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

• Tác giả: Wilma Barcellini, Giorgia Battipaglia, Filippo Achille Brioschi, Daniela Carlino, Annalisa Condorelli, Valeria Di Giacomo, Bruno Fattizzo, Anna Paola Iori, Claudia Leoni, Sarah Marktel, Maria Antonietta Marzilli, Elisabetta Metafuni, Maurizio Miglino, Esther Natalie Oliva, Andrea Patriarca, Alessandra Ricco, Grazia Sanpaolo, Antonella Sau, Simona Sica, Annarita Trolese
• Ngôn ngữ: eng
• Ký hiệu phân loại: 347.064 Kinds of evidence
• Thông tin xuất bản: England : British journal of haematology , 2025
• Bộ sưu tập: NCBI
• ID: 548496

Tóm tắt

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.