Low Rate of Clinically Evident Extravascular Hemolysis in Patients with Paroxysmal Nocturnal Hemoglobinuria Treated with a Complement C5 Inhibitor: Results from a Large, Multicenter, US Real-World Study

Purpose: Most patients with paroxysmal nocturnal hemoglobinuria (PNH) treated with a complement protein 5 (C5) inhibitor achieve full control of terminal complement activity and intravascular hemolysis. The minority remains anemic and transfusion dependent despite this control. Etiology for ongoing...

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Veröffentlicht in:Journal of blood medicine 2022-01, Vol.13, p.425-437
Hauptverfasser: Shammo, Jamile, Gajra, Ajeet, Patel, Yogesh, Tomazos, Ioannis, Kish, Jonathan, Hill, Anita, Sierra, J. Rafael, Araten, David
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Sprache:eng
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Zusammenfassung:Purpose: Most patients with paroxysmal nocturnal hemoglobinuria (PNH) treated with a complement protein 5 (C5) inhibitor achieve full control of terminal complement activity and intravascular hemolysis. The minority remains anemic and transfusion dependent despite this control. Etiology for ongoing anemia is multifactorial and includes bone marrow failure, breakthrough hemolysis, extravascular hemolysis (EVH) and nutritional deficiencies. Patients and Methods: To evaluate the potential etiologies of hemoglobin levels
ISSN:1179-2736
1179-2736
DOI:10.2147/JBM.S361863