title: [Eculizumab in paroxysmal nocturnal hemoglobinuria].
abstract: Paroxysmal nocturnal hemoglobinuria is a rare acquired clonal of the hematopoietic stem cell due to acquired mutation of the PIG-A gene. This results in the lack of two GPI-anchored membrane proteins involved in the inhibition of complement attack, thus explaining red cells hemolysis. The development of an anti-C5 monoclonal antibody (eculizumab) had profoundly modified the treatment of the the hemolytic form of the disease.
authors.affiliation: Service d'hématologie-greffe, Inserm U728, Hôpital Saint-Louis, AP-HP, 1, avenue Claude Vellefaux, 75010 Paris, France. gerard.socie@sls.aphp.fr
authors.forename: Gérard
authors.initials: G
authors.lastname: Socié
authors.forename: Nathalie
authors.initials: N
authors.lastname: Varoqueaux
authors.forename: Régis
authors.initials: R
authors.lastname: Peffault de Latour
mesh_terms: D000911:Antibodies, Monoclonal / Q000627:therapeutic use*; D061067:Antibodies, Monoclonal, Humanized; D016026:Bone Marrow Transplantation; D018958:CD55 Antigens / Q000502:physiology; D018957:CD59 Antigens / Q000502:physiology; D002986:Clinical Trials as Topic; D003167:Complement Activation; D003182:Complement C5 / Q000037:antagonists & inhibitors* / Q000276:immunology / Q000502:physiology; D017261:Glycosylphosphatidylinositols / Q000502:physiology; D006457:Hemoglobinuria, Paroxysmal / Q000175:diagnosis / Q000188:drug therapy* / Q000235:genetics / Q000276:immunology / Q000601:surgery; D006461:Hemolysis; D006801:Humans; D008565:Membrane Proteins / Q000172:deficiency / Q000235:genetics; D008954:Models, Biological; D014184:Transplantation, Homologous; D055815:Young Adult
chemical_list: D000911:Antibodies, Monoclonal; D061067:Antibodies, Monoclonal, Humanized; D018958:CD55 Antigens; D018957:CD59 Antigens; D003182:Complement C5; D017261:Glycosylphosphatidylinositols; D008565:Membrane Proteins; C080086:phosphatidylinositol glycan-class A protein; C051590:CD59 protein, human; C481642:eculizumab