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Autologous hematopoietic stem cell transplantation vs intravenous pulse cyclophosphamide in diffuse cutaneous systemic sclerosis: a randomized clinical trial.

Identifieur interne : 000358 ( PubMed/Corpus ); précédent : 000357; suivant : 000359

Autologous hematopoietic stem cell transplantation vs intravenous pulse cyclophosphamide in diffuse cutaneous systemic sclerosis: a randomized clinical trial.

Auteurs : Jacob M. Van Laar ; Dominique Farge ; Jacob K. Sont ; Kamran Naraghi ; Zora Marjanovic ; Jérôme Larghero ; Annemie J. Schuerwegh ; Erik W A. Marijt ; Madelon C. Vonk ; Anton V. Schattenberg ; Marco Matucci-Cerinic ; Alexandre E. Voskuyl ; Arjan A. Van De Loosdrecht ; Thomas Daikeler ; Ina Kötter ; Marc Schmalzing ; Thierry Martin ; Bruno Lioure ; Stefan M. Weiner ; Alexander Kreuter ; Christophe Deligny ; Jean-Marc Durand ; Paul Emery ; Klaus P. Machold ; Francoise Sarrot-Reynauld ; Klaus Warnatz ; Daniel F P. Adoue ; Joël Constans ; Hans-Peter Tony ; Nicoletta Del Papa ; Athanasios Fassas ; Andrea Himsel ; David Launay ; Andrea Lo Monaco ; Pierre Philippe ; Isabelle Quéré ; Éric Rich ; Rene Westhovens ; Bridget Griffiths ; Riccardo Saccardi ; Frank H. Van Den Hoogen ; Willem E. Fibbe ; Gérard Socié ; Alois Gratwohl ; Alan Tyndall

Source :

RBID : pubmed:25058083

English descriptors

Abstract

High-dose immunosuppressive therapy and autologous hematopoietic stem cell transplantation (HSCT) have shown efficacy in systemic sclerosis in phase 1 and small phase 2 trials.

DOI: 10.1001/jama.2014.6368
PubMed: 25058083

Links to Exploration step

pubmed:25058083

Le document en format XML

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<name sortKey="Matucci Cerinic, Marco" sort="Matucci Cerinic, Marco" uniqKey="Matucci Cerinic M" first="Marco" last="Matucci-Cerinic">Marco Matucci-Cerinic</name>
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<name sortKey="Van De Loosdrecht, Arjan A" sort="Van De Loosdrecht, Arjan A" uniqKey="Van De Loosdrecht A" first="Arjan A" last="Van De Loosdrecht">Arjan A. Van De Loosdrecht</name>
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<name sortKey="Daikeler, Thomas" sort="Daikeler, Thomas" uniqKey="Daikeler T" first="Thomas" last="Daikeler">Thomas Daikeler</name>
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<name sortKey="Kotter, Ina" sort="Kotter, Ina" uniqKey="Kotter I" first="Ina" last="Kötter">Ina Kötter</name>
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<name sortKey="Martin, Thierry" sort="Martin, Thierry" uniqKey="Martin T" first="Thierry" last="Martin">Thierry Martin</name>
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<name sortKey="Weiner, Stefan M" sort="Weiner, Stefan M" uniqKey="Weiner S" first="Stefan M" last="Weiner">Stefan M. Weiner</name>
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<nlm:affiliation>2.Medizinische Abteilung Krankenhaus der Barmherzigen Brüder Trier, Trier, Germany.</nlm:affiliation>
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<nlm:affiliation>Department of Dermatology, Venereology, and Allergology, HELIOS St. Elisabeth Hospital Oberhausen, Oberhausen,Germany.</nlm:affiliation>
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<author>
<name sortKey="Deligny, Christophe" sort="Deligny, Christophe" uniqKey="Deligny C" first="Christophe" last="Deligny">Christophe Deligny</name>
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<nlm:affiliation>Service Médecine Interne, Hôpital Pierre Zobda Quitman, Fort-de France, Martinique.</nlm:affiliation>
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<name sortKey="Durand, Jean Marc" sort="Durand, Jean Marc" uniqKey="Durand J" first="Jean-Marc" last="Durand">Jean-Marc Durand</name>
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<nlm:affiliation>Department of Internal Medicine CHU La Conception, Assistance Publique-Hôpitaux de Marseille, Marseille, France.</nlm:affiliation>
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<name sortKey="Emery, Paul" sort="Emery, Paul" uniqKey="Emery P" first="Paul" last="Emery">Paul Emery</name>
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<nlm:affiliation>Leeds Institute of Rheumatic and Musculoskeletal Medicine, University of Leeds, and NIHR Leeds Musculoskeletal Biomedical Research Unit, Leeds Teaching Hospitals NHS Trust, Leeds, United Kingdom.</nlm:affiliation>
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<name sortKey="Machold, Klaus P" sort="Machold, Klaus P" uniqKey="Machold K" first="Klaus P" last="Machold">Klaus P. Machold</name>
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<nlm:affiliation>Klinische Abteilung für Rheumatologie, Medizinische Universität, Vienna, Austria.</nlm:affiliation>
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<name sortKey="Sarrot Reynauld, Francoise" sort="Sarrot Reynauld, Francoise" uniqKey="Sarrot Reynauld F" first="Francoise" last="Sarrot-Reynauld">Francoise Sarrot-Reynauld</name>
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<nlm:affiliation>Pôle Pluridisciplinaire de Médecine, Centre Hospitalier Universitaire de Grenoble, Grenoble, France.</nlm:affiliation>
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<name sortKey="Warnatz, Klaus" sort="Warnatz, Klaus" uniqKey="Warnatz K" first="Klaus" last="Warnatz">Klaus Warnatz</name>
