{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,4,16]],"date-time":"2026-04-16T18:31:17Z","timestamp":1776364277899,"version":"3.51.2"},"reference-count":22,"publisher":"Public Library of Science (PLoS)","issue":"4","license":[{"start":{"date-parts":[[2026,4,16]],"date-time":"2026-04-16T00:00:00Z","timestamp":1776297600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"name":"Roche France SAS"}],"content-domain":{"domain":["www.plosone.org"],"crossmark-restriction":false},"short-container-title":["PLoS One"],"abstract":"<jats:sec id=\"sec001\">\n                    <jats:title>Introduction<\/jats:title>\n                    <jats:p>Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired stem-cell disease causing anemia, aplasia and thromboses, affecting ~1\/80,000 patients in France. Treatment mainly involves C5 inhibitors (C5i) and iterative red-blood-cell transfusions. Recent real-world French data on PNH epidemiology and management are scarce in the era of complement inhibitors. This study described PNH epidemiology, patients\u2019 characteristics, and C5i use.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec id=\"sec002\">\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>This was a non-interventional claims study using secondary data from the French national hospital database. Patients with a hospital diagnosis of PNH (ICD-10 code D59.5) over 2018\u20132022 were selected, after exclusion of those with other C5i-treated diseases. A subgroup of C5i initiators was followed from initiation to last hospitalization, to describe C5i treatment patterns.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec id=\"sec003\">\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>The study included 897 patients: 496 (55.3%) receiving a C5i and 218 (24.3%) initiating treatment over 2018\u20132022. In 2022, 725 PNH patients were identified (prevalence ~1\/94,000). Mean (SD) age was 52.1 (21.1) and 45.7 (19.4) years overall and among C5i initiators, respectively. C5i initiators had 20.9 (6.9) dispensations annually. Most patients initiated with eculizumab (n\u2009=\u2009196, 89.9%), and almost half of them (n\u2009=\u200980, 40.8%) switched to ravulizumab.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec id=\"sec004\">\n                    <jats:title>Conclusion<\/jats:title>\n                    <jats:p>PNH prevalence was\u2009~\u20091\/94,000. Most patients initiated eculizumab, with 40.8% switching to ravulizumab.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1371\/journal.pone.0339715","type":"journal-article","created":{"date-parts":[[2026,4,16]],"date-time":"2026-04-16T17:28:21Z","timestamp":1776360501000},"page":"e0339715","update-policy":"https:\/\/doi.org\/10.1371\/journal.pone.corrections_policy","source":"Crossref","is-referenced-by-count":0,"title":["Epidemiology and care management of Paroxysmal Nocturnal Hemoglobinuria (PNH) in a real-world setting in France: Description from the French National Hospitalization Database"],"prefix":"10.1371","volume":"21","author":[{"given":"R\u00e9gis","family":"Peffault de Latour","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Sophie","family":"Pibre","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Julien","family":"Vercruyssen","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Marie Laetitia","family":"Desjuzeur-Bellais","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7664-4521","authenticated-orcid":true,"given":"David","family":"Pau","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9799-1757","authenticated-orcid":true,"given":"St\u00e8ve","family":"B\u00e9nard","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3326-6262","authenticated-orcid":true,"given":"Louis","family":"Chillotti","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Nathalie","family":"Grandfils","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"340","published-online":{"date-parts":[[2026,4,16]]},"reference":[{"key":"pone.0339715.ref001","unstructured":"Orphanet. 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ULTOMIRIS (ravulizumab) [Internet]. Haute Autorit\u00e9 de Sant\u00e9. [cited 2024 Jul 23]. 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