Palliative Therapies for PNH

Blood transfusions: useful for temporarily decreasing the symptoms associated with anemia; however, iron overload can be an issue.¹

Corticosteroids: while effective in some patients with PNH to temporarily improve hemoglobin levels and reduce hemolysis, their use is limited by their side effect profile.¹

Anticoagulants: their role in prophylaxis remains unclear; it has never been proven in a controlled trial to prevent thrombosis in patients with PNH. Many patients with PNH experience thrombocytopenia and can be at risk for hemorrhage, so the use of anticoagulant therapy may be contraindicated in these patients.^1,2

Androgen therapy: has been used historically to treat anemia but has been reserved primarily for patients with some degree of bone marrow hypoplasia. Androgens offer rapid onset of action but are associated with virilizing effects in women and prostatism in men. Some data have also suggested an association between androgens and the development of Budd-Chiari syndrome in patients with PNH.¹

Supplements such as folic acid and iron: these can be administered to support erythropoiesis.¹

Next: Bone Marrow Transplantation