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<record><TEI><teiHeader><fileDesc><titleStmt><title xml:lang="en">Baseline characteristics and disease burden in patients in the International Paroxysmal Nocturnal Hemoglobinuria Registry</title><author><name sortKey="Schrezenmeier, Hubert" sort="Schrezenmeier, Hubert" uniqKey="Schrezenmeier H" first="Hubert" last="Schrezenmeier">Hubert Schrezenmeier</name><affiliation><nlm:aff id="af1-0990922">Institute of Clinical Transfusion Medicine and Immunogenetics, German Red Cross Blood Transfusion Service Baden-Württemberg–Hessen, and Institute of Transfusion Medicine, University of Ulm, Germany</nlm:aff></affiliation></author><author><name sortKey="Muus, Petra" sort="Muus, Petra" uniqKey="Muus P" first="Petra" last="Muus">Petra Muus</name><affiliation><nlm:aff id="af2-0990922">Radboud University Medical Centre, Nijmegen, The Netherlands</nlm:aff></affiliation></author><author><name sortKey="Socie, Gerard" sort="Socie, Gerard" uniqKey="Socie G" first="Gérard" last="Socié">Gérard Socié</name><affiliation><nlm:aff id="af3-0990922">Hôpital Saint-Louis and Institut National de la Santé et de la Recherche Médicale, Paris, France</nlm:aff></affiliation></author><author><name sortKey="Szer, Jeffrey" sort="Szer, Jeffrey" uniqKey="Szer J" first="Jeffrey" last="Szer">Jeffrey Szer</name><affiliation><nlm:aff id="af4-0990922">Royal Melbourne Hospital, Australia</nlm:aff></affiliation></author><author><name sortKey="Urbano Ispizua, Alvaro" sort="Urbano Ispizua, Alvaro" uniqKey="Urbano Ispizua A" first="Alvaro" last="Urbano-Ispizua">Alvaro Urbano-Ispizua</name><affiliation><nlm:aff id="af5-0990922">Hospital Clinic, University of Barcelona, Institute of Research Josep Carreras, Barcelona, Spain</nlm:aff></affiliation></author><author><name sortKey="Maciejewski, Jaroslaw P" sort="Maciejewski, Jaroslaw P" uniqKey="Maciejewski J" first="Jaroslaw P." last="Maciejewski">Jaroslaw P. Maciejewski</name><affiliation><nlm:aff id="af6-0990922">Taussig Cancer Center, Cleveland Clinic, Cleveland, OH, USA</nlm:aff></affiliation></author><author><name sortKey="Brodsky, Robert A" sort="Brodsky, Robert A" uniqKey="Brodsky R" first="Robert A." last="Brodsky">Robert A. Brodsky</name><affiliation><nlm:aff id="af7-0990922">Johns Hopkins University School of Medicine, Baltimore, MD, USA</nlm:aff></affiliation></author><author><name sortKey="Bessler, Monica" sort="Bessler, Monica" uniqKey="Bessler M" first="Monica" last="Bessler">Monica Bessler</name><affiliation><nlm:aff id="af8-0990922">Department of Hematology, University of Pennsylvania School of Medicine, and Children’s Hospital of Philadelphia, PA, USA</nlm:aff></affiliation></author><author><name sortKey="Kanakura, Yuzuru" sort="Kanakura, Yuzuru" uniqKey="Kanakura Y" first="Yuzuru" last="Kanakura">Yuzuru Kanakura</name><affiliation><nlm:aff id="af9-0990922">Osaka University Graduate School of Medicine, Japan</nlm:aff></affiliation></author><author><name sortKey="Rosse, Wendell" sort="Rosse, Wendell" uniqKey="Rosse W" first="Wendell" last="Rosse">Wendell Rosse</name><affiliation><nlm:aff id="af10-0990922">Duke University Medical Center, Durham, NC, USA</nlm:aff></affiliation></author><author><name sortKey="Khursigara, Gus" sort="Khursigara, Gus" uniqKey="Khursigara G" first="Gus" last="Khursigara">Gus Khursigara</name><affiliation><nlm:aff id="af11-0990922">Alexion Pharmaceuticals, Inc., Cheshire, CT, USA</nlm:aff></affiliation></author><author><name sortKey="Bedrosian, Camille" sort="Bedrosian, Camille" uniqKey="Bedrosian C" first="Camille" last="Bedrosian">Camille