[Stem cell transplantation for paroxysmal nocturnal hemoglobinuria]

Paroxysmal nocturnal hemoglobinuria (PNH) represents a rare clonal disorder of hematopoiesis clinically characterized by acquired hemolytic anemia, intravascular hemolysis, hemoglobinuria and frequent occurrence of venous thrombosis. Stem cell transplantation is indicated in patients with severe bone marrow aplasia, repeated massive hemolysis or recurrent life threatening thrombotic complications. Almost 90 transplanted patients with PNH have been published. We report a case of successful allogeneic peripheral blood stem cell transplantation performed in a 24 years old woman with a severe form of PNH with frequent episodes of massive intravascular hemolysis. The patient is now alive completely engrafted 900 days after transplantation without signs of chronic GVHD and without recurrent infections. This case represents the first successfully transplanted patient with PNH in our country.