Acute attacks There is disagreement as to whether steroids (such as prednisolone) can decrease the severity of hemolytic crises. Transfusion therapy may be needed; in addition to correcting significant anemia, this suppresses the production of<unk>  cells by the bone marrow, and indirectly the severity of the hemolysis. Iron deficiency develops with time, due to losses in urine, and may have to be treated if present. Iron therapy can result in more hemolysis as more<unk>  cells are produced. Long-term<unk>  is a chronic condition. In patients with only a small clone and few problems, monitoring of the flow cytometry every six months gives information on the severity and risk of potential complications. Given the high risk of thrombosis in<unk> , preventive treatment with warfarin decreases the risk of thrombosis in those with a large clone (50% of white blood cells type III). Episodes of thrombosis are treated as they would in other patients, but, given that<unk>  is a persisting underlying cause, it is likely that treatment with warfarin or similar drugs needs to be continued long-term after an episode of thrombosis. Eculizumab In 2007, the drug eculizumab was approved for the treatment of<unk> . Prior to eculizumab, the median life expectancy of an individual with<unk>  was approximately 10 years. Since that time, short and mid-term studies of patients on eculizumab demonstrate that the drug returns the patient to a normal life expectancy, improves quality of life, and decreases the need for blood transfusions. Eculizumab is controversial due to its high cost, as it is among the most expensive pharmaceuticals in the world, with a price of US$440,000 per person per year. Eculizumab is a humanized monoclonal antibody that acts as a terminal complement inhibitor. The drug interferes with the formation of the membrane attack complex in erythrocytes by binding to C5, compensating for the loss of protective function that results from CD59 deficiency. This alleviates the primary source of intravascular hemolysis, but it does not reduce the opsonization of erythrocytes caused by CD55 deficiency, so patients who receive this medication often still experience mild to moderate hemolysis. The U.S. Food and Drug Administration (FDA) has issued a black-box warning as those who take the medication have a 1,000 to 2,000-fold greater risk of invasive meningococcal disease. People on eculizumab are strongly advised to receive meningococcal vaccination at least two weeks prior to starting therapy and to consider preventative antibiotics for the duration of treatment. Pegcetacoplan Pegcetacoplan was approved for medical use in the United States in May 2021.
Paroxysmal nocturnal hemoglobinuria
Paroxysmal cold hemoglobinuria
Hereditary spherocytosis
Beta thalassemia
Congenital hemolytic anemia
Hemoglobinemia
Alpha-thalassemia
Congenital dyserythropoietic anemia type II