Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired disease characterized by chronic complement-mediated hemolysis. C5 inhibition controls intravascular hemolysis in untreated PNH but cannot address extravascular hemolysis. Pegcetacoplan, a pegylated peptide targeting proximal complement protein C3, potentially inhibits both intravascular and extravascular hemolysis.

Pegcetacoplan versus Eculizumab in Paroxysmal Nocturnal Hemoglobinuria / Hillmen, P., Szer, J., Weitz, I., Röth, A., Höchsmann, B., Panse, J., Usuki, K., Griffin, M., Kiladjian, J., de Castro, C., Nishimori, H., Tan, L., Hamdani, M., Deschatelets, P., Francois, C., Grossi, F., Ajayi, T., Risitano, A., de la Tour, R.P.. - In: THE NEW ENGLAND JOURNAL OF MEDICINE. - ISSN 0028-4793. - 384:11(2021), pp. 1028-1037-1037. [10.1056/NEJMoa2029073]

Pegcetacoplan versus Eculizumab in Paroxysmal Nocturnal Hemoglobinuria

Risitano, Antonio;
2021

Abstract

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired disease characterized by chronic complement-mediated hemolysis. C5 inhibition controls intravascular hemolysis in untreated PNH but cannot address extravascular hemolysis. Pegcetacoplan, a pegylated peptide targeting proximal complement protein C3, potentially inhibits both intravascular and extravascular hemolysis.
2021
Pegcetacoplan versus Eculizumab in Paroxysmal Nocturnal Hemoglobinuria / Hillmen, P., Szer, J., Weitz, I., Röth, A., Höchsmann, B., Panse, J., Usuki, K., Griffin, M., Kiladjian, J., de Castro, C., Nishimori, H., Tan, L., Hamdani, M., Deschatelets, P., Francois, C., Grossi, F., Ajayi, T., Risitano, A., de la Tour, R.P.. - In: THE NEW ENGLAND JOURNAL OF MEDICINE. - ISSN 0028-4793. - 384:11(2021), pp. 1028-1037-1037. [10.1056/NEJMoa2029073]
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/11588/847919
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