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Eculizumab for Paroxysmal Nocturnal Hemoglobinuria: How does it work? How does PNH happen? Is it safe? How is it administered? How often? Side effects?

Paroxysmal Nocturnal Hemoglobinuria is a very rare, and life-threatening condition of the blood wherein your body destroys red blood cells (the cells which give you oxygen.) It is characterized by the hemolysis (aka the breakdown of those cells), thrombosis (clots of blood in your veins or arteries), and even impaired bone marrow function. Eculizumab is one of the most useful medicines for PNH, because of the significant improvements it yields in patients with the condition. 

PNH is an acquired disorder, which results from a mutation in a specific gene called the PIGA gene. This gene is responsible for the synthesis of proteins which protect your red blood cells from getting destroyed by your immune system. When these proteins are not produced because of the PIGA gene mutation, you acquire the symptoms of PNH. 

Symptoms of PNH include but are not limited to: dark urine, especially at night or in the morning, since hemoglobin is being released into it; blood clots causing thrombosis or even heart attacks; anemia, fatigue, increased infections due to a lack of proper bone marrow function. 

Eculizumab is a monoclonal antibody. It basically binds onto the part of the red blood cell that your immune system destroys, and acts kind of like a blocked door (there's no way for the RBC to get destroyed since its attack point is covered.) The specific part that eculizumab bonds onto, is called the C5 protein (the protein which makes the attack point eventually)

Eculizumab is administered through an IV. Usually, it is given in doses of 600mg per week for a month, and then spread out to 900mg every two weeks. Its effect on the body is heavily monitored, through analysis of hemolysis markers that the doctor does. They check enzyme levels, blood counts, and kidney function. 

However, it is important to note that not all people can take eculizumab. This especially includes people who are prone to meningococcal infections. Eculizumab increases the risk of infections. Most people are advised to get vaccinated against Neisseria meningitidis two weeks before they begin with an eculizumab regimen. 

Side effects of the medicine include: headache, fever, nausea, and fatigue after the IV is removed; the higher risk of infections as discussed above; back pain, and diarrhea for a few days after the IV. 


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