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IPIG 2025 | Treating patients with PNH and bone marrow failure

In this video, Jeff Szer, MBBS, FRACP, Peter MacCallum Cancer Centre, Melbourne, Australia, briefly comments on the effectiveness of treatment in patients with paroxysmal nocturnal hemoglobinuria (PNH) and a bone marrow failure syndrome, such as aplastic anemia. According to Prof. Szer, available data suggest that treatment for one condition does not impact the responsiveness to therapy for the other, and that they should be regarded as related but independent clinical entities when therapy is selected. This interview took place at the 2nd International PNH Interest Group (IPIG) Conference in Paris, France.

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Transcript

This is a complicated question and it refers to whether the impact of treatment for bone marrow failure, for example, either aplastic anemia or hypocellular myelodysplasia, impacts on the disease PNH or impacts on its treatment and vice versa. And I think most of the evidence that we have, and much of this came from our previous registry from the global registry that was running under the Alexion banner for many years...

This is a complicated question and it refers to whether the impact of treatment for bone marrow failure, for example, either aplastic anemia or hypocellular myelodysplasia, impacts on the disease PNH or impacts on its treatment and vice versa. And I think most of the evidence that we have, and much of this came from our previous registry from the global registry that was running under the Alexion banner for many years. But the data that we had suggested that there was actually no difference in the responsiveness of patients with aplastic anemia and PNH that required treatment, either of the aplastic anemia treated with immunosuppressive therapy or the PNH treated with complement blockade. So they need to be regarded as related but independent clinical entities when therapy is chosen.

 

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Disclosures

Financial relationship (not shares) with Alexion, Sobi, Novartis.