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 PNH with thrombosis:

"How do we treat this? Well, for the acute thrombosis, anticoagulation. In this case, we wanted to lyse the clots, but because she had an abdominal wall hematoma, the interventionalist did not want to attempt that. But she was placed on anticoagulation. And for the PNH, the options for treatment are best supportive care with transfusions, iron and folate. But in the modern era where there’s clear evidence of efficacy, you would want to use a complement inhibitor, especially a C5 inhibitor, which is what we used in this case. And there are two C5 inhibitors that are approved — eculizumab [Soliris, Alexion], which is every 2 weeks, ravulizumab [Ultomiris, Alexion], which is every 7 to 8 weeks. And there’s also an approved C3 inhibitor that’s a SubQ injection biweekly."