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. 2025 Apr;206(4):1246-1249.
doi: 10.1111/bjh.20025. Epub 2025 Feb 22.

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

Elisabetta Metafuni  1 , Filippo Achille Brioschi  2 , Andrea Patriarca  3 , Claudia Leoni  4   5 , Giorgia Battipaglia  6 , Daniela Carlino  7 , Annalisa Condorelli  8 , Valeria Di Giacomo  9 , Sarah Marktel  10 , Maria Antonietta Marzilli  11 , Maurizio Miglino  12 , Esther Natalie Oliva  13 , Antonella Sau  14 , Alessandra Ricco  15 , Grazia Sanpaolo  16 , Annarita Trolese  17 , Anna Paola Iori  18 , Simona Sica  1 , Wilma Barcellini  4 , Bruno Fattizzo  4   5
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Real-world evidence of pegcetacoplan in patients with paroxysmal nocturnal haemoglobinuria: A nationwide Italian study

Elisabetta Metafuni et al. Br J Haematol. 2025 Apr.

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.

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References

REFERENCES

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