Bedrosian</name><affiliation><nlm:aff id="af11-0990922">Alexion Pharmaceuticals, Inc., Cheshire, CT, USA</nlm:aff></affiliation></author><author><name sortKey="Hillmen, Peter" sort="Hillmen, Peter" uniqKey="Hillmen P" first="Peter" last="Hillmen">Peter Hillmen</name><affiliation><nlm:aff id="af12-0990922">Department of Haematology, St James’ University Hospital, Leeds, UK</nlm:aff></affiliation></author></titleStmt><publicationStmt><idno type="wicri:source">PMC</idno><idno type="pmid">24488565</idno><idno type="pmc">4008114</idno><idno type="url">http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4008114</idno><idno type="RBID">PMC:4008114</idno><idno type="doi">10.3324/haematol.2013.093161</idno><date when="2014">2014</date><idno type="wicri:Area/Pmc/Corpus">001857</idno><idno type="wicri:explorRef" wicri:stream="Pmc" wicri:step="Corpus" wicri:corpus="PMC">001857</idno></publicationStmt><sourceDesc><biblStruct><analytic><title xml:lang="en" level="a" type="main">Baseline characteristics and disease burden in patients in the International Paroxysmal Nocturnal Hemoglobinuria Registry</title><author><name sortKey="Schrezenmeier, Hubert" sort="Schrezenmeier, Hubert" uniqKey="Schrezenmeier H" first="Hubert" last="Schrezenmeier">Hubert Schrezenmeier</name><affiliation><nlm:aff id="af1-0990922">Institute of Clinical Transfusion Medicine and Immunogenetics, German Red Cross Blood Transfusion Service Baden-Württemberg–Hessen, and Institute of Transfusion Medicine, University of Ulm, Germany</nlm:aff></affiliation></author><author><name sortKey="Muus, Petra" sort="Muus, Petra" uniqKey="Muus P" first="Petra" last="Muus">Petra Muus</name><affiliation><nlm:aff id="af2-0990922">Radboud University Medical Centre, Nijmegen, The Netherlands</nlm:aff></affiliation></author><author><name sortKey="Socie, Gerard" sort="Socie, Gerard" uniqKey="Socie G" first="Gérard" last="Socié">Gérard Socié</name><affiliation><nlm:aff id="af3-0990922">Hôpital Saint-Louis and Institut National de la Santé et de la Recherche Médicale, Paris, France</nlm:aff></affiliation></author><author><name sortKey="Szer, Jeffrey" sort="Szer, Jeffrey" uniqKey="Szer J" first="Jeffrey" last="Szer">Jeffrey Szer</name><affiliation><nlm:aff id="af4-0990922">Royal Melbourne Hospital, Australia</nlm:aff></affiliation></author><author><name sortKey="Urbano Ispizua, Alvaro" sort="Urbano Ispizua, Alvaro" uniqKey="Urbano Ispizua A" first="Alvaro" last="Urbano-Ispizua">Alvaro Urbano-Ispizua</name><affiliation><nlm:aff id="af5-0990922">Hospital Clinic, University of Barcelona, Institute of Research Josep Carreras, Barcelona, Spain</nlm:aff></affiliation></author><author><name sortKey="Maciejewski, Jaroslaw P" sort="Maciejewski, Jaroslaw P" uniqKey="Maciejewski J" first="Jaroslaw P." last="Maciejewski">Jaroslaw P. Maciejewski</name><affiliation><nlm:aff id="af6-0990922">Taussig Cancer Center, Cleveland Clinic, Cleveland, OH, USA</nlm:aff></affiliation></author><author><name sortKey="Brodsky, Robert A" sort="Brodsky, Robert A" uniqKey="Brodsky R" first="Robert A." last="Brodsky">Robert A. Brodsky</name><affiliation><nlm:aff id="af7-0990922">Johns Hopkins University School of Medicine, Baltimore, MD, USA</nlm:aff></affiliation></author><author><name sortKey="Bessler, Monica" sort="Bessler, Monica" uniqKey="Bessler M" first="Monica" last="Bessler">Monica Bessler</name><affiliation><nlm:aff id="af8-0990922">Department of Hematology, University of Pennsylvania School of Medicine, and Children’s Hospital of Philadelphia, PA, USA</nlm:aff></affiliation></author><author><name sortKey="Kanakura, Yuzuru" sort="Kanakura, Yuzuru" uniqKey="Kanakura