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ASH 2025 | A real-world analysis of the outcomes of patients with PNH treated with iptacopan
Iptacopan was the first oral complement inhibitor approved by the US Food and Drug Administration (FDA) for the management of patients with paroxysmal nocturnal hemoglobinuria (PNH). Carmelo Gurnari, MD, Cleveland Clinic, Cleveland, OH, outlines the findings of a real-world analysis of data from patients treated with this agent, either as first-line treatment or after prior intravenous therapy. This interview took place at the 67th ASH Annual Meeting and Exposition, held in Orlando, FL.
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Transcript
The current treatment landscape of paroxysmal nocturnal hemoglobinuria has dramatically changed and this is because now we have the opportunity to treat these patients not anymore with biweekly or every eight weeks administration routes that are intravenous, but only with oral pills. So this, of course, is a major breakthrough in the treatment of these patients that need to be treated chronically lifelong...
The current treatment landscape of paroxysmal nocturnal hemoglobinuria has dramatically changed and this is because now we have the opportunity to treat these patients not anymore with biweekly or every eight weeks administration routes that are intravenous, but only with oral pills. So this, of course, is a major breakthrough in the treatment of these patients that need to be treated chronically lifelong. So, of course, there’s a lot of interest also from the patients themselves to explore new treatment options.
And Iptacopan, which is a new molecule that has been approved a couple of years ago by the FDA, is indeed offering a new opportunity for these patients. So we at the Cleveland Clinic have a large practice of PNH patients, and we analyzed the treatment outcomes and also the safety profile of this new molecule in a real-world setting, like right after the FDA approval. We analyzed 62 patients, 12 of whom were treatment-naive, and the other 50 were switched because of patient preference. They heard about this oral treatment and of course they wanted to be off the intravenous, right? So they switched.
And we found that indeed all patients had improvement in hemoglobin level. Of course, the ones that were treatment-naive had more improvement because they had a disease that needed to be controlled. And the safety profile was absolutely good, like in the registrative trial, we didn’t have any thrombosis or any safety concern. The breakthrough hemolytic events were mostly due to infection and did not cause any harm to the patient or treatment changes or top-up transfusions, none of it. And therefore, we are absolutely happy that our patients show a safety profile of the drug and an increase in all the parameters that we look at, like increasing hemoglobin, decreasing reticulocytes and LDH, and all the parameters that we look at when we treat patients with PNH.
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