Several complement inhibitors have been approved for paroxysmal nocturnal hemoglobinuria (PNH), including pegcetacoplan, eculizumab, r**ulizumab, and iptacopan. These drugs reduce hemolysis and its sequelae associated with PNH by targeting different complement factors.
Complement inhibitors have been shown to be effective in relieving PNH symptoms and improving quality of life, eliminating associated complications. However, they do not change the underlying defects of the disease and therefore need to be sustained**.
Allogeneic hematopoietic stem cell transplantation is a method that can be used to minimize PNH, but it requires finding a histologically compatible donor and is associated with significant morbidity and mortality. Therefore, this ** method is usually reserved for severe cases, such as cases of PNH with aplastic anemia or transformation into leukemia.
In addition to medications and transplant surgery, patient education and lifestyle modifications are also an important part of PNH. Patients should maintain good lifestyle habits, including adequate rest, a balanced diet, and appropriate exercise. At the same time, avoiding factors that may induce hemolysis, such as infections and certain medications, is also key.
Overall, paroxysmal nocturnal hemoglobinuria (PNH) is a rare and complex condition that requires a comprehensive ** and management. Morbidity and mortality can be reduced and patients' quality of life can be improved through reasonable morbidity and patient education.