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<nlm:affiliation>Division of Rheumatology and Clinical Immunology, University Medical Center Freiburg, Freiburg, Germany.</nlm:affiliation>
</affiliation>
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<name sortKey="Adoue, Daniel F P" sort="Adoue, Daniel F P" uniqKey="Adoue D" first="Daniel F P" last="Adoue">Daniel F P. Adoue</name>
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<nlm:affiliation>Service Médecine Interne, Centre Hospitalier Universitaire Toulouse.</nlm:affiliation>
</affiliation>
</author>
<author>
<name sortKey="Constans, Joel" sort="Constans, Joel" uniqKey="Constans J" first="Joël" last="Constans">Joël Constans</name>
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<nlm:affiliation>Service Médecine Interne et Médecine Vasculaire, Hôpital St-André, Centre Hospitalier Universitaire de Bordeaux, Bordeaux, France.</nlm:affiliation>
</affiliation>
</author>
<author>
<name sortKey="Tony, Hans Peter" sort="Tony, Hans Peter" uniqKey="Tony H" first="Hans-Peter" last="Tony">Hans-Peter Tony</name>
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<nlm:affiliation>Department of Rheumatology and Clinical Immunology, University of Würzburg Medical Center, Würzburg, Germany.</nlm:affiliation>
</affiliation>
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<name sortKey="Del Papa, Nicoletta" sort="Del Papa, Nicoletta" uniqKey="Del Papa N" first="Nicoletta" last="Del Papa">Nicoletta Del Papa</name>
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<nlm:affiliation>Day Hospital Reumatologia, Ospedale G. Pini, Milan, Italy.</nlm:affiliation>
</affiliation>
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<author>
<name sortKey="Fassas, Athanasios" sort="Fassas, Athanasios" uniqKey="Fassas A" first="Athanasios" last="Fassas">Athanasios Fassas</name>
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<nlm:affiliation>Department of Hematology, Cell and Gene Therapy Center, George Papanicolaou Hospital, Thessaloniki, Greece.</nlm:affiliation>
</affiliation>
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<name sortKey="Himsel, Andrea" sort="Himsel, Andrea" uniqKey="Himsel A" first="Andrea" last="Himsel">Andrea Himsel</name>
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<nlm:affiliation>Department of Rheumatology, University Hospital Frankfurt, Frankfurt, Germany.</nlm:affiliation>
</affiliation>
</author>
<author>
<name sortKey="Launay, David" sort="Launay, David" uniqKey="Launay D" first="David" last="Launay">David Launay</name>
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<nlm:affiliation>Service de Médecine Interne, Hôpital Claude-Huriez, Lille, France.</nlm:affiliation>
</affiliation>
</author>
<author>
<name sortKey="Lo Monaco, Andrea" sort="Lo Monaco, Andrea" uniqKey="Lo Monaco A" first="Andrea" last="Lo Monaco">Andrea Lo Monaco</name>
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<nlm:affiliation>Section and Unit of Rheumatology, Department of Medical Sciences, University of Ferrara, Italy.</nlm:affiliation>
</affiliation>
</author>
<author>
<name sortKey="Philippe, Pierre" sort="Philippe, Pierre" uniqKey="Philippe P" first="Pierre" last="Philippe">Pierre Philippe</name>
<affiliation>
<nlm:affiliation>Service de Médecine Interne, Centre Hospitalier Universitaire Estaing, Clermont-Ferrand, France.</nlm:affiliation>
</affiliation>
</author>
<author>
<name sortKey="Quere, Isabelle" sort="Quere, Isabelle" uniqKey="Quere I" first="Isabelle" last="Quéré">Isabelle Quéré</name>
<affiliation>
<nlm:affiliation>Department of Internal Medicine, Montpellier University Hospital, Montpellier, France.</nlm:affiliation>
</affiliation>
</author>
<author>
<name sortKey="Rich, Eric" sort="Rich, Eric" uniqKey="Rich E" first="Éric" last="Rich">Éric Rich</name>
<affiliation>
<nlm:affiliation>Centre Hospitalier de l'Université de Montréal, Montréal, Québec, Canada.</nlm:affiliation>
</affiliation>
</author>
<author>
<name sortKey="Westhovens, Rene" sort="Westhovens, Rene" uniqKey="Westhovens R" first="Rene" last="Westhovens">Rene Westhovens</name>
<affiliation>
<nlm:affiliation>Skeletal Biology and Engineering Research Center, Department of Development and Regeneration KU Leuven, Rheumatology, University Hospitals, Leuven, Belgium.</nlm:affiliation>
</affiliation>
</author>
<author>
<name sortKey="Griffiths, Bridget" sort="Griffiths, Bridget" uniqKey="Griffiths B" first="Bridget" last="Griffiths">Bridget Griffiths</name>
<affiliation>
<nlm:affiliation>Department of Rheumatology, Freeman Hospital, Newcastle upon Tyne, United Kingdom.</nlm:affiliation>
</affiliation>
</author>
<author>
<name sortKey="Saccardi, Riccardo" sort="Saccardi, Riccardo" uniqKey="Saccardi R" first="Riccardo" last="Saccardi">Riccardo Saccardi</name>
<affiliation>
<nlm:affiliation>Department of Hematology, Careggi University Hospital, Florence, Italy.</nlm:affiliation>
</affiliation>
</author>
<author>
<name sortKey="Van Den Hoogen, Frank H" sort="Van Den Hoogen, Frank H" uniqKey="Van Den Hoogen F" first="Frank H" last="Van Den Hoogen">Frank H. Van Den Hoogen</name>
<affiliation>
<nlm:affiliation>Department of Rheumatology, Maartenskliniek, Nijmegen, the Netherlands.</nlm:affiliation>
