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Symposium
  • EHA •
  • 13 June, 2026 •
  • •

From Labs to Lives: Redefining Success in Rare Diseases Paroxysmal Nocturnal Haemoglobinuria (PNH) and Chronic Immune Thrombocytopenia (ITP)

Danny Hsu, Ana Boban, María L Lozano

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Video
Publication
  • EHA •
  • 13 June, 2026 •
  • •

Consistency of Response by Baseline Platelet Count in Pacritinib-treated patients in the Real World: MY-PAC Analysis

James Rossetti, Douglas Tremblay, Abiola Oladapo, Michael Marrone, Caleb Paydar, Djibril Liassou, Sarah A. Buckley, Purvi Suthar, Adina Geprifti, Michael Vredenburg , Bruce Feinberg, Naveen Pemmaraju

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Poster
Publication
  • EHA •
  • 13 June, 2026 •
  • •

Real-world hemoglobin profiles and transfusion burden in patients with paroxysmal nocturnal hemoglobinuria receiving complement inhibitors: A US claims analysis

Carlos de Castro, Peter Blandino, Fiston Vuvu, Enrico Zanardo, Lynn Huynh, Robert Schell, Nathaniel Downes, Yixin Yang, Mei Sheng Duh, Peter Hillmen

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Poster
Publication
  • ERA •
  • 4 June, 2026 •
  • •

Decreasing rates of injection site reactions over time: Long-term outcomes across multiple indications support pegcetacoplan for C3G/primary IC-MPGN

Nabil Melhem, Antonio Mastrangelo, Daniel Zecher, Abdallah S Geara, Carla M Nester, Sheena Patel, Liza Alkhori Franzén, Andrew Carrel, Carlos de Castro, Daniel P Gale, Richard J. Kelly

Available materials

Oral Presentation
Publication
  • ASH •
  • 8 December, 2025 •
  • •

Real-World Clinical Characteristics and Treatment Outcomes in PNH Patients Prescribed Pegcetacoplan: Insights of Complement Inhibitor-Experienced and -Naïve Patients Across Europe, the United States and Canada  

Jens Panse , Koo Wilson, Brianne Kerr, Sam Williamson, Susanna Libby, Yasmin Taylor, Peter Hillmen, David Dingli

Publication
  • ASH •
  • 8 December, 2025 •
  • •

Patient-reported outcomes of avatrombopag for chronic immune thrombocytopenia: Interim analysis of the Phase 4 adopt study

María Eva Mingot Castellano, María Teresa Álvarez Román, María Luisa Lozano, Wolfgang Miesbach, Hafiz Qureshi, Vickie McDonald, Jessica Zhang, Viridiana Cano Garcia, Nina Skuban

Publication
  • ASH •
  • 7 December, 2025 •
  • •

Early Results From the Ongoing Pegcetacoplan Silo of the International Paroxysmal Nocturnal Hemoglobinuria Interest Group Registry 

Carlos de Castro, Peter Hillmen, Peter Blandino, David Araten

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