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 selected treatment and the results of the case:

So, what happened to this patient? Well, we started her on a C5 inhibitor because she had a very large PNH clone. You can see on the graphs that are attached. So, the one on the bottom is her hemoglobin. This is without transfusion. She actually became a Jehovah's Witness in the interim since her initial presentation. So, you could see her hemoglobin, and you could see on its own, she recovered. And she runs a hemoglobin now of around 9 to 10 [mg/dL]. Her LDH dropped dramatically.

And you could take this curve and superimpose it on the clinical trial curves. It’s exactly the same — very reproducible. And here you can see her platelet count, which very interestingly enough started at 107,000[/µL]and then completely normalized. So, in some patients, the thrombocytopenia is related to a consumptive coagulopathy that they have and consumption. And you could see recovery of the cytopenias may occur with complement inhibition. Pancytopenia and PNH may be independent of AA and, again, may be due to the effect of complement on the PNH cells.