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, Peter; Szer, Jeff; Weitz, Ilene; Röth, Alexander; Höchsmann, Britta; Panse, Jens; Usuki, Kensuke; Griffin, Morag; Kiladjian, Jean-Jacques; de Castro, Carlos; Nishimori, Hisakazu; Tan, Lisa; Hamdani, Mohamed; Deschatelets, Pascal; Francois, Cedric; Grossi, Federico; Ajayi, Temitayo; Risitano, Antonio; de la Tour, Régis Peffault. - 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, Peter; Szer, Jeff; Weitz, Ilene; Röth, Alexander; Höchsmann, Britta; Panse, Jens; Usuki, Kensuke; Griffin, Morag; Kiladjian, Jean-Jacques; de Castro, Carlos; Nishimori, Hisakazu; Tan, Lisa; Hamdani, Mohamed; Deschatelets, Pascal; Francois, Cedric; Grossi, Federico; Ajayi, Temitayo; Risitano, Antonio; de la Tour, Régis Peffault. - In: THE NEW ENGLAND JOURNAL OF MEDICINE. - ISSN 0028-4793. - 384:11(2021), pp. 1028-1037-1037. [10.1056/NEJMoa2029073]
File in questo prodotto:
Non ci sono file associati a questo prodotto.

I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.

Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/11588/847919
Citazioni
  • ???jsp.display-item.citation.pmc??? ND
  • Scopus 171
  • ???jsp.display-item.citation.isi??? 163
social impact