Pegcetacoplan versus Eculizumab in Paroxysmal Nocturnal Hemoglobinuria

Anemia associated with PNH is caused by hemolysis. The C5 inhibitor eculizumab blocks intravascular hemolysis, but anemia often persists owing to extravascular hemolysis. Pegcetacoplan, an inhibitor of C3, prevents extravascular hemolysis. After 16 weeks of treatment, hemoglobin increased nearly 4 g...

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Vydané v:	The New England journal of medicine Ročník 384; číslo 11; s. 1028 - 1037
Hlavní autori:	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
Médium:	Journal Article
Jazyk:	English
Vydavateľské údaje:	United States Massachusetts Medical Society 18.03.2021
Predmet:	Adult Adverse events Aged Aged, 80 and over Anemia Antibodies, Monoclonal, Humanized - adverse effects Antibodies, Monoclonal, Humanized - therapeutic use Blood diseases Chronic illnesses Complement C3 - antagonists & inhibitors Complement C5 - antagonists & inhibitors Complement component C3 Complement component C5 Complement Inactivating Agents - adverse effects Complement Inactivating Agents - therapeutic use Complement system Diarrhea Diarrhea - chemically induced Drug Therapy, Combination Erythrocyte Transfusion Fatigue Hemoglobin Hemoglobins - analysis Hemoglobinuria, Paroxysmal - blood Hemoglobinuria, Paroxysmal - drug therapy Hemoglobinuria, Paroxysmal - therapy Hemolysis Humans Injections, Subcutaneous - adverse effects Intravenous administration L-Lactate dehydrogenase Lactic acid Meningitis Middle Aged Monoclonal antibodies Paroxysmal nocturnal hemoglobinuria Patients Peptides - adverse effects Peptides - therapeutic use Peptides, Cyclic Thrombosis
ISSN:	0028-4793, 1533-4406, 1533-4406
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Shrnutí:	Anemia associated with PNH is caused by hemolysis. The C5 inhibitor eculizumab blocks intravascular hemolysis, but anemia often persists owing to extravascular hemolysis. Pegcetacoplan, an inhibitor of C3, prevents extravascular hemolysis. After 16 weeks of treatment, hemoglobin increased nearly 4 g per deciliter in 41 patients treated with pegcetacoplan, and 85% no longer needed transfusion.
Bibliografia:	ObjectType-Article-1 SourceType-Scholarly Journals-1 ObjectType-Feature-2 content type line 14 content type line 23 ObjectType-Undefined-3
ISSN:	0028-4793 1533-4406 1533-4406
DOI:	10.1056/NEJMoa2029073