Y" first="Yuzuru" last="Kanakura">Yuzuru Kanakura</name><affiliation><nlm:aff id="af9-0990922">Osaka University Graduate School of Medicine, Japan</nlm:aff></affiliation></author><author><name sortKey="Rosse, Wendell" sort="Rosse, Wendell" uniqKey="Rosse W" first="Wendell" last="Rosse">Wendell Rosse</name><affiliation><nlm:aff id="af10-0990922">Duke University Medical Center, Durham, NC, USA</nlm:aff></affiliation></author><author><name sortKey="Khursigara, Gus" sort="Khursigara, Gus" uniqKey="Khursigara G" first="Gus" last="Khursigara">Gus Khursigara</name><affiliation><nlm:aff id="af11-0990922">Alexion Pharmaceuticals, Inc., Cheshire, CT, USA</nlm:aff></affiliation></author><author><name sortKey="Bedrosian, Camille" sort="Bedrosian, Camille" uniqKey="Bedrosian C" first="Camille" last="Bedrosian">Camille Bedrosian</name><affiliation><nlm:aff id="af11-0990922">Alexion Pharmaceuticals, Inc., Cheshire, CT, USA</nlm:aff></affiliation></author><author><name sortKey="Hillmen, Peter" sort="Hillmen, Peter" uniqKey="Hillmen P" first="Peter" last="Hillmen">Peter Hillmen</name><affiliation><nlm:aff id="af12-0990922">Department of Haematology, St James’ University Hospital, Leeds, UK</nlm:aff></affiliation></author></analytic><series><title level="j">Haematologica</title><idno type="ISSN">0390-6078</idno><idno type="eISSN">1592-8721</idno><imprint><date when="2014">2014</date></imprint></series></biblStruct></sourceDesc></fileDesc><profileDesc><textClass></textClass></profileDesc></teiHeader><front><div type="abstract" xml:lang="en"><p>Paroxysmal nocturnal hemoglobinuria is a rare, acquired disease associated with hemolytic anemia, bone marrow failure, thrombosis, and, frequently, poor quality of life. The International PNH Registry is a worldwide, observational, non-interventional study collecting safety, effectiveness, and quality-of-life data from patients with a confirmed paroxysmal nocturnal hemoglobinuria diagnosis or detectable paroxysmal nocturnal hemoglobinuria clone, irrespective of treatment. In addition to evaluating the long-term safety and effectiveness of eculizumab in a global population, the registry aims to improve diagnosis, optimize patient management and outcomes, and enhance the understanding of the natural history of paroxysmal nocturnal hemoglobinuria. Here we report the characteristics of the first 1610 patients enrolled. Median disease duration was 4.6 years. Median granulocyte paroxysmal nocturnal hemoglobinuria clone size was 68.1% (range 0.01–100%). Overall, 16% of patients had a history of thrombotic events and 14% a history of impaired renal function. Therapies included anticoagulation (31%), immunosuppression (19%), and eculizumab (25%). Frequently reported symptoms included fatigue (80%), dyspnea (64%), hemoglobinuria (62%), abdominal pain (44%), and chest pain (33%). Patients suffered from poor quality of life; 23% of patients had been hospitalized due to paroxysmal nocturnal hemoglobinuria-related complications and 17% stated that paroxysmal nocturnal hemoglobinuria was the reason they were not working or were working less. This international registry will provide an ongoing, valuable resource to further the clinical understanding of paroxysmal nocturnal hemoglobinuria.</p></div></front></TEI><pmc article-type="research-article"><pmc-comment>The publisher of this article does not allow downloading of the full text in XML form.</pmc-comment>
