Dr. Babushok on Treatment Advances in Paroxysmal Nocturnal Hemoglobinuria

Daria V. Babushok, MD, PhD, an assistant professor of medicine, Perelman School of Medicine, University of Pennsylvania, discusses treatment advances in paroxysmal nocturnal hemoglobinuria (PNH).

PNH is an extremely rare blood disorder that can be diagnosed in the presence of a PNH clone on flow cytometry, says Babushok. The treatment decision is guided by the size of the PNH clone. If the patient has significant hemolysis and a very large clone, they are at risk for significant hemolytic anemia and thrombotic events. As such, the patient should be treated with complement inhibitors, says Babushok.

There are 2 FDA-approved complement inhibitors, one is eculizumab (Soliris) and the other is ravulizumab-cwvz (Ultomiris), adds Babushok. These drugs are being compared head-to-head in the newly diagnosed setting, as well as in previously treated patients. Although the drugs are very similar, the half-life of ravulizumab is approximately three times higher than that of eculizumab. Moreover, the agents are given on different schedules. Eculizumab is given every 2 weeks, whereas ravulizumab is given every 2 months. Notably, both agents require immunization with the meningococcal vaccine as well as for all encapsulated organisms, concludes Babushok.