PNH Clinical Case Review

Ilene C. Wetiz, MD, professor of clinical medicine at the Keck School of Medicine at the University of Southern California, discusses the treatment options for this case:

So, what are the treatment options? If the patient is aplastic, then you need to treat the aplasia. And you could do that with immunosuppressive therapy or bone marrow transplantation. If they have a large clone, especially that evidence of hemolysis, then you want to think about giving complement inhibition. And as we discussed in the earlier slides, eculizumab [Soliris, Alexion] and ravulizumab [Ultomiris, Alexion], and now pegcetacoplan APL-2, Apellis Pharmaceuticals], are approved.