Real‐world evidence of pegcetacoplan in patients with paroxysmal nocturnal haemoglobinuria: A nationwide Italian study

• Published in: British journal of haematology Vol. 206; no. 4; pp. 1246 - 1249
• Main Authors: Metafuni, Elisabetta, Brioschi, Filippo Achille, Patriarca, Andrea, Leoni, Claudia, Battipaglia, Giorgia, Carlino, Daniela, Condorelli, Annalisa, Di Giacomo, Valeria, Marktel, Sarah, Marzilli, Maria Antonietta, Miglino, Maurizio, Oliva, Esther Natalie, Sau, Antonella, Ricco, Alessandra, Sanpaolo, Grazia, Trolese, Annarita, Iori, Anna Paola, Sica, Simona, Barcellini, Wilma, Fattizzo, Bruno
• Format: Journal Article
• Language: English
• Published: England Blackwell Publishing Ltd 01.04.2025
• Subjects: Adult; Aged; Antibodies, Monoclonal, Humanized - administration & dosage; Antibodies, Monoclonal, Humanized - adverse effects; Antibodies, Monoclonal, Humanized - therapeutic use; Bone marrow transplantation; Female; Hemoglobin; Hemoglobinuria, Paroxysmal - blood; Hemoglobinuria, Paroxysmal - drug therapy; Hemoglobinuria, Paroxysmal - epidemiology; Humans; Italy - epidemiology; Male; Middle Aged; Italy
• ISSN: 0007-1048; 1365-2141; 1365-2141
• DOI: 10.1111/bjh.20025

In this study, we collected real‐world evidence on the use of pegcetacoplan among 22 Italian patients with paroxysmal nocturnal haemoglobinuria showing suboptimal response to anti‐C5 treatments eculizumab and ravulizumab. Most patients exhibited a complete or good response as per the criteria of the European Bone Marrow Transplant group (Risitano et al. Front Immunol 2019) and median haemoglobin improvement from baseline was +3.6 g/dL. During the 6‐month follow‐up, 27% of patients displayed a breakthrough haemolytic event mainly managed with supportive treatment. No thromboses occurred.