</affiliation>
</author>
<author>
<name sortKey="Fibbe, Willem E" sort="Fibbe, Willem E" uniqKey="Fibbe W" first="Willem E" last="Fibbe">Willem E. Fibbe</name>
<affiliation>
<nlm:affiliation>Department of Immunohematology and Blood Transfusion, Leiden University Medical Center, Leiden, the Netherlands.</nlm:affiliation>
</affiliation>
</author>
<author>
<name sortKey="Socie, Gerard" sort="Socie, Gerard" uniqKey="Socie G" first="Gérard" last="Socié">Gérard Socié</name>
<affiliation>
<nlm:affiliation>Hematology/Transplantation, AP-HP Hôpital Saint-Louis, Paris 7 University, France.</nlm:affiliation>
</affiliation>
</author>
<author>
<name sortKey="Gratwohl, Alois" sort="Gratwohl, Alois" uniqKey="Gratwohl A" first="Alois" last="Gratwohl">Alois Gratwohl</name>
<affiliation>
<nlm:affiliation>Department of Hematology, University Hospital Basel, Basel, Switzerland.</nlm:affiliation>
</affiliation>
</author>
<author>
<name sortKey="Tyndall, Alan" sort="Tyndall, Alan" uniqKey="Tyndall A" first="Alan" last="Tyndall">Alan Tyndall</name>
<affiliation>
<nlm:affiliation>Department of Rheumatology, University Hospital Basel, Basel, Switzerland.</nlm:affiliation>
</affiliation>
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<title xml:lang="en">Autologous hematopoietic stem cell transplantation vs intravenous pulse cyclophosphamide in diffuse cutaneous systemic sclerosis: a randomized clinical trial.</title>
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<nlm:affiliation>Internal Medicine and Vascular Disease Unit, AP-HP Hôpital Saint-Louis, Paris 7 University, France.</nlm:affiliation>
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<name sortKey="Sont, Jacob K" sort="Sont, Jacob K" uniqKey="Sont J" first="Jacob K" last="Sont">Jacob K. Sont</name>
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<nlm:affiliation>Department of Medical Decision Making, Leiden University Medical Center, Leiden, the Netherlands.</nlm:affiliation>
</affiliation>
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<name sortKey="Naraghi, Kamran" sort="Naraghi, Kamran" uniqKey="Naraghi K" first="Kamran" last="Naraghi">Kamran Naraghi</name>
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<nlm:affiliation>Department of Rheumatology, The James Cook University Hospital, Middlesbrough, United Kingdom.</nlm:affiliation>
</affiliation>
</author>
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<name sortKey="Marjanovic, Zora" sort="Marjanovic, Zora" uniqKey="Marjanovic Z" first="Zora" last="Marjanovic">Zora Marjanovic</name>
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<nlm:affiliation>Service d'Hématologie Clinique et de Thérapie Cellulaire, AP-HP Hôpital Saint-Antoine, Paris 6 University, Paris, France.</nlm:affiliation>
</affiliation>
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<name sortKey="Larghero, Jerome" sort="Larghero, Jerome" uniqKey="Larghero J" first="Jérôme" last="Larghero">Jérôme Larghero</name>
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<nlm:affiliation>Clinical Investigation Center in Biotherapies and Cell Therapy Unit, AP-HP Hôpital Saint-Louis, Paris 7 University, France.</nlm:affiliation>
</affiliation>
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<name sortKey="Schuerwegh, Annemie J" sort="Schuerwegh, Annemie J" uniqKey="Schuerwegh A" first="Annemie J" last="Schuerwegh">Annemie J. Schuerwegh</name>
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<nlm:affiliation>Department of Rheumatology, Leiden University Medical Center, Leiden, the Netherlands.</nlm:affiliation>
</affiliation>
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<name sortKey="Marijt, Erik W A" sort="Marijt, Erik W A" uniqKey="Marijt E" first="Erik W A" last="Marijt">Erik W A. Marijt</name>
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<nlm:affiliation>Department of Hematology, Leiden University Medical Center, Leiden, the Netherlands.</nlm:affiliation>
</affiliation>
</author>
<author>
<name sortKey="Vonk, Madelon C" sort="Vonk, Madelon C" uniqKey="Vonk M" first="Madelon C" last="Vonk">Madelon C. Vonk</name>
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<nlm:affiliation>Department of Rheumatology, Radboud University Nijmegen Medical Center, Nijmegen, the Netherlands.</nlm:affiliation>
</affiliation>
</author>
<author>
<name sortKey="Schattenberg, Anton V" sort="Schattenberg, Anton V" uniqKey="Schattenberg A" first="Anton V" last="Schattenberg">Anton V. Schattenberg</name>
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<nlm:affiliation>Department of Hematology, Radboud University Nijmegen Medical Center, Nijmegen, the Netherlands.</nlm:affiliation>
</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>
<nlm:affiliation>Department of Biomedicine, Division of Rheumatology AOUC and Department of Experimental and Clinical Medicine, University of Florence, Florence.</nlm:affiliation>
</affiliation>
</author>
<author>
<name sortKey="Voskuyl, Alexandre E" sort="Voskuyl, Alexandre E" uniqKey="Voskuyl A" first="Alexandre E" last="Voskuyl">Alexandre E. Voskuyl</name>
<affiliation>
<nlm:affiliation>Department of Rheumatology, VU University Medical Center, Amsterdam, the Netherlands.</nlm:affiliation>
</affiliation>
</author>
<author>
