: 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.
Real‐world evidence of pegcetacoplan in patients with paroxysmal nocturnal haemoglobinuria: A nationwide Italian study / 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. - In: BRITISH JOURNAL OF HAEMATOLOGY. - ISSN 0007-1048. - 206:4(2025), pp. 1246-1249. [10.1111/bjh.20025]
Real‐world evidence of pegcetacoplan in patients with paroxysmal nocturnal haemoglobinuria: A nationwide Italian study
Battipaglia, Giorgia;
2025
Abstract
: 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.I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.
