C3 is a key molecule in this system whose activation triggers most of the downstream effects. Upon activation, complement proteins conduct immune activity against invading organisms or foreign materials. Instead, research into the pathogenesis of PNH has revealed that the disease arises from chronic and uncontrolled complement activation, causing dysfunction in all 3 blood cell lines: red blood cells, white blood cells (both granulocytes and monocytes), and platelets.Ī component of the immune system, the complement system is composed of more than 30 proteins generated in the liver and present in the blood circulation and within body tissues. 3,4 These conditions are not solely explained by either anemia or hemolysis. Frequent manifestations of PNH include smooth muscle dystonias, chronic kidney disease, and thrombosis. In fact, it is now known that PNH has more profound consequences. 2 Contributing to the confusion surrounding PNH is the name itself, which seems to suggest that the condition is limited to hemolysis and anemia. 1 This therapeutic advance, with the consequent improved clinical outcomes, has now made it important to recognize and diagnose these patients in an effective and timely fashion.Īlthough PNH has been recognized for more than a century, a deeper understanding of the underlying biology of the disease has only recently become available. However, with better understanding of the pathophysiology of PNH, the management of these patients has now progressed beyond just supportive care to a very specific targeted treatment of the underlying disease process of complement-mediated hemolysis. As such, early diagnosis was generally not considered to be a compelling issue. Until recently, supportive and symptomatic treatment was all that could be offered to these patients. Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired clonal hematopoietic stem cell disorder that results in complement-mediated hemolysis arising from the affected stem cell population, which in turn produces severe and even potentially fatal clinical consequences. Recent advances in treatment have emphasized the importance of early diagnosis. The gold standard test for PNH diagnosis consists of high-sensitivity flow cytometry performed on a peripheral blood sample. There are 3 broad patient categories in whom the incidence of PNH is significantly greater as compared with the general population: patients with hemolysis or hemoglobinuria, patients with bone marrow failure syndromes, and patients with unexplained or unusual thrombosis. Patients may present with a variety of clinical manifestations, such as anemia, thrombosis, kidney disease, smooth muscle dystonias, abdominal pain, dyspnea, and extreme fatigue.
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The clinical features of this disease arise as a result of complement-mediated hemolysis in unprotected red cells, leukocytes, and platelets as well as the release of free hemoglobin that occurs with erythrocyte destruction. Paroxysmal Nocturnal Hemoglobinuria: Pathogenesis, Testing, and DiagnosisĪbstract: Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired clonal hematopoietic stem cell disorder with far-reaching multisystem effects that can lead to life-threatening consequences. All rights reserved, including the right of reproduction, in whole or in part, in any form. ©2013 Millennium Medical Publishing, Inc., 611 Broadway, Suite 310, New York, NY 10012. No claims or endorsements are made for any drug or compound at present under clinical investigation. Readers are strongly urged to consult any relevant primary literature.
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Volume 11, Issue 9, Supplement 13 September 2013 Paroxysmal Nocturnal Hemoglobinuria: Pathogenesis, Testing, and Diagnosis Plus a Case Report and a Q&A on the Role of the Hematopathologist
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