Rare Diseases, Oncohematologist Sau: “Natural history of paroxysmal nocturnal hemoglobinuria changed in 15 years thanks to new drugs”

“Paroxysmal nocturnal hemoglobinuria is a rare, chronic, serious disease, which is characterized by a symptomatic triad, consisting of severe hemolytic anemia, a propensity of patients to thrombotic events, sometimes even serious, and a bone marrow defect. In the last 10-15 years the natural history of this disease has changed thanks to the advent of specific drugs for the disease, which therefore make management easier for us doctors and for patients”. This was stated by Antonella Sau, medical director of the Pediatric Oncohematology Unit of the ASL of Pescara, on the sidelines of the 51st national congress of the Sie – Italian Society of Hematology, taking place at the MiCo in Milan from 23 to 25 September and also attended by Sobi, a biopharmaceutical company with a focus on rare or little-known hematological diseases, and oncohematology.