Ten Years of Clinical Experience With Eculizumab in Patients With Paroxysmal Nocturnal Hemoglobinuria
Paroxysmal nocturnal hemoglobinuria (PNH) arises from a somatic mutation in the phosphatidylinositol glycan class A, X-linked gene, responsible for a deficiency in glycosyl phosphatidylinositol-anchored proteins. The absence of one of the glycosyl phosphatidylinositol-anchored protein complement regulatory proteins (CD59) leads to hemolysis. Clinical manifestations include chronic hemolysis, thromboembolic disease, infectious complications, chronic kidney injury, pulmonary hypertension, and smooth muscle dysfunction.
Source: Seminars in Hematology - Category: Hematology Authors: Flore Sicre de Fontbrune, R égis Peffault de Latour Tags: Research Article Source Type: research