie</li><li>Pologne</li><li>Royaume-Uni</li><li>Suisse</li><li>États-Unis</li></country><region><li>Berlin</li><li>Californie</li><li>Connecticut</li><li>District de Darmstadt</li><li>District de Dresde</li><li>Hesse (Land)</li><li>Saxe (Land)</li><li>Île-de-France</li></region><settlement><li>Berlin</li><li>Dresde</li><li>Francfort-sur-le-Main</li><li>Orsay</li></settlement><orgName><li>Université Paris-Sud</li></orgName></list><tree><country name="Royaume-Uni"><noRegion><name sortKey="Denton, Christopher P" sort="Denton, Christopher P" uniqKey="Denton C" first="Christopher P" last="Denton">Christopher P. Denton</name></noRegion></country><country name="France"><noRegion><name sortKey="Avouac, Jerome" sort="Avouac, Jerome" uniqKey="Avouac J" first="Jerome" last="Avouac">Jerome Avouac</name></noRegion><name sortKey="Humbert, Marc" sort="Humbert, Marc" uniqKey="Humbert M" first="Marc" last="Humbert">Marc Humbert</name></country><country name="Allemagne"><region name="Berlin"><name sortKey="Behrens, Frank" sort="Behrens, Frank" uniqKey="Behrens F" first="Frank" last="Behrens">Frank Behrens</name></region><name sortKey="Foeldvari, Ivan" sort="Foeldvari, Ivan" uniqKey="Foeldvari I" first="Ivan" last="Foeldvari">Ivan Foeldvari</name><name sortKey="Huscher, Doerte" sort="Huscher, Doerte" uniqKey="Huscher D" first="Doerte" last="Huscher">Doerte Huscher</name><name sortKey="Opitz, Christian F" sort="Opitz, Christian F" uniqKey="Opitz C" first="Christian F" last="Opitz">Christian F. 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Seibold</name></country><country name="Pologne"><noRegion><name sortKey="Kowal Bielecka, Otylia" sort="Kowal Bielecka, Otylia" uniqKey="Kowal Bielecka O" first="Otylia" last="Kowal-Bielecka">Otylia Kowal-Bielecka</name></noRegion></country><country name="Italie"><noRegion><name sortKey="Matucci Cerinic, Marco" sort="Matucci Cerinic, Marco" uniqKey="Matucci Cerinic M" first="Marco" last="Matucci-Cerinic">Marco Matucci-Cerinic</name></noRegion></country><country name="Australie"><noRegion><name sortKey="Nash, Peter" sort="Nash, Peter" uniqKey="Nash P" first="Peter" last="Nash">Peter Nash</name></noRegion></country><country name="Suisse"><noRegion><name sortKey="Distler, Oliver" sort="Distler, Oliver" uniqKey="Distler O" first="Oliver" last="Distler">Oliver Distler</name></noRegion></country></tree></affiliations></record>Pour manipuler ce document sous Unix (Dilib)
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