Efficacy and Safety of Pegcetacoplan in PNH Patients with Extra-Vascular Hemolysis Under Anti-C5: The Real-Life Experience of the National French Reference Center
Abstract
Introduction: Pegcetacoplan (PEG), the first proximal C3 inhibitor controls intravascular hemolysis without inducing extravascular hemolysis (EVH). PEG was shown to be superior to eculizumab in improving change in hemoglobin level in patients with persistent anemia du to EVH in patients under anti-C5 in a randomized control trial (PEGASUS trial). PEG is now approved in France for the treatment of adult patient who remain anemic after at least 3 months of anti-C5 treatment. Here we report the real-life experience of the French national reference center.
Methods: clinical and biological data of all consecutive's patients who received PEG outside a clinical trial and who have agreed to have their data recorded in the French national center database were analyzed.
Results: From June 2022 to July 2024, 39 patients were included. Twenty-six (66.7%) were female and median age at diagnosis of PNH was 36.4 years (IQR [24.8; 61.3]). Thirteen patients (33.3%) had previously received an immunosuppressive therapy for associated aplastic anemia and 7 (17.9%) of them had experienced a thrombotic event. The median time from anti-C5 start to PEG initiation was 39.6 months (IQR [20.25 ;65.43] (6.689 ;198.1). Twelve patients (30.8%) were receiving ravulizumab and 27 (69.2%) eculizumab. Median hemoglobin level, reticulocytes counts and bilirubin level during the 6 months preceding PEG were 8.8 g/dL (IQR [8.25 ;9.475]), 183.3 x 109/L (IQR [142.4 ;253]), 21 mmol/L, (IQR, [15.9 ;46]),…
How strong is the evidence?
Evidence Hub reads this as a randomized trial, done in people. Its evidence level is 2 of 5 (High · randomized trial). Randomized trials of a reasonable size are the strongest single-study design. Reported size: 36 participants or records.
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