Paroxysmal nocturnal haemoglobinuria treatment with eculizumab is associated with a positive direct antiglobulin test

• Published in: Vox sanguinis 2012-02, Vol.102 (2), p.159-166
• Main Authors: Höchsmann, B., Leichtle, R., von Zabern, I., Kaiser, S., Flegel, W. A., Schrezenmeier, H.
• Format: Article
• Language: English
• Subjects: Adolescent; Adult; Aged; Antibodies, Anti-Idiotypic - immunology; Antibodies, Monoclonal, Humanized - therapeutic use; Child; direct antiglobulin test positive by C3d; Drug therapy; eculizumab; Female; Ferritins - blood; Ferritins - metabolism; Hemoglobinuria, Paroxysmal - blood; Hemoglobinuria, Paroxysmal - drug therapy; Hemoglobinuria, Paroxysmal - enzymology; Hemoglobinuria, Paroxysmal - immunology; Humans; L-Lactate Dehydrogenase - blood; L-Lactate Dehydrogenase - metabolism; Male; Middle Aged; paroxysmal nocturnal haemoglobinuria; Retrospective Studies; Tests; Young Adult
• ISSN: 0042-9007; 1423-0410
• DOI: 10.1111/j.1423-0410.2011.01530.x

Background/Objectives  Paroxysmal nocturnal haemoglobinuria (PNH) is characterized by intravascular haemolysis with a negative direct antiglobulin test (DAT). Eculizumab is a humanized monoclonal antibody that inhibits complement component C5 and is approved for PNH treatment. Recent publications demonstrated that some patients with PNH develop a positive DAT during eculizumab treatment. These published clinical trials investigated a highly selected patient population. Therefore, it seems important to study this topic in a general PNH patient population with a longer follow‐up. Materials and Methods  We analysed haemolytic activity, RBC transfusion requirement, effect on DAT and ferritin levels in 41 patients with PNH before and during eculizumab therapy with a median follow‐up of 24 months (range 1–63 months). Results  During eculizumab therapy, median LDH decreased (1657–258 U/l; P