<front><journal-meta><journal-id journal-id-type="nlm-ta">Haematologica</journal-id><journal-id journal-id-type="iso-abbrev">Haematologica</journal-id><journal-id journal-id-type="hwp">haematol</journal-id><journal-id journal-id-type="publisher-id">Haematologica</journal-id><journal-title-group><journal-title>Haematologica</journal-title></journal-title-group><issn pub-type="ppub">0390-6078</issn><issn pub-type="epub">1592-8721</issn><publisher><publisher-name>Ferrata Storti Foundation</publisher-name></publisher></journal-meta><article-meta><article-id pub-id-type="pmid">24488565</article-id><article-id pub-id-type="pmc">4008114</article-id><article-id pub-id-type="doi">10.3324/haematol.2013.093161</article-id><article-id pub-id-type="publisher-id">0990922</article-id><article-categories><subj-group subj-group-type="heading"><subject>Articles</subject><subj-group subj-group-type="heading"><subject>Paroxysmal Nocturnal Hemoglobinuria</subject></subj-group></subj-group></article-categories><title-group><article-title>Baseline characteristics and disease burden in patients in the International Paroxysmal Nocturnal Hemoglobinuria Registry</article-title></title-group><contrib-group><contrib contrib-type="author"><name><surname>Schrezenmeier</surname><given-names>Hubert</given-names></name><xref ref-type="aff" rid="af1-0990922">1</xref></contrib><contrib contrib-type="author"><name><surname>Muus</surname><given-names>Petra</given-names></name><xref ref-type="aff" rid="af2-0990922">2</xref></contrib><contrib contrib-type="author"><name><surname>Socié</surname><given-names>Gérard</given-names></name><xref ref-type="aff" rid="af3-0990922">3</xref></contrib><contrib contrib-type="author"><name><surname>Szer</surname><given-names>Jeffrey</given-names></name><xref ref-type="aff" rid="af4-0990922">4</xref></contrib><contrib contrib-type="author"><name><surname>Urbano-Ispizua</surname><given-names>Alvaro</given-names></name><xref ref-type="aff" rid="af5-0990922">5</xref></contrib><contrib contrib-type="author"><name><surname>Maciejewski</surname><given-names>Jaroslaw P.</given-names></name><xref ref-type="aff" rid="af6-0990922">6</xref></contrib><contrib contrib-type="author"><name><surname>Brodsky</surname><given-names>Robert A.</given-names></name><xref ref-type="aff" rid="af7-0990922">7</xref></contrib><contrib contrib-type="author"><name><surname>Bessler</surname><given-names>Monica</given-names></name><xref ref-type="aff" rid="af8-0990922">8</xref></contrib><contrib contrib-type="author"><name><surname>Kanakura</surname><given-names>Yuzuru</given-names></name><xref ref-type="aff" rid="af9-0990922">9</xref></contrib><contrib contrib-type="author"><name><surname>Rosse</surname><given-names>Wendell</given-names></name><xref ref-type="aff" rid="af10-0990922">10</xref></contrib><contrib contrib-type="author"><name><surname>Khursigara</surname><given-names>Gus</given-names></name><xref ref-type="aff" rid="af11-0990922">11</xref></contrib><contrib contrib-type="author"><name><surname>Bedrosian</surname><given-names>Camille</given-names></name><xref ref-type="aff" rid="af11-0990922">11</xref></contrib><contrib contrib-type="author"><name><surname>Hillmen</surname><given-names>Peter</given-names></name><xref ref-type="aff" rid="af12-0990922">12</xref><xref ref-type="corresp" rid="c1-0990922"></xref></contrib></contrib-group><aff id="af1-0990922"><label>1</label>Institute of Clinical Transfusion Medicine and Immunogenetics, German Red Cross Blood Transfusion Service Baden-Württemberg–Hessen, and Institute of Transfusion Medicine, University of Ulm, Germany</aff><aff id="af2-0990922"><label>2</label>Radboud University Medical Centre, Nijmegen, The Netherlands</aff><aff id="af3-0990922"><label>3</label>Hôpital