As part of scientific exchange, Novartis is providing the most recent abstract(s) accepted by the referenced medical congress. The scientific information may include data/information on investigational use(s) of compounds/drugs for which efficacy and safety have not been established. Prescribing information may vary depending on local health authority approval in each country. Before prescribing any product, always refer to the Prescribing Information (PI), Summary of Product Characteristics (SmPC), or product information as appropriate in your country. Information is not intended to promote or otherwise commercialize (directly or indirectly) any off-label or unapproved uses of Novartis products.
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APPULSE-PNH: Oral iptacopan monotherapy demonstrates clinically meaningful hemoglobin (Hb) increases in patients (pts) with paroxysmal nocturnal hemoglobinuria (PNH) and Hb ≥10 g/dL on anti-C5 therapy
Austin Kulasekararaj*, Flore Sicre de Fontbrune, Anna Gaya Vallas, Ilene Weitz, David Kuter, David J Araten, Bhumika J Patel, Abhay Singh, Jun Ho Jang, Richard J Kelly, Eros Di Bona, Michael Loschi, Vinod Pullarkat, Jörg Schubert, Alessandro Vannucchi, Mustafa Nuri Yenerel, Régis Peffault de Latour, Navin Mahajan, Luca Monaco, Tao Ding, Tomasz Lawniczek, Philippe Ferber, Marion Dahlke, Antonio Maria Risitano
Oral presentation #S183
Brown Hall 2 | Friday, June 13, 2025 | 17:45 – 18:00 CEST
Indirect treatment comparison of iptacopan vs. pegcetacoplan in complement inhibitor naïve paroxysmal nocturnal hemoglobinuria patients
Phillip Scheinberg, Régis Peffault de Latour, Maria-Magdalena Balp, Anggie Wiyani*, Jason Steenkamp, Becky Hooper, Lucas Torres, Diego Kashiura, Austin Kulasekararaj
Poster presentation #PF1303
TBU | Friday, June 13, 2025 | 18:30 – 19:30 CEST
The 2-year safety and efficacy of iptacopan monotherapy in patients with paroxysmal nocturnal hemoglobinuria (PNH) from APPLY- and APPOINT-PNH studies who entered the roll-over extension program (REP)
Antonio Risitano, Austin Kulasekararaj, Phillip Scheinberg, Yasutaka Ueda, Alexander Röth, Bing Han, Li Zhang, Eros Di Bona, Rong Fu, Morag Griffin, Richard Kelly, Jens Panse, Hubert Schrezenmeier, Marion Dahlke, Philippe Ferber, Rajeshri Karki, Silvia Sanz, Olivier Somenzi, Luca Monaco, Erik Houtsma, Régis Peffault de Latour*
Poster presentation #PF660
Poster Hall | Friday, June 13, 2025 | 18:30 – 19:30 CEST
Effectiveness and safety of iptacopan in paroxysmal nocturnal hemoglobinuria patients with persistent anemia after C5 inhibition: The real-world experience of the French early access program
Edouard Forcade, Michael Loschi, Myriam Aroichane, Anne Filipovics, Régis Peffault de Latour*, Flore Sicre de Fontbrune
Poster presentation #PF671
Poster Hall | Friday, June 13, 2025 | 18:30 – 19:30 CEST
Obtaining insights on PNH management with iptacopan in everyday clinical practice: A research collaboration with the IPIG PNH Registry
Jilles Fermont, Alexandra S Deeks, Jamile M Shammo, Régis Peffault de Latour, Richard J Kelly
Publication only #PB2806
A phase 1, randomized, participant- and investigator-blinded, placebo-controlled study to explore the safety and pharmacokinetics of supratherapeutic multiple doses of iptacopan in healthy volunteers
Robert Schmouder, Melissa Hackling, Bharti Shah, S. Eralp Bellibas, Kenneth Kulmatycki
Publication only #PB3628
Health-related quality of life and preference for treatment modalities of patients with paroxysmal nocturnal hemoglobinuria (PNH): Results of a real-world study
Alexander Röth*, Maria-Magdalena Balp, Anggie Wiyani, Yasmin Taylor, Silvia Sanz
Poster presentation #PS2291
Poster Hall | Saturday, June 14, 2025 | 18:30 – 19:30 CEST
Real-world outcomes and clinical burden of patients with paroxysmal nocturnal hemoglobinuria
Talha Munir*, Eloise Beggiato, Maria-Magdalena Balp, Anggie Wiyani, Silvia Sanz, Yasmin Taylor, Alexander Röth
Poster presentation #PF670
Poster Hall | Friday, June 13, 2025 | 18:30 – 19:30 CEST
The daily impact of fatigue in paroxysmal nocturnal haemoglobinuria (PNH): An ethnographic study
Bing Han, Gifty Amporful Cobby, Maria-Magdalena Balp, Josefin Snellman, Olivier Somenzi, Scarlett Che, Barry Katsof, Jordi Cruz, Heithor Zanini, Ben Walker, Rachel Hiley, Rachael Eddleston, Sayoko Murakami, Monika Oliver
Publication only #PB3791
Complement activation in patients with severe staphylococcus aureus bloodstream infection
Stephan Moser, Anette Orjuela León, Sabina Pfister, Anna Schubart, Tobias Junt, Marten Trendelenburg, Michael Osthoff
Publication only #PB3608
Consensus algorithm for screening and diagnosis of paroxysmal nocturnal hemoglobinuria in Greece: Insights from a multi-specialty expert Delphi-panel
Eleni Gavriilaki, Maria Angelopoulou, Vasiliki Antonopoulou, Evangelia Dounousi, George Giannakoulas, Olga Giouleme, John Koskinas, Zisis Kratiras, Evangelos Liberopoulos, Konstantina Konstantopoulou, Maria Papaioannou, Elena Solomou
Publication only #PB2818
