ISTH 2023 | The value of pegcetacoplan in the PNH treatment landscape
Pranavi Sanka, MD, The Warren Alpert Medical School of Brown University, Providence, RI, briefly discusses the value of pegcetacoplan in the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH). This interview took place at the 31st Congress of the International Society on Thrombosis and Haemostasis (ISTH), held in Montréal, Canada.
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Transcript (edited for clarity)
So PNH, it is a disease that carries a great deal of morbidity and mortality. And I think with the advent of these complement inhibitors, that’s really changed kind of the treatment landscape of PNH overall. But then in around 20% of patients who have PNH who are receiving the C5 inhibitors already, they continue to have hemolysis, as we saw in our patient as well. And this new medication, the C3 inhibitor which can target both intravascular and extravascular hemolysis, a lot of people are seeing the resolution of the hemolysis, so I think it will play a better role in the future...
So PNH, it is a disease that carries a great deal of morbidity and mortality. And I think with the advent of these complement inhibitors, that’s really changed kind of the treatment landscape of PNH overall. But then in around 20% of patients who have PNH who are receiving the C5 inhibitors already, they continue to have hemolysis, as we saw in our patient as well. And this new medication, the C3 inhibitor which can target both intravascular and extravascular hemolysis, a lot of people are seeing the resolution of the hemolysis, so I think it will play a better role in the future. So this drug was approved in the past couple of years or so and there was a clinical trial that was published in the New England Journal of Medicine in 2021 that kind of went through that actually compared eculizumab to pegcetacoplan and it showed that pegcetacoplan was overall a better drug in reducing hemolysis in patients with PNH.
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