Pegcetacoplan Is Superior to Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria Regardless of Prior Transfusion Requirement

Paroxysmal nocturnal hemoglobinuria and eculizumab.

Effect of eculizumab on hemolysis and transfusion requirements in patients with paroxysmal nocturnal hemoglobinuria.

BACKGROUND Paroxysmal nocturnal hemoglobinuria (PNH) arises from a somatic mutation of the PIG-A gene in a hematopoietic stem cell and the subsequent production of blood cells with a deficiency of surface proteins that protect the cells against attack by the complement system. We tested the clinical efficacy of eculizumab, a humanized antibody that inhibits the activation of terminal complement...

TRANSFUSION MEDICINE Effect of the complement inhibitor eculizumab on thromboembolism in patients with paroxysmal nocturnal hemoglobinuria

1Leeds General Infirmary, Leeds, United Kingdom; 2Radboud University Medical Center, Nijmegen, The Netherlands; 3University Essen, Essen, Germany; 4Federico II University, Naples, Italy; 5Saarland University Medical School, Homburg-Saarland, Germany; 6Istituto Toscano Tumori, Florence, Italy; 7Institute of Transfusion Medicine, University Hospital, Ulm, Germany; 8Royal Melbourne Hospital, Melbo...

The pathophysiology of paroxysmal nocturnal hemoglobinuria and treatment with eculizumab

Paroxysmal nocturnal hemoglobinuria is a rare disorder of hemopoietic stem cells. Affected individuals have a triad of clinical associations - intravascular hemolysis, an increased risk of thromboembolism, and bone marrow failure. Most of the symptoms experienced in this disease occur due to the absence of complement regulatory proteins on the surface of the red blood cells. Complement activati...

Intestinal perforation in a patient with paroxysmal nocturnal hemoglobinuria

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare hematologic disorder that manifests with hemolytic anemia, thrombosis, and peripheral blood cytopenias.Acute abdominal pain is one of the PNH clinical manifestations due to venous thrombosis of intra-abdominal sites including hepatic, portal, mesenteric, and splenic veins.Eculizumaband allogeneic bone marrow transplantation (BMT) arethe only w...