In the era of eculizumab, although the complications such as hemolysis and thrombosis can be taken under control, cure and the remission of the disease is stil a rare entity. Įculizumab, a complement inhibitor, is a monoclonal antibody which decreases risk of thrombosis and hemolysis in patients with PNH by inhibiting complement system. A retrospective analysis consisted of 80 patients, and 12 patients had spontaneous clinical remission. During the course of the disease, spontaneous remission is a rare entity. Other causes of death are hemorrhage, renal and cardiac problems which can be related to thrombosis and intravascular hemolysis, infections, transformation to myelodysplastic syndrome or aplastic anemia. One of the most common causes of mortality and morbidity in PNH patients is thromboembolism. Myelodysplastic syndrome and acute leukemias can also be seen in the course of the disease. Hemolysis, bone marrow failure with cytopenias and life-threatening thrombotic episodes are the most common clinical manifestations. The clinical manifestations and presenting symptoms are diverse at the presentation and during the course of the disease due to the effected organs and systems. Especially the absence of CD55 (decay accelerating factor) and CD59 (membrane inhibitory of reactive lysis) proteins which are linked to the membrane by GPI anchor proteins leads to intravascular hemolysis, generation of inflammatory mediators and the other symptoms of the disease. As a result, increased sensitivity of red blood cells to complement system, intravascular hemolysis and generation of inflammatory mediators occur. It results in partial or complete absence of certain GPI-linked proteins. Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired clonal disorder of the hematopoietic stem cells caused by a mutation in X-linked phosphatidylinositol glycan class A (PIG-A) gene. Keywords: Paroxysmal nocturnal hemoglobinuria Spontaneous remission Eculizumab Introduction We, hereby, report a case whose disease undergone spontaneous remission under treatment of eculizumab treatment. But eculizumab does not have any impact on, or may increase, clone status and nature of the disease. Eculizumab, which is a monoclonal antibody, has been established as the effective approach not only for prevention of hemolysis, but also for prevention of potential organ damages such as thromboses and failure of target organs. The only curative treatment option for PNH has been still allogeneic stem cell transplantation from HLA-matched donor. Paroxysmal nocturnal hemoglobinuria (PNH) is a very rare acquired disease which is characterized by increased sensitivity to complement proteins.
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