<name sortKey="Van De Loosdrecht, Arjan A" sort="Van De Loosdrecht, Arjan A" uniqKey="Van De Loosdrecht A" first="Arjan A" last="Van De Loosdrecht">Arjan A. Van De Loosdrecht</name>
<affiliation>
<nlm:affiliation>Department of Hematology, VU University Medical Center, Amsterdam, the Netherlands.</nlm:affiliation>
</affiliation>
</author>
<author>
<name sortKey="Daikeler, Thomas" sort="Daikeler, Thomas" uniqKey="Daikeler T" first="Thomas" last="Daikeler">Thomas Daikeler</name>
<affiliation>
<nlm:affiliation>Department of Rheumatology, University Hospital Basel, Basel, Switzerland.</nlm:affiliation>
</affiliation>
</author>
<author>
<name sortKey="Kotter, Ina" sort="Kotter, Ina" uniqKey="Kotter I" first="Ina" last="Kötter">Ina Kötter</name>
<affiliation>
<nlm:affiliation>Department of Internal Medicine II, University Hospital, Tübingen, Germany.</nlm:affiliation>
</affiliation>
</author>
<author>
<name sortKey="Schmalzing, Marc" sort="Schmalzing, Marc" uniqKey="Schmalzing M" first="Marc" last="Schmalzing">Marc Schmalzing</name>
<affiliation>
<nlm:affiliation>Department of Internal Medicine II, University Hospital, Tübingen, Germany.</nlm:affiliation>
</affiliation>
</author>
<author>
<name sortKey="Martin, Thierry" sort="Martin, Thierry" uniqKey="Martin T" first="Thierry" last="Martin">Thierry Martin</name>
<affiliation>
<nlm:affiliation>Department of Clinical Immunology, Strasbourg University Hospital, Strasbourg, France.</nlm:affiliation>
</affiliation>
</author>
<author>
<name sortKey="Lioure, Bruno" sort="Lioure, Bruno" uniqKey="Lioure B" first="Bruno" last="Lioure">Bruno Lioure</name>
<affiliation>
<nlm:affiliation>Service d'Hématologie et d'Oncologie, Unité de Greffe de Cellules Souches Hématopoïétiques, Centre Hospitalier Universitaire Hautepierre, Strasbourg, France.</nlm:affiliation>
</affiliation>
</author>
<author>
<name sortKey="Weiner, Stefan M" sort="Weiner, Stefan M" uniqKey="Weiner S" first="Stefan M" last="Weiner">Stefan M. Weiner</name>
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<nlm:affiliation>2.Medizinische Abteilung Krankenhaus der Barmherzigen Brüder Trier, Trier, Germany.</nlm:affiliation>
</affiliation>
</author>
<author>
<name sortKey="Kreuter, Alexander" sort="Kreuter, Alexander" uniqKey="Kreuter A" first="Alexander" last="Kreuter">Alexander Kreuter</name>
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<nlm:affiliation>Department of Dermatology, Venereology, and Allergology, HELIOS St. Elisabeth Hospital Oberhausen, Oberhausen,Germany.</nlm:affiliation>
</affiliation>
</author>
<author>
<name sortKey="Deligny, Christophe" sort="Deligny, Christophe" uniqKey="Deligny C" first="Christophe" last="Deligny">Christophe Deligny</name>
<affiliation>
<nlm:affiliation>Service Médecine Interne, Hôpital Pierre Zobda Quitman, Fort-de France, Martinique.</nlm:affiliation>
</affiliation>
</author>
<author>
<name sortKey="Durand, Jean Marc" sort="Durand, Jean Marc" uniqKey="Durand J" first="Jean-Marc" last="Durand">Jean-Marc Durand</name>
<affiliation>
<nlm:affiliation>Department of Internal Medicine CHU La Conception, Assistance Publique-Hôpitaux de Marseille, Marseille, France.</nlm:affiliation>
</affiliation>
</author>
<author>
<name sortKey="Emery, Paul" sort="Emery, Paul" uniqKey="Emery P" first="Paul" last="Emery">Paul Emery</name>
<affiliation>
<nlm:affiliation>Leeds Institute of Rheumatic and Musculoskeletal Medicine, University of Leeds, and NIHR Leeds Musculoskeletal Biomedical Research Unit, Leeds Teaching Hospitals NHS Trust, Leeds, United Kingdom.</nlm:affiliation>
</affiliation>
</author>
<author>
<name sortKey="Machold, Klaus P" sort="Machold, Klaus P" uniqKey="Machold K" first="Klaus P" last="Machold">Klaus P. Machold</name>
<affiliation>
<nlm:affiliation>Klinische Abteilung für Rheumatologie, Medizinische Universität, Vienna, Austria.</nlm:affiliation>
</affiliation>
</author>
<author>
<name sortKey="Sarrot Reynauld, Francoise" sort="Sarrot Reynauld, Francoise" uniqKey="Sarrot Reynauld F" first="Francoise" last="Sarrot-Reynauld">Francoise Sarrot-Reynauld</name>
<affiliation>
<nlm:affiliation>Pôle Pluridisciplinaire de Médecine, Centre Hospitalier Universitaire de Grenoble, Grenoble, France.</nlm:affiliation>
</affiliation>
</author>
<author>
<name sortKey="Warnatz, Klaus" sort="Warnatz, Klaus" uniqKey="Warnatz K" first="Klaus" last="Warnatz">Klaus Warnatz</name>
<affiliation>
<nlm:affiliation>Division of Rheumatology and Clinical Immunology, University Medical Center Freiburg, Freiburg, Germany.</nlm:affiliation>
</affiliation>
</author>
<author>
<name sortKey="Adoue, Daniel F P" sort="Adoue, Daniel F P" uniqKey="Adoue D" first="Daniel F P" last="Adoue">Daniel F P. Adoue</name>
<affiliation>
<nlm:affiliation>Service Médecine Interne, Centre Hospitalier Universitaire Toulouse.</nlm:affiliation>
</affiliation>
</author>
<author>
<name sortKey="Constans, Joel" sort="Constans, Joel" uniqKey="Constans J" first="Joël" last="Constans">Joël Constans</name>
<affiliation>
<nlm:affiliation>Service Médecine Interne et Médecine Vasculaire, Hôpital St-André, Centre Hospitalier Universitaire de Bordeaux, Bordeaux, France.</nlm:affiliation>
</affiliation>
</author>
<author>
<name sortKey="Tony, Hans Peter" sort="Tony, Hans Peter" uniqKey="Tony H" first="Hans-Peter" last="Tony">Hans-Peter Tony</name>
<affiliation>
