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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Published in:The New England journal of medicine Vol. 384; no. 11; pp. 1028 - 1037
Main Authors: 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
Format: Journal Article
Language:English
Published: United States Massachusetts Medical Society 18.03.2021
Subjects:
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
Online Access:Get full text
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Summary: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.
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ISSN:0028-4793
1533-4406
1533-4406
DOI:10.1056/NEJMoa2029073