Pegcetacoplan for Breakthrough Hemolysis in an Adolescent With Classical Paroxysmal Nocturnal Hemoglobinuria on Eculizumab

Catherine Njeru^{1

2}, Danielle Baker¹, Hayley Merkeley^{2

3}, Ali Amid^{1

2}

Affiliations

Catherine Njeru et al. Pediatr Blood Cancer. 2025 May.

. 2025 May;72(5):e31649.

doi: 10.1002/pbc.31649. Epub 2025 Mar 4.

Catherine Njeru^{1

2}, Danielle Baker¹, Hayley Merkeley^{2

3}, Ali Amid^{1

2}

No abstract available

References

1. R. A. Brodsky, “Paroxysmal Nocturnal Hemoglobinuria,” Blood 124, no. 18 (2014): 2804–2811.
1. P. Hillmen, N. S. Young, J. Schubert, et al., “The Complement Inhibitor Eculizumab in Paroxysmal Nocturnal Hemoglobinuria,” New England Journal of Medicine 355, no. 12 (2006): 1233–1243.
1. R. A. Brodsky, N. S. Young, E. Antonioli, et al., “Multicenter Phase 3 Study of the Complement Inhibitor Eculizumab for the Treatment of Patients With Paroxysmal Nocturnal Hemoglobinuria,” Blood 111, no. 4 (2008): 1840–1847.
1. P. Hillmen, P. Muus, A. Röth, et al., “Long‐Term Safety and Efficacy of Sustained Eculizumab Treatment in Patients With Paroxysmal Nocturnal Haemoglobinuria,” British Journal of Haematology 162, no. 1 (2013): 62–73.
1. P. Hillmen, J. Szer, I. Weitz, et al., “Pegcetacoplan versus Eculizumab in Paroxysmal Nocturnal Hemoglobinuria,” New England Journal of Medicine 384, no. 11 (2021): 1028–1037. Erratum in:New England Journal of Medicine 390, no. 11 (2024): 1060.