![]() 6 More recently, data from France reported a median survival of 22 years. 5 Prior to eculizumab, treatment was mainly supportive in nature with a median survival of 10 to 15 years for patients treated between 19. The red blood cells derived from these abnormal HSCs are extremely sensitive to complement mediated lysis which leads to many of the symptoms of the disease. ![]() 2– 4 PNH is a rare hematological disorder where hematological stem cells (HSCs) acquire an abnormality that is then passed on to their progeny. ![]() It has been shown to be a well tolerated and highly effective treatment for patients with PNH. 1 It was licensed by the Food and Drug Administration in March 2007 and by the European Medicines Agency in June 2007 for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). It is not a curative therapy but has a great benefit on those with this rare debilitating condition.Įculizumab is a humanized monoclonal antibody which binds to the complement protein 5 (C5), thereby inhibiting the formation of the terminal components of the complement cascade. Eculizumab prevents intravascular hemolysis, stabilizes hemoglobin levels, reduces or stops the need for blood transfusions, and improves fatigue and patient quality of life as well as reducing pulmonary hypertension, decreasing the risk of thrombosis and protecting against worsening renal function. Eculizumab is a monoclonal antibody treatment which specifically binds to the complement protein C5, preventing its cleavage, and so halts the complement cascade and prevents the formation of the terminal complement proteins. Complement activation is thus not checked and causes destruction of these cells. Most of the symptoms experienced in this disease occur due to the absence of complement regulatory proteins on the surface of the red blood cells. Affected individuals have a triad of clinical associations – intravascular hemolysis, an increased risk of thromboembolism, and bone marrow failure. Paroxysmal nocturnal hemoglobinuria is a rare disorder of hemopoietic stem cells.
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