Treatment-Paroxysmal nocturnal hemoglobinuria

• supportive care : iron, folate, transfusions

• anticoagulation.

• Allogeneic HSCT for hypoplasia or severe thrombosis.

• Pegcetacoplan (binds C3, prevents complement cascade activation)

• superior to eculizumab (Ab ❌ terminal complement C5s)

• in ↓ hemolysis & stabilizing Hb levels (NEJM 2021;384:1028).

• Eculizumab in pregnancy (NEJM 2015;373:1032);

• meningococcal vaccination 1️⃣