{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,22]],"date-time":"2026-10-22T23:54:01Z","timestamp":1792713241147,"version":"4.5.0"},"reference-count":34,"publisher":"Wiley","issue":"1","license":[{"start":{"date-parts":[[2013,4,25]],"date-time":"2013-04-25T00:00:00Z","timestamp":1366848000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Br J Haematol"],"published-print":{"date-parts":[[2013,7]]},"abstract":"<jats:title>Summary<\/jats:title>\n                  <jats:p>\n                    Paroxysmal nocturnal haemoglobinuria (\n                    <jats:styled-content style=\"fixed-case\">PNH<\/jats:styled-content>\n                    ) is characterized by chronic, uncontrolled complement activation resulting in elevated intravascular haemolysis and morbidities, including fatigue, dyspnoea, abdominal pain, pulmonary hypertension, thrombotic events (\n                    <jats:styled-content style=\"fixed-case\">TE<\/jats:styled-content>\n                    s) and chronic kidney disease (\n                    <jats:styled-content style=\"fixed-case\">CKD<\/jats:styled-content>\n                    ). The long\u2010term safety and efficacy of eculizumab, a humanized monoclonal antibody that inhibits terminal complement activation, was investigated in 195 patients over 66\u00a0months. Four patient deaths were reported, all unrelated to treatment, resulting in a 3\u2010year survival estimate of 97\u00b76%. All patients showed a reduction in lactate dehydrogenase levels, which was sustained over the course of treatment (median reduction of 86\u00b79% at 36\u00a0months), reflecting inhibition of chronic haemolysis.\n                    <jats:styled-content style=\"fixed-case\">TE<\/jats:styled-content>\n                    s decreased by 81\u00b78%, with 96\u00b74% of patients remaining free of\n                    <jats:styled-content style=\"fixed-case\">TE<\/jats:styled-content>\n                    s. Patients also showed a time\u2010dependent improvement in renal function: 93\u00b71% of patients exhibited improvement or stabilization in\n                    <jats:styled-content style=\"fixed-case\">CKD<\/jats:styled-content>\n                    score at 36\u00a0months. Transfusion independence increased by 90\u00b70% from baseline, with the number of red blood cell units transfused decreasing by 54\u00b77%. Eculizumab was well tolerated, with no evidence of cumulative toxicity and a decreasing occurrence of adverse events over time. Eculizumab has a substantial impact on the symptoms and complications of\n                    <jats:styled-content style=\"fixed-case\">PNH<\/jats:styled-content>\n                    and results a significant improvement in patient survival.\n                  <\/jats:p>","DOI":"10.1111\/bjh.12347","type":"journal-article","created":{"date-parts":[[2013,4,25]],"date-time":"2013-04-25T08:36:35Z","timestamp":1366878995000},"page":"62-73","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":353,"title":["Long\u2010term safety and efficacy of sustained eculizumab treatment in patients with paroxysmal nocturnal haemoglobinuria"],"prefix":"10.1111","volume":"162","author":[{"given":"Peter","family":"Hillmen","sequence":"first","affiliation":[{"name":"St James's University Hospital Leeds UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Petra","family":"Muus","sequence":"additional","affiliation":[{"name":"Radboud 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