{"database":"biostudies-literature","file_versions":[],"scores":null,"additional":{"omics_type":["Unknown"],"volume":["115(2)"],"submitter":["de Latour RP"],"funding":["Swedish Orphan Biovitrum AB","Swedish Orphan Biovitrum"],"pubmed_abstract":["<h4>Background</h4>Paroxysmal nocturnal haemoglobinuria (PNH) is an ultra-rare, acquired non-malignant haematological disorder characterised by thrombosis risk, serious complications and debilitating symptoms in untreated patients.<h4>Objective</h4>This anchored indirect treatment comparison (ITC) evaluated efficacy data between proximal complement 3 inhibitor (C3i) pegcetacoplan and factor B inhibitor, iptacopan, in patients with PNH previously treated with complement 5 inhibitors (C5i; eculizumab, ravulizumab).<h4>Methods</h4>Respective pivotal studies provided patient-level trial data for pegcetacoplan (16-week PEGASUS [NCT03500549]) and published data for iptacopan (24-week APPLY PNH [NCT04558918]). Differences in study design, duration and statistical methods between PEGASUS and APPLY"],"journal":["European journal of haematology"],"pagination":["125-133"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC12224562"],"repository":["biostudies-literature"],"pubmed_title":["Anchored Indirect Treatment Comparison Finds Comparable Effects of Pegcetacoplan and Iptacopan in Paroxysmal Nocturnal Haemoglobinuria."],"pmcid":["PMC12224562"],"pubmed_authors":["de Latour RP","Hillmen P","Wojciechowski P","Szer J","Kulasekararaj A","Wilson K","Czech B","Dingli D","Nazir J","Hakimi Z"],"additional_accession":[]},"is_claimable":false,"name":"Anchored Indirect Treatment Comparison Finds Comparable Effects of Pegcetacoplan and Iptacopan in Paroxysmal Nocturnal Haemoglobinuria.","description":"<h4>Background</h4>Paroxysmal nocturnal haemoglobinuria (PNH) is an ultra-rare, acquired non-malignant haematological disorder characterised by thrombosis risk, serious complications and debilitating symptoms in untreated patients.<h4>Objective</h4>This anchored indirect treatment comparison (ITC) evaluated efficacy data between proximal complement 3 inhibitor (C3i) pegcetacoplan and factor B inhibitor, iptacopan, in patients with PNH previously treated with complement 5 inhibitors (C5i; eculizumab, ravulizumab).<h4>Methods</h4>Respective pivotal studies provided patient-level trial data for pegcetacoplan (16-week PEGASUS [NCT03500549]) and published data for iptacopan (24-week APPLY PNH [NCT04558918]). Differences in study design, duration and statistical methods between PEGASUS and APPLY","dates":{"release":"2025-01-01T00:00:00Z","publication":"2025 Aug","modification":"2026-06-02T08:53:28.735Z","creation":"2026-04-16T03:12:37.778Z"},"accession":"S-EPMC12224562","cross_references":{"pubmed":["40285403"],"doi":["10.1111/ejh.14422"]}}