<nlm:affiliation>Department of Rheumatology and Clinical Immunology, University of Würzburg Medical Center, Würzburg, Germany.</nlm:affiliation>
</affiliation>
</author>
<author>
<name sortKey="Del Papa, Nicoletta" sort="Del Papa, Nicoletta" uniqKey="Del Papa N" first="Nicoletta" last="Del Papa">Nicoletta Del Papa</name>
<affiliation>
<nlm:affiliation>Day Hospital Reumatologia, Ospedale G. Pini, Milan, Italy.</nlm:affiliation>
</affiliation>
</author>
<author>
<name sortKey="Fassas, Athanasios" sort="Fassas, Athanasios" uniqKey="Fassas A" first="Athanasios" last="Fassas">Athanasios Fassas</name>
<affiliation>
<nlm:affiliation>Department of Hematology, Cell and Gene Therapy Center, George Papanicolaou Hospital, Thessaloniki, Greece.</nlm:affiliation>
</affiliation>
</author>
<author>
<name sortKey="Himsel, Andrea" sort="Himsel, Andrea" uniqKey="Himsel A" first="Andrea" last="Himsel">Andrea Himsel</name>
<affiliation>
<nlm:affiliation>Department of Rheumatology, University Hospital Frankfurt, Frankfurt, Germany.</nlm:affiliation>
</affiliation>
</author>
<author>
<name sortKey="Launay, David" sort="Launay, David" uniqKey="Launay D" first="David" last="Launay">David Launay</name>
<affiliation>
<nlm:affiliation>Service de Médecine Interne, Hôpital Claude-Huriez, Lille, France.</nlm:affiliation>
</affiliation>
</author>
<author>
<name sortKey="Lo Monaco, Andrea" sort="Lo Monaco, Andrea" uniqKey="Lo Monaco A" first="Andrea" last="Lo Monaco">Andrea Lo Monaco</name>
<affiliation>
<nlm:affiliation>Section and Unit of Rheumatology, Department of Medical Sciences, University of Ferrara, Italy.</nlm:affiliation>
</affiliation>
</author>
<author>
<name sortKey="Philippe, Pierre" sort="Philippe, Pierre" uniqKey="Philippe P" first="Pierre" last="Philippe">Pierre Philippe</name>
<affiliation>
<nlm:affiliation>Service de Médecine Interne, Centre Hospitalier Universitaire Estaing, Clermont-Ferrand, France.</nlm:affiliation>
</affiliation>
</author>
<author>
<name sortKey="Quere, Isabelle" sort="Quere, Isabelle" uniqKey="Quere I" first="Isabelle" last="Quéré">Isabelle Quéré</name>
<affiliation>
<nlm:affiliation>Department of Internal Medicine, Montpellier University Hospital, Montpellier, France.</nlm:affiliation>
</affiliation>
</author>
<author>
<name sortKey="Rich, Eric" sort="Rich, Eric" uniqKey="Rich E" first="Éric" last="Rich">Éric Rich</name>
<affiliation>
<nlm:affiliation>Centre Hospitalier de l'Université de Montréal, Montréal, Québec, Canada.</nlm:affiliation>
</affiliation>
</author>
<author>
<name sortKey="Westhovens, Rene" sort="Westhovens, Rene" uniqKey="Westhovens R" first="Rene" last="Westhovens">Rene Westhovens</name>
<affiliation>
<nlm:affiliation>Skeletal Biology and Engineering Research Center, Department of Development and Regeneration KU Leuven, Rheumatology, University Hospitals, Leuven, Belgium.</nlm:affiliation>
</affiliation>
</author>
<author>
<name sortKey="Griffiths, Bridget" sort="Griffiths, Bridget" uniqKey="Griffiths B" first="Bridget" last="Griffiths">Bridget Griffiths</name>
<affiliation>
<nlm:affiliation>Department of Rheumatology, Freeman Hospital, Newcastle upon Tyne, United Kingdom.</nlm:affiliation>
</affiliation>
</author>
<author>
<name sortKey="Saccardi, Riccardo" sort="Saccardi, Riccardo" uniqKey="Saccardi R" first="Riccardo" last="Saccardi">Riccardo Saccardi</name>
<affiliation>
<nlm:affiliation>Department of Hematology, Careggi University Hospital, Florence, Italy.</nlm:affiliation>
</affiliation>
</author>
<author>
<name sortKey="Van Den Hoogen, Frank H" sort="Van Den Hoogen, Frank H" uniqKey="Van Den Hoogen F" first="Frank H" last="Van Den Hoogen">Frank H. Van Den Hoogen</name>
<affiliation>
<nlm:affiliation>Department of Rheumatology, Maartenskliniek, Nijmegen, the Netherlands.</nlm:affiliation>
</affiliation>
</author>
<author>
<name sortKey="Fibbe, Willem E" sort="Fibbe, Willem E" uniqKey="Fibbe W" first="Willem E" last="Fibbe">Willem E. Fibbe</name>
<affiliation>
<nlm:affiliation>Department of Immunohematology and Blood Transfusion, Leiden University Medical Center, Leiden, the Netherlands.</nlm:affiliation>
</affiliation>
</author>
<author>
<name sortKey="Socie, Gerard" sort="Socie, Gerard" uniqKey="Socie G" first="Gérard" last="Socié">Gérard Socié</name>
<affiliation>
<nlm:affiliation>Hematology/Transplantation, AP-HP Hôpital Saint-Louis, Paris 7 University, France.</nlm:affiliation>
</affiliation>
</author>
<author>
<name sortKey="Gratwohl, Alois" sort="Gratwohl, Alois" uniqKey="Gratwohl A" first="Alois" last="Gratwohl">Alois Gratwohl</name>
<affiliation>
<nlm:affiliation>Department of Hematology, University Hospital Basel, Basel, Switzerland.</nlm:affiliation>
</affiliation>
</author>
<author>
<name sortKey="Tyndall, Alan" sort="Tyndall, Alan" uniqKey="Tyndall A" first="Alan" last="Tyndall">Alan Tyndall</name>
<affiliation>
<nlm:affiliation>Department of Rheumatology, University Hospital Basel, Basel, Switzerland.</nlm:affiliation>
</affiliation>
</author>
</analytic>
<series>
<title level="j">JAMA</title>
<idno type="eISSN">1538-3598</idno>
<imprint>
<date when="2014" type="published">2014</date>
</imprint>
</series>
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<keywords scheme="KwdEn" xml:lang="en">
<term>Adult</term>
<term>Autografts</term>
