Pathophysiology,diagnosis, and treatment of paroxysmal nocturnal hemoglobinuria: a review |
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| Authors: | Bérangère Devalet François Mullier Bernard Chatelain Jean‐Michel Dogné Christian Chatelain |
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| Affiliation: | 1. Department of Hematology, Namur Thrombosis and Hemostasis Center (NTHC), CHU Dinant‐Godinne UCL Namur, Yvoir, Belgium;2. Hematology Laboratory, Namur Thrombosis and Hemostasis Center (NTHC), CHU Dinant‐Godinne UCL Namur, Yvoir, Belgium;3. Department of Pharmacy, Namur Thrombosis and Hemostasis Center (NTHC), University of Namur, Namur, Belgium |
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| Abstract: | Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired disorder of the hematopoietic stem cell that makes blood cells more sensitive to the action of complement. Patients experience intravascular hemolysis, smooth muscle dystonia, renal failure, arterial and pulmonary hypertension, recurrent infectious diseases and an increased risk of notably dreadful thrombotic complications. The diagnosis is made by flow cytometry. Efforts have been recently performed to improve the sensitivity and the standardization of this technique. PNH is frequently associated with aplastic anemia or low‐risk myelodysplasia and may be asymptomatic. Management of the classical form of PNH has been dramatically revolutionized by the development of eculizumab, which brings benefits in terms of hemolysis, quality of life, renal function, thrombotic risk, and life expectancy. Prophylaxis and treatment of arterial and venous thrombosis currently remain a challenge in PNH. |
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| Keywords: | hemoglobinuria hemolysis thrombosis eculizumab anticoagulation |
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