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IPIG 2025 | Post hoc analysis of the APPLY-PNH trial: the effect of iptacopan on hematological parameters in PNH
In this interview, Austin Kulasekararaj, MBBS, MD, MRCP, FRCPath, King’s College Hospital NHS Foundation Trust, London, UK, briefly discusses the findings of the post hoc analysis of the APPLY-PNH (NCT04558918) trial, which assessed the effect of oral iptacopan monotherapy on hematological parameters in patients with paroxysmal nocturnal hemoglobinuria (PNH) who had previously received C5 inhibitor treatment. This interview took place at the 2nd International PNH Interest Group (IPIG) Conference in Paris, France.
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Transcript
So as we all know, iptacopan has been presented in multiple sessions both in APPLY and APPOINT PNH trials, i.e. looking at patients who either had previous treatment with C5 inhibitors, i.e. the APPLY-PNH trial or APPOINT where patients were treated upfront with iptacopan. So what we did in this post-hoc analysis in APPLY was to look at, more than the primary and secondary endpoints we reported in the trial, to see what category patients belong to from a hematological response as per the EBMT criteria reported in Frontiers by Antonio Risitano...
So as we all know, iptacopan has been presented in multiple sessions both in APPLY and APPOINT PNH trials, i.e. looking at patients who either had previous treatment with C5 inhibitors, i.e. the APPLY-PNH trial or APPOINT where patients were treated upfront with iptacopan. So what we did in this post-hoc analysis in APPLY was to look at, more than the primary and secondary endpoints we reported in the trial, to see what category patients belong to from a hematological response as per the EBMT criteria reported in Frontiers by Antonio Risitano. And we were quite pleased to see that similar to the primary and secondary endpoint with improvement in hemoglobin and also proportion of patients avoiding transfusions and achieving a hemoglobin more than 12, we had a significant proportion of patients changing from their baseline response which was not optimal to a very good optimal response including complete and good responses, reiterating the fact that this response criteria does hold true in the era of proximal inhibition as well.
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Disclosures
Research support (to institution): Celgene/BMS, Novartis; Consultant: Samsung, Novo Nordisk, Alexion/AstraZeneca, Arrowhead Pharmaceuticals, Silence Therapeutics, AdaRx, Ono Pharma; Speaker’s fees: Alexion/AstraZeneca, Amgen, Celgene/BMS, Pfizer, Novartis, Ra Pharmaceuticals/UCB, Roche, Sobi, Janssen; Scientific advisory board/data monitoring committee: Alexion/Astra Zeneca, Apellis, Amgen, Agios, BioCryst, Celgene/BMS, Geron, Novartis, Pfizer, Regeneron, Roche, Sobi, Janssen, Samsung.