<term>Cyclophosphamide (administration & dosage)</term>
<term>Cyclophosphamide (adverse effects)</term>
<term>Female</term>
<term>Hematopoietic Stem Cell Transplantation (adverse effects)</term>
<term>Humans</term>
<term>Immunosuppressive Agents (administration & dosage)</term>
<term>Immunosuppressive Agents (adverse effects)</term>
<term>Male</term>
<term>Middle Aged</term>
<term>Scleroderma, Diffuse (drug therapy)</term>
<term>Survival Analysis</term>
</keywords>
<keywords scheme="MESH" type="chemical" qualifier="administration & dosage" xml:lang="en">
<term>Cyclophosphamide</term>
<term>Immunosuppressive Agents</term>
</keywords>
<keywords scheme="MESH" type="chemical" qualifier="adverse effects" xml:lang="en">
<term>Cyclophosphamide</term>
<term>Immunosuppressive Agents</term>
</keywords>
<keywords scheme="MESH" qualifier="adverse effects" xml:lang="en">
<term>Hematopoietic Stem Cell Transplantation</term>
</keywords>
<keywords scheme="MESH" qualifier="drug therapy" xml:lang="en">
<term>Scleroderma, Diffuse</term>
</keywords>
<keywords scheme="MESH" xml:lang="en">
<term>Adult</term>
<term>Autografts</term>
<term>Female</term>
<term>Humans</term>
<term>Male</term>
<term>Middle Aged</term>
<term>Survival Analysis</term>
</keywords>
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<front>
<div type="abstract" xml:lang="en">High-dose immunosuppressive therapy and autologous hematopoietic stem cell transplantation (HSCT) have shown efficacy in systemic sclerosis in phase 1 and small phase 2 trials.</div>
</front>
</TEI>
<pubmed>
<MedlineCitation Status="MEDLINE" Owner="NLM">
<PMID Version="1">25058083</PMID>
<DateCreated>
<Year>2014</Year>
<Month>07</Month>
<Day>25</Day>
</DateCreated>
<DateCompleted>
<Year>2014</Year>
<Month>08</Month>
<Day>11</Day>
</DateCompleted>
<DateRevised>
<Year>2016</Year>
<Month>11</Month>
<Day>22</Day>
</DateRevised>
<Article PubModel="Print">
<Journal>
<ISSN IssnType="Electronic">1538-3598</ISSN>
<JournalIssue CitedMedium="Internet">
<Volume>311</Volume>
<Issue>24</Issue>
<PubDate>
<Year>2014</Year>
<Month>Jun</Month>
<Day>25</Day>
</PubDate>
</JournalIssue>
<Title>JAMA</Title>
<ISOAbbreviation>JAMA</ISOAbbreviation>
</Journal>
<ArticleTitle>Autologous hematopoietic stem cell transplantation vs intravenous pulse cyclophosphamide in diffuse cutaneous systemic sclerosis: a randomized clinical trial.</ArticleTitle>
<Pagination>
<MedlinePgn>2490-8</MedlinePgn>
</Pagination>
<ELocationID EIdType="doi" ValidYN="Y">10.1001/jama.2014.6368</ELocationID>
<Abstract>
<AbstractText Label="IMPORTANCE" NlmCategory="OBJECTIVE">High-dose immunosuppressive therapy and autologous hematopoietic stem cell transplantation (HSCT) have shown efficacy in systemic sclerosis in phase 1 and small phase 2 trials.</AbstractText>
<AbstractText Label="OBJECTIVE" NlmCategory="OBJECTIVE">To compare efficacy and safety of HSCT vs 12 successive monthly intravenous pulses of cyclophosphamide.</AbstractText>
<AbstractText Label="DESIGN, SETTING, AND PARTICIPANTS" NlmCategory="METHODS">The Autologous Stem Cell Transplantation International Scleroderma (ASTIS) trial, a phase 3, multicenter, randomized (1:1), open-label, parallel-group, clinical trial conducted in 10 countries at 29 centers with access to a European Group for Blood and Marrow Transplantation-registered transplant facility. From March 2001 to October 2009, 156 patients with early diffuse cutaneous systemic sclerosis were recruited and followed up until October 31, 2013.</AbstractText>
<AbstractText Label="INTERVENTIONS" NlmCategory="METHODS">HSCT vs intravenous pulse cyclophosphamide.</AbstractText>
<AbstractText Label="MAIN OUTCOMES AND MEASURES" NlmCategory="METHODS">The primary end point was event-free survival, defined as time from randomization until the occurrence of death or persistent major organ failure.</AbstractText>
<AbstractText Label="RESULTS" NlmCategory="RESULTS">A total of 156 patients were randomly assigned to receive HSCT (n = 79) or cyclophosphamide (n = 77). During a median follow-up of 5.8 years, 53 events occurred: 22 in the HSCT group (19 deaths and 3 irreversible organ failures) and 31 in the control group (23 deaths and 8 irreversible organ failures). During the first year, there were more events in the HSCT group (13 events [16.5%], including 8 treatment-related deaths) than in the control group (8 events [10.4%], with no treatment-related deaths). At 2 years, 14 events (17.7%) had occurred cumulatively in the HSCT group vs 14 events (18.2%) in the control group; at 4 years, 15 events (19%) had occurred cumulatively in the HSCT group vs 20 events (26%) in the control group. Time-varying hazard ratios (modeled with treatment × time interaction) for event-free survival were 0.35 (95% CI, 0.16-0.74) at 2 years and 0.34 (95% CI, 0.16-0.74) at 4 years.</AbstractText>
<AbstractText Label="CONCLUSIONS AND RELEVANCE" NlmCategory="CONCLUSIONS">Among patients with early diffuse cutaneous systemic sclerosis, HSCT was associated with increased treatment-related mortality in the first year after treatment. However, HCST conferred a significant long-term event-free survival benefit.</AbstractText>
<AbstractText Label="TRIAL REGISTRATION" NlmCategory="BACKGROUND">isrctn.org Identifier: ISRCTN54371254.</AbstractText>
</Abstract>
