paroxysmal nocturnal hemoglobinuria definition

3, 4, 5, 6 In the last decade, PNH has received novel and much more in-depth attention and a new kind of therapy has become available, the complement blockade by the anti-C5 monoclonal antibody eculizumab. The CD59, whose action is to inhibit the terminal complement cascade leading to the destruction of red blood cells may be more important. Two complement regulatory proteins, the CD55 (decay accelerating factor ) and CD59 (homologous restriction factor or membrane inhibitor of reactive lysis ), were found to be missing from PNH blood cells, explaining the unusual sensitivity of RBCs to the hemolytic action of complement. Granulocytes and platelets are sensitive to the complement, as well. Compared with normal RBCs, PNH RBCs lyse more readily in the presence of the activated complement. 1, 2 Hemolysis in PNH is due to the action of the complement on abnormal red blood cells (RBCs). Paroxysmal nocturnal hemoglobinuria (PNH) is a chronic, multi-systemic, progressive and life-threatening disease characterized by intravascular hemolysis, thrombotic events, serious infections and bone marrow failure. These procedures express the opinions of experts and have been based on the best available evidence and international guidelines, with the purpose of increasing benefits and reducing harm to patients. In this consensus, we are aiming to review the diagnosis and treatment of the paroxysmal nocturnal hemoglobinuria patients, as well as the early recognition of its systemic complications. The treatment that has recently become available is the complement blockade by the anti-C5 monoclonal antibody eculizumab. The traditional therapy for paroxysmal nocturnal hemoglobinuria includes blood transfusion, anti-thrombosis prophylaxis or allogeneic bone marrow transplantation. Early diagnosis, using flow cytometry performed on peripheral blood, the gold standard test to confirm the diagnosis of paroxysmal nocturnal hemoglobinuria, is essential for improved patient management and prognosis. Paroxysmal nocturnal hemoglobinuria results from the expansion of a clone of hematopoietic cells that due to an inactivating mutation of the X-linked gene PIG-A are deficient in glycosylphosphatidylinositol-linked proteins. Paroxysmal nocturnal hemoglobinuria is a chronic, multi-systemic, progressive and life-threatening disease characterized by intravascular hemolysis, thrombotic events, serious infections and bone marrow failure.

Paroxysmal nocturnal hemoglobinuria definition