{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,3]],"date-time":"2026-07-03T04:18:21Z","timestamp":1783052301468,"version":"3.54.6"},"reference-count":22,"publisher":"Wiley","issue":"2","license":[{"start":{"date-parts":[[2015,3,29]],"date-time":"2015-03-29T00:00:00Z","timestamp":1427587200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["European J of Haematology"],"published-print":{"date-parts":[[2015,8]]},"abstract":"<jats:title>Abstract<\/jats:title><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 acquired haematopoietic stem cell disorder characterised by persistent haemolysis and platelet activation, severe end\u2010organ damage, an increased risk of thrombosis and early mortality. We present the case of a 56\u2010year\u2010old male with long\u2010standing <jats:styled-content style=\"fixed-case\">PNH<\/jats:styled-content> and significant disease\u2010related morbidity who underwent steroid therapy for approximately 15\u00a0yrs before treatment with eculizumab, a humanized monoclonal antibody that blocks the terminal phase of the complement cascade at the C5 level.<\/jats:p><\/jats:sec><jats:sec><jats:title>Case history<\/jats:title><jats:p>The patient presented with a severely impaired quality of life in 1997 and was diagnosed with <jats:styled-content style=\"fixed-case\">PNH<\/jats:styled-content> 8\u00a0months later, soon after which he was commenced on steroid therapy with prednisone. During long\u2010term steroid therapy with progressive increases in prednisone dose, the patient had frequent haemolytic episodes as well as thrombosis and renal complications. He also experienced Cushing's syndrome with arterial hypertension, insulin\u2010dependent diabetes mellitus, osteoporosis, acne and portal fibrosis. Eculizumab therapy was started in late\u20102009 and led to rapid improvements in haemoglobin and lactate dehydrogenase levels with a complete cessation of haemolytic episodes. Eculizumab has been well tolerated.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>Long\u2010term steroid therapy was not effective in controlling <jats:styled-content style=\"fixed-case\">PNH<\/jats:styled-content> in this patient and was associated with significant comorbidities. Treatment with eculizumab led to major improvements, even after such a long period with relatively uncontrolled disease.<\/jats:p><\/jats:sec>","DOI":"10.1111\/ejh.12480","type":"journal-article","created":{"date-parts":[[2015,3,11]],"date-time":"2015-03-11T07:11:02Z","timestamp":1426057862000},"page":"177-180","source":"Crossref","is-referenced-by-count":5,"title":["Paroxysmal nocturnal haemoglobinuria: to prednisone or not to prednisone? \u2013 a case report of a patient previously treated with steroids for 15\u00a0yrs and significant response on eculizumab"],"prefix":"10.1111","volume":"95","author":[{"given":"Alexander","family":"R\u00f6th","sequence":"first","affiliation":[{"name":"Department of Haematology West German Cancer Center University Hospital University of Duisburg\u2010Essen Essen Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ferras","family":"Alashkar","sequence":"additional","affiliation":[{"name":"Department of Haematology West German Cancer Center University Hospital University of Duisburg\u2010Essen Essen Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"D\u00f6rte","family":"Herich\u2010Terh\u00fcrne","sequence":"additional","affiliation":[{"name":"Department of Haematology West German Cancer Center University Hospital University of Duisburg\u2010Essen Essen Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ulrich","family":"D\u00fchrsen","sequence":"additional","affiliation":[{"name":"Department of Haematology West German Cancer Center University Hospital University of Duisburg\u2010Essen Essen Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2015,3,29]]},"reference":[{"key":"e_1_2_6_2_1","doi-asserted-by":"publisher","DOI":"10.1097\/MOH.0b013e328351c348"},{"key":"e_1_2_6_3_1","doi-asserted-by":"publisher","DOI":"10.1002\/0471142735.im1305s11"},{"key":"e_1_2_6_4_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood.V86.9.3277.bloodjournal8693277"},{"key":"e_1_2_6_5_1","doi-asserted-by":"publisher","DOI":"10.1007\/s12185-013-1346-4"},{"key":"e_1_2_6_6_1","first-page":"2","article-title":"Paroxysmal nocturnal hemoglobinuria: pathogenesis, testing, and diagnosis","volume":"11","author":"Sharma VR","year":"2013","journal-title":"Clin Adv Hematol Oncol"},{"key":"e_1_2_6_7_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2009-03-195966"},{"key":"e_1_2_6_8_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2012-09-311381"},{"key":"e_1_2_6_9_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(09)60001-5"},{"key":"e_1_2_6_10_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2008-04-151613"},{"key":"e_1_2_6_11_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2005-04-1717"},{"key":"e_1_2_6_12_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJM199511093331904"},{"key":"e_1_2_6_13_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(95)12360-1"},{"key":"e_1_2_6_14_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1600-0609.2011.01701.x"},{"key":"e_1_2_6_15_1","doi-asserted-by":"publisher","DOI":"10.1002\/ajh.21757"},{"key":"e_1_2_6_16_1","doi-asserted-by":"publisher","DOI":"10.1002\/ajh.2830250108"},{"key":"e_1_2_6_17_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood.V60.1.20.20"},{"key":"e_1_2_6_18_1","doi-asserted-by":"publisher","DOI":"10.1159\/000204254"},{"key":"e_1_2_6_19_1","doi-asserted-by":"publisher","DOI":"10.2147\/BTT.S1420"},{"key":"e_1_2_6_20_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2007-06-094136"},{"key":"e_1_2_6_21_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa031688"},{"key":"e_1_2_6_22_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa061648"},{"key":"e_1_2_6_23_1","doi-asserted-by":"publisher","DOI":"10.1111\/bjh.12347"}],"container-title":["European Journal of Haematology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.wiley.com\/onlinelibrary\/tdm\/v1\/articles\/10.1111%2Fejh.12480","content-type":"unspecified","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1111\/ejh.12480","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,10,3]],"date-time":"2023-10-03T03:04:33Z","timestamp":1696302273000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1111\/ejh.12480"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2015,3,29]]},"references-count":22,"journal-issue":{"issue":"2","published-print":{"date-parts":[[2015,8]]}},"alternative-id":["10.1111\/ejh.12480"],"URL":"https:\/\/doi.org\/10.1111\/ejh.12480","archive":["Portico"],"relation":{},"ISSN":["0902-4441","1600-0609"],"issn-type":[{"value":"0902-4441","type":"print"},{"value":"1600-0609","type":"electronic"}],"subject":[],"published":{"date-parts":[[2015,3,29]]}}}