biostudies-literatureUnknown115(2)de Latour RPSwedish Orphan Biovitrum ABSwedish Orphan Biovitrum<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 APPLYEuropean journal of haematology125-133https://www.ebi.ac.uk/biostudies/studies/S-EPMC12224562biostudies-literatureAnchored Indirect Treatment Comparison Finds Comparable Effects of Pegcetacoplan and Iptacopan in Paroxysmal Nocturnal Haemoglobinuria.PMC12224562de Latour RPHillmen PWojciechowski PSzer JKulasekararaj AWilson KCzech BDingli DNazir JHakimi ZfalseAnchored Indirect Treatment Comparison Finds Comparable Effects of Pegcetacoplan and Iptacopan in Paroxysmal Nocturnal Haemoglobinuria.<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 APPLY2025-01-01T00:00:00Z2025 Aug2026-06-02T08:53:28.735Z2026-04-16T03:12:37.778ZS-EPMC122245624028540310.1111/ejh.14422