Details

Autor(en) / Beteiligte

• Hillmen, Peter
• Hall, Claire
• Marsh, Judith C.W
• Elebute, Modupe
• Bombara, Michael P
• Petro, Beth E
• Cullen, Matthew J
• Richards, Stephen J
• Rollins, Scott A
• Mojcik, Christopher F
• Rother, Russell P

Titel

Effect of Eculizumab on Hemolysis and Transfusion Requirements in Patients with Paroxysmal Nocturnal Hemoglobinuria

Ist Teil von

• The New England journal of medicine, 2004-02, Vol.350 (6), p.552-559

Ort / Verlag

Boston, MA: Massachusetts Medical Society

Erscheinungsjahr

2004

Quelle

MEDLINE

Beschreibungen/Notizen

• The cause of the hemolytic anemia of paroxysmal nocturnal hemoglobinuria (PNH) is excessive susceptibility of erythrocytes to the lytic effects of the membrane-attack complex of the complement system. Cleavage of C5 initiates assembly of this complex. Eculizumab, a humanized antibody that blocks cleavage of C5, reduces signs of hemolytic anemia and transfusion requirements in patients with PNH. A study of a humanized antibody that blocks cleavage of C5. The major clinical signs of paroxysmal nocturnal hemoglobinuria (PNH) are intravascular hemolysis, venous thrombosis, and hemoglobinuria. 1 The disease arises from a somatic mutation of the PIG-A gene in a pluripotent hematopoietic stem cell. PIG-A encodes a protein that is essential for the synthesis of glycosylphosphatidylinositol (GPI), a lipid moiety that is embedded in the plasma membrane, where it serves to anchor a wide variety of proteins to the cell surface. The mutant stem cell subsequently expands to form a hematopoietic clone with a deficiency in proteins that are normally attached to the cell surface by the GPI anchor. 2 , 3 The . . .