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<Author ValidYN="Y">
<LastName>van Laar</LastName>
<ForeName>Jacob M</ForeName>
<Initials>JM</Initials>
<AffiliationInfo>
<Affiliation>Institute of Cellular Medicine, Newcastle University, Newcastle upon Tyne, United Kingdom.</Affiliation>
</AffiliationInfo>
</Author>
<Author ValidYN="Y">
<LastName>Farge</LastName>
<ForeName>Dominique</ForeName>
<Initials>D</Initials>
<AffiliationInfo>
<Affiliation>Internal Medicine and Vascular Disease Unit, AP-HP Hôpital Saint-Louis, Paris 7 University, France.</Affiliation>
</AffiliationInfo>
</Author>
<Author ValidYN="Y">
<LastName>Sont</LastName>
<ForeName>Jacob K</ForeName>
<Initials>JK</Initials>
<AffiliationInfo>
<Affiliation>Department of Medical Decision Making, Leiden University Medical Center, Leiden, the Netherlands.</Affiliation>
</AffiliationInfo>
</Author>
<Author ValidYN="Y">
<LastName>Naraghi</LastName>
<ForeName>Kamran</ForeName>
<Initials>K</Initials>
<AffiliationInfo>
<Affiliation>Department of Rheumatology, The James Cook University Hospital, Middlesbrough, United Kingdom.</Affiliation>
</AffiliationInfo>
</Author>
<Author ValidYN="Y">
<LastName>Marjanovic</LastName>
<ForeName>Zora</ForeName>
<Initials>Z</Initials>
<AffiliationInfo>
<Affiliation>Service d'Hématologie Clinique et de Thérapie Cellulaire, AP-HP Hôpital Saint-Antoine, Paris 6 University, Paris, France.</Affiliation>
</AffiliationInfo>
</Author>
<Author ValidYN="Y">
<LastName>Larghero</LastName>
<ForeName>Jérôme</ForeName>
<Initials>J</Initials>
<AffiliationInfo>
<Affiliation>Clinical Investigation Center in Biotherapies and Cell Therapy Unit, AP-HP Hôpital Saint-Louis, Paris 7 University, France.</Affiliation>
</AffiliationInfo>
</Author>
<Author ValidYN="Y">
<LastName>Schuerwegh</LastName>
<ForeName>Annemie J</ForeName>
<Initials>AJ</Initials>
<AffiliationInfo>
<Affiliation>Department of Rheumatology, Leiden University Medical Center, Leiden, the Netherlands.</Affiliation>
</AffiliationInfo>
</Author>
<Author ValidYN="Y">
<LastName>Marijt</LastName>
<ForeName>Erik W A</ForeName>
<Initials>EW</Initials>
<AffiliationInfo>
<Affiliation>Department of Hematology, Leiden University Medical Center, Leiden, the Netherlands.</Affiliation>
</AffiliationInfo>
</Author>
<Author ValidYN="Y">
<LastName>Vonk</LastName>
<ForeName>Madelon C</ForeName>
<Initials>MC</Initials>
<AffiliationInfo>
<Affiliation>Department of Rheumatology, Radboud University Nijmegen Medical Center, Nijmegen, the Netherlands.</Affiliation>
</AffiliationInfo>
</Author>
<Author ValidYN="Y">
<LastName>Schattenberg</LastName>
<ForeName>Anton V</ForeName>
<Initials>AV</Initials>
<AffiliationInfo>
<Affiliation>Department of Hematology, Radboud University Nijmegen Medical Center, Nijmegen, the Netherlands.</Affiliation>
</AffiliationInfo>
</Author>
<Author ValidYN="Y">
<LastName>Matucci-Cerinic</LastName>
<ForeName>Marco</ForeName>
<Initials>M</Initials>
<AffiliationInfo>
<Affiliation>Department of Biomedicine, Division of Rheumatology AOUC and Department of Experimental and Clinical Medicine, University of Florence, Florence.</Affiliation>
</AffiliationInfo>
</Author>
<Author ValidYN="Y">
<LastName>Voskuyl</LastName>
<ForeName>Alexandre E</ForeName>
<Initials>AE</Initials>
<AffiliationInfo>
<Affiliation>Department of Rheumatology, VU University Medical Center, Amsterdam, the Netherlands.</Affiliation>
</AffiliationInfo>
</Author>
<Author ValidYN="Y">
<LastName>van de Loosdrecht</LastName>
<ForeName>Arjan A</ForeName>
<Initials>AA</Initials>
<AffiliationInfo>
<Affiliation>Department of Hematology, VU University Medical Center, Amsterdam, the Netherlands.</Affiliation>
</AffiliationInfo>
</Author>
<Author ValidYN="Y">
<LastName>Daikeler</LastName>
<ForeName>Thomas</ForeName>
<Initials>T</Initials>
<AffiliationInfo>
<Affiliation>Department of Rheumatology, University Hospital Basel, Basel, Switzerland.</Affiliation>
</AffiliationInfo>
</Author>
<Author ValidYN="Y">
<LastName>Kötter</LastName>
<ForeName>Ina</ForeName>
<Initials>I</Initials>
<AffiliationInfo>
<Affiliation>Department of Internal Medicine II, University Hospital, Tübingen, Germany.</Affiliation>
</AffiliationInfo>
</Author>
<Author ValidYN="Y">
<LastName>Schmalzing</LastName>
<ForeName>Marc</ForeName>
<Initials>M</Initials>
<AffiliationInfo>
<Affiliation>Department of Internal Medicine II, University Hospital, Tübingen, Germany.</Affiliation>
</AffiliationInfo>
</Author>
<Author ValidYN="Y">
<LastName>Martin</LastName>
<ForeName>Thierry</ForeName>
<Initials>T</Initials>
<AffiliationInfo>
<Affiliation>Department of Clinical Immunology, Strasbourg University Hospital, Strasbourg, France.</Affiliation>
</AffiliationInfo>
</Author>
<Author ValidYN="Y">
<LastName>Lioure</LastName>
<ForeName>Bruno</ForeName>
<Initials>B</Initials>
<AffiliationInfo>
<Affiliation>Service d'Hématologie et d'Oncologie, Unité de Greffe de Cellules Souches Hématopoïétiques, Centre Hospitalier Universitaire Hautepierre, Strasbourg, France.</Affiliation>
</AffiliationInfo>
</Author>
<Author ValidYN="Y">
<LastName>Weiner</LastName>
<ForeName>Stefan M</ForeName>
<Initials>SM</Initials>
<AffiliationInfo>
<Affiliation>2.Medizinische Abteilung Krankenhaus der Barmherzigen Brüder Trier, Trier, Germany.</Affiliation>
</AffiliationInfo>
</Author>
<Author ValidYN="Y">
