Bibliographic Details
| Title: |
Real‐world evidence of pegcetacoplan in patients with paroxysmal nocturnal haemoglobinuria: A nationwide Italian study. |
| Authors: |
Metafuni, Elisabetta1 (AUTHOR), Brioschi, Filippo Achille2 (AUTHOR), Patriarca, Andrea3 (AUTHOR), Leoni, Claudia4,5 (AUTHOR), Battipaglia, Giorgia6 (AUTHOR), Carlino, Daniela7 (AUTHOR), Condorelli, Annalisa8 (AUTHOR), Di Giacomo, Valeria9 (AUTHOR), Marktel, Sarah10 (AUTHOR), Marzilli, Maria Antonietta11 (AUTHOR), Miglino, Maurizio12 (AUTHOR), Oliva, Esther Natalie13 (AUTHOR), Sau, Antonella14 (AUTHOR), Ricco, Alessandra15 (AUTHOR), Sanpaolo, Grazia16 (AUTHOR), Trolese, Annarita17 (AUTHOR), Iori, Anna Paola18 (AUTHOR), Sica, Simona1 (AUTHOR), Barcellini, Wilma4 (AUTHOR), Fattizzo, Bruno4,5 (AUTHOR) bruno.fattizzo@unimi.it |
| Source: |
British Journal of Haematology. Apr2025, Vol. 206 Issue 4, p1246-1249. 4p. |
| Subject Terms: |
*PAROXYSMAL hemoglobinuria, *HEMOGLOBINS, *MEDICAL research, *HEMATOLOGIC agents, *ADVERSE health care events, *EMPIRICAL research, *COMPLEMENT inhibition |
| Abstract: |
The article presents a study on the real-world use of pegcetacoplan in 22 Italian patients with paroxysmal nocturnal haemoglobinuria who had suboptimal responses to anti-C5 treatments. Most patients showed significant improvements in haemoglobin levels, with a median increase of +3.6 g/dL, and experienced breakthrough haemolytic events that were mainly managed with supportive treatment. The study highlights the effectiveness of pegcetacoplan in improving haemoglobin levels and reducing transfusion requirements, with some patients experiencing adverse events leading to discontinuation of the treatment. [Extracted from the article] |
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| Database: |
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