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IPIG 2025 | Long-term outcomes of pegcetacoplan treatment in patients with PNH & baseline Hb levels over 10 g/dL
Jens Panse, MD, Aachen University Hospital, Aachen, Germany, discusses the long-term outcomes of pegcetacoplan treatment in patients with paroxysmal nocturnal hemoglobinuria (PNH) who presented with baseline hemoglobin levels above 10 g/dL. Drawing on data from both the PEGASUS and PRINCE trials (NCT03500549 and NCT04085601, respectively), Dr Panse highlights that these patients maintained stable hemoglobin and lactate dehydrogenase (LDH) levels, and their reticulocyte counts decreased over time. With up to three years of follow-up, the treatment demonstrated a favorable safety profile in this subgroup of patients. This interview took place at the 2nd International PNH Interest Group (IPIG) Conference in Paris, France.
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Transcript
Within all the SWITCH trials, there were patients that had a low hemoglobin level, but within the PEGASUS trial, there were a couple of patients that had a hemoglobin level above 10 grams per deciliter. So were the patients within the PRINCE trial. And this analysis was based on the outcome for this specific patient population who started with a better than normal hemoglobin level and also showed to have a very, very nice increase of their hemoglobin over the course of time...
Within all the SWITCH trials, there were patients that had a low hemoglobin level, but within the PEGASUS trial, there were a couple of patients that had a hemoglobin level above 10 grams per deciliter. So were the patients within the PRINCE trial. And this analysis was based on the outcome for this specific patient population who started with a better than normal hemoglobin level and also showed to have a very, very nice increase of their hemoglobin over the course of time. And since the long-term follow-up for both the PEGASUS and the PRINCE trial is now two and a half or rather three years in the PEGASUS, we analyzed those patient groups together and it shows that those patients had very, very stable hemoglobin levels, very, very stable decrease in reticulocytes, very very stable LDH levels and there were only two treatment-related adverse events which are not treatment-related to the drug but rather related to other effects. So this group of patients had a very very nice outcome in both of the trials PRINCE and PEGASUS.
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Disclosures
Consulting fees: Alexion, Sobi, Omeros, Sandoz; Payment or honoraria for lectures, presentations, speakers bureaus, manuscript writing or educational events: Alexion, Amgen, Apellis, AstraZeneca, Blueprint Medicines, BMS, Boehringer Ingelheim, Gilead, MSD, Novartis, Pfizer, Roche, Sanofi, Sobi; Support for attending meetings and/or travel: Alexion, Blueprint Medicines, Boehringer Ingelheim, Novartis, Omeros, Pfizer, Roche, Sobi; Participation on a Data Safety Monitoring Board or Advisory Board: Alexion, Amgen, Apellis, AstraZeneca, Blueprint Medicines, BMS, Boehringer Ingelheim, Gilead, MSD, Novartis, Omeros, Pfizer, Roche, Sanofi, Sobi; Leadership or fiduciary role in other board, society, committee or advocacy group, paid or unpaid: www.lichterzellen.de; Receipt of equipment, materials, drugs, medical writing, gifts or other services: Apellis, Blueprint Medicines, Novartis, Roche, Sobi.