<LastName>Kreuter</LastName>
<ForeName>Alexander</ForeName>
<Initials>A</Initials>
<AffiliationInfo>
<Affiliation>Department of Dermatology, Venereology, and Allergology, HELIOS St. Elisabeth Hospital Oberhausen, Oberhausen,Germany.</Affiliation>
</AffiliationInfo>
</Author>
<Author ValidYN="Y">
<LastName>Deligny</LastName>
<ForeName>Christophe</ForeName>
<Initials>C</Initials>
<AffiliationInfo>
<Affiliation>Service Médecine Interne, Hôpital Pierre Zobda Quitman, Fort-de France, Martinique.</Affiliation>
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</Author>
<Author ValidYN="Y">
<LastName>Durand</LastName>
<ForeName>Jean-Marc</ForeName>
<Initials>JM</Initials>
<AffiliationInfo>
<Affiliation>Department of Internal Medicine CHU La Conception, Assistance Publique-Hôpitaux de Marseille, Marseille, France.</Affiliation>
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</Author>
<Author ValidYN="Y">
<LastName>Emery</LastName>
<ForeName>Paul</ForeName>
<Initials>P</Initials>
<AffiliationInfo>
<Affiliation>Leeds Institute of Rheumatic and Musculoskeletal Medicine, University of Leeds, and NIHR Leeds Musculoskeletal Biomedical Research Unit, Leeds Teaching Hospitals NHS Trust, Leeds, United Kingdom.</Affiliation>
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</Author>
<Author ValidYN="Y">
<LastName>Machold</LastName>
<ForeName>Klaus P</ForeName>
<Initials>KP</Initials>
<AffiliationInfo>
<Affiliation>Klinische Abteilung für Rheumatologie, Medizinische Universität, Vienna, Austria.</Affiliation>
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<Author ValidYN="Y">
<LastName>Sarrot-Reynauld</LastName>
<ForeName>Francoise</ForeName>
<Initials>F</Initials>
<AffiliationInfo>
<Affiliation>Pôle Pluridisciplinaire de Médecine, Centre Hospitalier Universitaire de Grenoble, Grenoble, France.</Affiliation>
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</Author>
<Author ValidYN="Y">
<LastName>Warnatz</LastName>
<ForeName>Klaus</ForeName>
<Initials>K</Initials>
<AffiliationInfo>
<Affiliation>Division of Rheumatology and Clinical Immunology, University Medical Center Freiburg, Freiburg, Germany.</Affiliation>
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<Author ValidYN="Y">
<LastName>Adoue</LastName>
<ForeName>Daniel F P</ForeName>
<Initials>DF</Initials>
<AffiliationInfo>
<Affiliation>Service Médecine Interne, Centre Hospitalier Universitaire Toulouse.</Affiliation>
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</Author>
<Author ValidYN="Y">
<LastName>Constans</LastName>
<ForeName>Joël</ForeName>
<Initials>J</Initials>
<AffiliationInfo>
<Affiliation>Service Médecine Interne et Médecine Vasculaire, Hôpital St-André, Centre Hospitalier Universitaire de Bordeaux, Bordeaux, France.</Affiliation>
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</Author>
<Author ValidYN="Y">
<LastName>Tony</LastName>
<ForeName>Hans-Peter</ForeName>
<Initials>HP</Initials>
<AffiliationInfo>
<Affiliation>Department of Rheumatology and Clinical Immunology, University of Würzburg Medical Center, Würzburg, Germany.</Affiliation>
</AffiliationInfo>
</Author>
<Author ValidYN="Y">
<LastName>Del Papa</LastName>
<ForeName>Nicoletta</ForeName>
<Initials>N</Initials>
<AffiliationInfo>
<Affiliation>Day Hospital Reumatologia, Ospedale G. Pini, Milan, Italy.</Affiliation>
</AffiliationInfo>
</Author>
<Author ValidYN="Y">
<LastName>Fassas</LastName>
<ForeName>Athanasios</ForeName>
<Initials>A</Initials>
<AffiliationInfo>
<Affiliation>Department of Hematology, Cell and Gene Therapy Center, George Papanicolaou Hospital, Thessaloniki, Greece.</Affiliation>
</AffiliationInfo>
</Author>
<Author ValidYN="Y">
<LastName>Himsel</LastName>
<ForeName>Andrea</ForeName>
<Initials>A</Initials>
<AffiliationInfo>
<Affiliation>Department of Rheumatology, University Hospital Frankfurt, Frankfurt, Germany.</Affiliation>
</AffiliationInfo>
</Author>
<Author ValidYN="Y">
<LastName>Launay</LastName>
<ForeName>David</ForeName>
<Initials>D</Initials>
<AffiliationInfo>
<Affiliation>Service de Médecine Interne, Hôpital Claude-Huriez, Lille, France.</Affiliation>
</AffiliationInfo>
</Author>
<Author ValidYN="Y">
<LastName>Lo Monaco</LastName>
<ForeName>Andrea</ForeName>
<Initials>A</Initials>
<AffiliationInfo>
<Affiliation>Section and Unit of Rheumatology, Department of Medical Sciences, University of Ferrara, Italy.</Affiliation>
</AffiliationInfo>
</Author>
<Author ValidYN="Y">
<LastName>Philippe</LastName>
<ForeName>Pierre</ForeName>
<Initials>P</Initials>
<AffiliationInfo>
<Affiliation>Service de Médecine Interne, Centre Hospitalier Universitaire Estaing, Clermont-Ferrand, France.</Affiliation>
</AffiliationInfo>
</Author>
<Author ValidYN="Y">
<LastName>Quéré</LastName>
<ForeName>Isabelle</ForeName>
<Initials>I</Initials>
<AffiliationInfo>
<Affiliation>Department of Internal Medicine, Montpellier University Hospital, Montpellier, France.</Affiliation>
</AffiliationInfo>
</Author>
<Author ValidYN="Y">
<LastName>Rich</LastName>
<ForeName>Éric</ForeName>
<Initials>É</Initials>
<AffiliationInfo>
<Affiliation>Centre Hospitalier de l'Université de Montréal, Montréal, Québec, Canada.</Affiliation>
</AffiliationInfo>
</Author>
<Author ValidYN="Y">
<LastName>Westhovens</LastName>
<ForeName>Rene</ForeName>
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