{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,16]],"date-time":"2026-10-16T15:52:23Z","timestamp":1792165943930,"version":"4.4.0"},"reference-count":29,"publisher":"Wiley","issue":"9","license":[{"start":{"date-parts":[[2016,9,16]],"date-time":"2016-09-16T00:00:00Z","timestamp":1473984000000},"content-version":"vor","delay-in-days":15,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Internal Medicine Journal"],"published-print":{"date-parts":[[2016,9]]},"abstract":"<jats:sec><jats:title>Background<\/jats:title><jats:p>Paroxysmal nocturnal haemoglobinuria (<jats:styled-content style=\"fixed-case\">PNH<\/jats:styled-content>) is a rare disease. Although much progress has been made in the understanding of the pathophysiology of the disease, far less is known with respect to the clinical outcomes of patients with <jats:styled-content style=\"fixed-case\">PNH<\/jats:styled-content>. Few retrospective studies provide survival estimates, and even fewer have explored the clinical heterogeneity of the disease. Haemolytic and aplastic anaemia (<jats:styled-content style=\"fixed-case\">AA<\/jats:styled-content>) forms of the disease have been recognised as main disease categories, with the haemolytic form being associated with the worst prognosis by the largest studied cohort some years ago.<\/jats:p><\/jats:sec><jats:sec><jats:title>Aims<\/jats:title><jats:p>To describe mortality and causes of death in <jats:styled-content style=\"fixed-case\">PNH<\/jats:styled-content> overall and by <jats:styled-content style=\"fixed-case\">PNH<\/jats:styled-content> classification and to evaluate risk factors associated with mortality.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>We analysed data of 2356 patients enrolled in the International <jats:styled-content style=\"fixed-case\">PNH<\/jats:styled-content> Registry with multivariate analyses, using time\u2010dependent covariates. Patients were classified into haemolytic, <jats:styled-content style=\"fixed-case\">AA<\/jats:styled-content>\/<jats:styled-content style=\"fixed-case\">PNH<\/jats:styled-content> syndrome or intermediate <jats:styled-content style=\"fixed-case\">PNH<\/jats:styled-content>.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Overall, 122 (5.2%) patients died after enrolment, the incidence according to subcategories being 5.1, 11.7, 2.0 and 4.8% for patients with haemolytic <jats:styled-content style=\"fixed-case\">PNH<\/jats:styled-content>, <jats:styled-content style=\"fixed-case\">AA\u2010PNH<\/jats:styled-content>, intermediate and insufficient data respectively. Older age and decreased performance status also affected survival in multivariate analysis. Improved outcome of patients with haemolytic <jats:styled-content style=\"fixed-case\">PNH<\/jats:styled-content> suggests that eculizumab treatment in <jats:styled-content style=\"fixed-case\">PNH<\/jats:styled-content> may be associated with improved survival.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>A detailed analysis of clinical presentations and causes of death in patients with <jats:styled-content style=\"fixed-case\">PNH<\/jats:styled-content>, overall and by disease subcategories, provide evidence that in the current era, patients with haemolytic <jats:styled-content style=\"fixed-case\">PNH<\/jats:styled-content> are no longer those who harbour the worst prognosis. This finding differs sharply from what has been previously reported.<\/jats:p><\/jats:sec>","DOI":"10.1111\/imj.13160","type":"journal-article","created":{"date-parts":[[2016,6,15]],"date-time":"2016-06-15T17:45:57Z","timestamp":1466012757000},"page":"1044-1053","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":73,"title":["Changing prognosis in paroxysmal nocturnal haemoglobinuria disease subcategories: an analysis of the International <scp>PNH<\/scp> Registry"],"prefix":"10.1111","volume":"46","author":[{"given":"G.","family":"Soci\u00e9","sequence":"first","affiliation":[{"name":"Hematology Transplantation, AP\u2010HP, Hospital Saint Louis University Paris VII Denis Diderot and Inserm UMR 1160  Paris France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"H.","family":"Schrezenmeier","sequence":"additional","affiliation":[{"name":"Institute of Transfusion Medicine University of Ulm Ulm and Institute of Clinical Transfusion Medicine and Immunogenetics, German Red Cross Blood Transfusion Service Baden\u2010W\u00fcrttemberg\u2010Hessen and University Hospital Ulm  Ulm Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"P.","family":"Muus","sequence":"additional","affiliation":[{"name":"Department of Hematology Radboud UMC  Nijmegen the Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"I.","family":"Lisukov","sequence":"additional","affiliation":[{"name":"Institution of Children Hematology and Transplantation  St. Petersburg Russia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"A.","family":"R\u00f6th","sequence":"additional","affiliation":[{"name":"University Hospital Essen  Essen Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"A.","family":"Kulasekararaj","sequence":"additional","affiliation":[{"name":"Kings College Hospital  London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"J. 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