{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,18]],"date-time":"2026-08-18T04:49:07Z","timestamp":1787028547001,"version":"build-2736575974"},"reference-count":23,"publisher":"MDPI AG","issue":"16","license":[{"start":{"date-parts":[[2024,8,6]],"date-time":"2024-08-06T00:00:00Z","timestamp":1722902400000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"name":"Apellis Pharmaceuticals, Inc."},{"name":"Swedish Orphan Biovitrum AB"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["IJMS"],"abstract":"<jats:p>Paroxysmal nocturnal haemoglobinuria (PNH) is a rare, potentially life-threatening haematological disease characterised by chronic complement-mediated haemolysis with multiple clinical consequences that impair quality of life. This post hoc analysis assessed haematological and clinical responses to the first targeted complement C3 inhibitor pegcetacoplan in patients with PNH and impaired bone marrow function in the PEGASUS (NCT03500549) and PRINCE (NCT04085601) studies. For patients with impaired bone marrow function, defined herein as haemoglobin &lt;10 g\/dL and absolute neutrophil count &lt;1.5 \u00d7 109 cells\/L, normalisation of the parameters may be difficult. Indeed, 20% and 43% had normalised haemoglobin in PEGASUS and PRINCE, respectively; 60% and 57% had normalised LDH, and 40% and 29% had normalised fatigue scores. A new set of parameters was applied using changes associated with clinically meaningful improvements, namely an increase in haemoglobin to \u22652 g\/dL above baseline, decrease in LDH to \u22641.5\u00d7 the upper limit of normal, and an increase in fatigue scores to \u22655 points above baseline. With these new parameters, 40% and 71% of PEGASUS and PRINCE patients had improved haemoglobin; 60% and 71% had an improvement in LDH, and 60% and 43% had an improvement in fatigue scores. Thus, even patients with impaired bone marrow function may achieve clinically meaningful improvements with pegcetacoplan.<\/jats:p>","DOI":"10.3390\/ijms25168591","type":"journal-article","created":{"date-parts":[[2024,8,6]],"date-time":"2024-08-06T15:24:16Z","timestamp":1722957856000},"page":"8591","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":4,"title":["Moving toward Individual Treatment Goals with Pegcetacoplan in Patients with PNH and Impaired Bone Marrow Function"],"prefix":"10.3390","volume":"25","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-6783-2301","authenticated-orcid":false,"given":"Jeff","family":"Szer","sequence":"first","affiliation":[{"name":"Department of Clinical Haematology, Peter MacCallum Cancer Centre, Royal Melbourne Hospital, Melbourne, VIC 3052, Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jens","family":"Panse","sequence":"additional","affiliation":[{"name":"Department of Oncology, Hematology, Hemostaseology and Stem Cell Transplantation, University Hospital RWTH Aachen, 52074 Aachen, Germany"},{"name":"Centre for Integrated Oncology (CIO) Aachen-Bonn-Cologne-D\u00fcsseldorf (ABCD), 52074 Aachen, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Austin","family":"Kulasekararaj","sequence":"additional","affiliation":[{"name":"Department of Haematological Medicine, King\u2019s College Hospital, National Institute of Health Research\/Wellcome King\u2019s Clinical Research Facility, London SE5 9RS, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0826-1649","authenticated-orcid":false,"given":"Monika","family":"Oliver","sequence":"additional","affiliation":[{"name":"Division of Hemtatology, Department of Apheresis Medicine, University of Alberta Hospital, University of Alberta, Edmonton, AB T6G 2B7, Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0857-8379","authenticated-orcid":false,"given":"Bruno","family":"Fattizzo","sequence":"additional","affiliation":[{"name":"Hematology Unit, Fondazione IRCCS Ca\u2019 Granda Ospedale Maggiore Policlinico, 20122 Milan, Italy"},{"name":"Department of Oncology and Hemato-Oncology, University of Milan, 20122 Milan, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jun-ichi","family":"Nishimura","sequence":"additional","affiliation":[{"name":"Department of Hematology and Oncology, Osaka University Graduate School of Medicine, Osaka 565-0871, Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Regina","family":"Horneff","sequence":"additional","affiliation":[{"name":"Swedish Orphan Biovitrum AB, 171 65 Stockholm, Sweden"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Johan","family":"Szamosi","sequence":"additional","affiliation":[{"name":"Swedish Orphan Biovitrum AB, 171 65 Stockholm, Sweden"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"R\u00e9gis","family":"Peffault de Latour","sequence":"additional","affiliation":[{"name":"French Reference Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria, 75010 Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2024,8,6]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","first-page":"1971","DOI":"10.1007\/s00277-022-04903-x","article-title":"Inhibition of C3 with pegcetacoplan results in normalization of hemolysis markers in paroxysmal nocturnal hemoglobinuria","volume":"101","author":"Wong","year":"2022","journal-title":"Ann. 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