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However, considering the risk of life\u2010threatening meningococcal disease, life\u2010long duration and costs, there are strict criteria for initiation of therapy. To evaluate the application and real\u2010world effectiveness of eculizumab in the Netherlands, a multicenter retrospective cohort study was conducted: indications and treatment outcomes were collected for 105 Dutch PNH patients. In all patients, eculizumab was initiated conforming to indications as formulated in the Dutch PNH guideline. According to recently published response criteria, 23.4% of the patients had reached a complete hematological response, 53.2% a good or partial response, and 23.4% a minor response after 12\u2009months of therapy. In the majority of patients the response remained stable during long\u2010term follow\u2010up. The degree and relevance of extravascular hemolysis significantly differed between response groups (<jats:italic>p<\/jats:italic>\u00a0=\u00a00.002). Improvements of EORTC\u2010QLQc30 and FACIT\u2010fatigue scores were observed, however patients reported lower scores than the general population. A detailed evaluation of 18 pregnancies during eculizumab showed no maternal or fetal deaths, and no thromboembolic events during pregnancy. This study demonstrates that the majority of patients benefit from eculizumab when adhering to the indications as formulated in the Dutch PNH guideline. However, novel therapies are needed to further improve real\u2010world outcomes, such as hematological responses and quality of life.<\/jats:p>","DOI":"10.1111\/ejh.13946","type":"journal-article","created":{"date-parts":[[2023,2,22]],"date-time":"2023-02-22T11:35:02Z","timestamp":1677065702000},"page":"648-658","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":9,"title":["Nationwide study of eculizumab in paroxysmal nocturnal hemoglobinuria: Evaluation of treatment indications and outcomes"],"prefix":"10.1111","volume":"110","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-6445-1079","authenticated-orcid":false,"given":"Charlotte C. 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