How I treat How I treat paroxysmal nocturnal hemoglobinuria
نویسنده
چکیده
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare clonal blood disorder that manifests with hemolytic anemia, bone marrow failure, and thrombosis. Many of the clinical manifestations of the disease result from complement-mediated intravascular hemolysis. Allogeneic bone marrow transplantation is the only curative therapy for PNH. Eculizumab, a monoclonal antibody that blocks terminal complement activation, is highly effective in reducing hemolysis, improving quality of life, and reducing the risk for thrombosis in PNH patients. Insights into the relevance of detecting PNH cells in PNH and other bone marrow failure disorders are highlighted, and indications for treating PNH patients with bone marrow transplantation and eculizumab are explored. (Blood. 2009;113:6522-6527)
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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...
متن کاملSuccessful use of eculizumab in an 86-year-old patient with paroxysmal nocturnal hemoglobinuria in Japan
Eculizumab was used to treat an 86-year-old male patient with paroxysmal nocturnal hemoglobinuria, the oldest reported case in Japan. As observed in younger patients, this drug rapidly suppressed hemolysis in the present patient, which allowed weaning from blood transfusion. Eculizumab treatment has been continued for 2 years and resulted in the alleviation of renal dysfunction. Despite the pat...
متن کاملImmune lysis of normal human and paroxysmal nocturnal hemoglobinuria (PNH) red blood cells. I. The sensitivity of PNH red cells to lysis by complement and specific antibody.
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Long Standing Eculizumab Treatment without Anticoagulant Therapy in High-Risk Thrombogenic Paroxysmal Nocturnal Hemoglobinuria
Paroxysmal nocturnal hemoglobinuria (PNH) is an ultra-orphan disease affecting all hematopoietic cell types. The abnormality of red blood cells in this disease predisposes to intravascular complement-mediated hemolysis. Eculizumab is an orphan drug used to treat this rare disease. Thrombosis is the key cause of death in PNH patients in about 40% to 67% of cases. We report the case of a woman pr...
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Leebeek FW. Myeloproliferative disease in the pathogenesis and survival of Budd-Chiari syndrome. Haematologica 2006;91: 1712-3. 3. Valla D, Dhumeaux D, Babany G, et al. Hepatic vein thrombosis in paroxysmal nocturnal hemoglobinuria. A spectrum from asymptomatic occlusion of hepatic venules to fatal Budd-Chiari syndrome. Gastroenterology 1987;93:569-75. 4. Hillmen P, Lewis SM, Bessler M, Luzzatt...
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