Bone Marrow Failure Disorders

As the authors note, "paroxysmal nocturnal hemoglobinuria (PNH) remains the prototype model of complement activation and inhibition." The authors review inhibitors of complement C5 (eculizumab and ravulizumab) and complement C3 (pegcetacoplan) approved for use in PNH, as well factor B and factor D inhibitors, novel agents under investigation that appear promising in this patient population.