Saint-Louis and Institut National de la Santé et de la Recherche Médicale, Paris, France</aff><aff id="af4-0990922"><label>4</label>Royal Melbourne Hospital, Australia</aff><aff id="af5-0990922"><label>5</label>Hospital Clinic, University of Barcelona, Institute of Research Josep Carreras, Barcelona, Spain</aff><aff id="af6-0990922"><label>6</label>Taussig Cancer Center, Cleveland Clinic, Cleveland, OH, USA</aff><aff id="af7-0990922"><label>7</label>Johns Hopkins University School of Medicine, Baltimore, MD, USA</aff><aff id="af8-0990922"><label>8</label>Department of Hematology, University of Pennsylvania School of Medicine, and Children’s Hospital of Philadelphia, PA, USA</aff><aff id="af9-0990922"><label>9</label>Osaka University Graduate School of Medicine, Japan</aff><aff id="af10-0990922"><label>10</label>Duke University Medical Center, Durham, NC, USA</aff><aff id="af11-0990922"><label>11</label>Alexion Pharmaceuticals, Inc., Cheshire, CT, USA</aff><aff id="af12-0990922"><label>12</label>Department of Haematology, St James’ University Hospital, Leeds, UK</aff><author-notes><corresp id="c1-0990922">Correspondence: <email>peter.hillmen@nhs.net</email></corresp></author-notes><pub-date pub-type="ppub"><month>5</month><year>2014</year></pub-date><volume>99</volume><issue>5</issue><fpage>922</fpage><lpage>929</lpage><history><date date-type="received"><day>12</day><month>6</month><year>2013</year></date><date date-type="accepted"><day>29</day><month>1</month><year>2014</year></date></history><permissions><copyright-statement>Copyright© Ferrata Storti Foundation</copyright-statement><copyright-year>2014</copyright-year></permissions><self-uri content-type="pdf" xlink:type="simple" xlink:href="99922.pdf"></self-uri><abstract><p>Paroxysmal nocturnal hemoglobinuria is a rare, acquired disease associated with hemolytic anemia, bone marrow failure, thrombosis, and, frequently, poor quality of life. The International PNH Registry is a worldwide, observational, non-interventional study collecting safety, effectiveness, and quality-of-life data from patients with a confirmed paroxysmal nocturnal hemoglobinuria diagnosis or detectable paroxysmal nocturnal hemoglobinuria clone, irrespective of treatment. In addition to evaluating the long-term safety and effectiveness of eculizumab in a global population, the registry aims to improve diagnosis, optimize patient management and outcomes, and enhance the understanding of the natural history of paroxysmal nocturnal hemoglobinuria. Here we report the characteristics of the first 1610 patients enrolled. Median disease duration was 4.6 years. Median granulocyte paroxysmal nocturnal hemoglobinuria clone size was 68.1% (range 0.01–100%). Overall, 16% of patients had a history of thrombotic events and 14% a history of impaired renal function. Therapies included anticoagulation (31%), immunosuppression (19%), and eculizumab (25%). Frequently reported symptoms included fatigue (80%), dyspnea (64%), hemoglobinuria (62%), abdominal pain (44%), and chest pain (33%). Patients suffered from poor quality of life; 23% of patients had been hospitalized due to paroxysmal nocturnal hemoglobinuria-related complications and 17% stated that paroxysmal nocturnal hemoglobinuria was the reason they were not working or were working less. This international registry will provide an ongoing, valuable resource to further the clinical understanding of paroxysmal nocturnal hemoglobinuria.</p></abstract></article-meta></front></pmc></record>Pour manipuler ce document sous Unix (Dilib)
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