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December 8, 2025BloodOpen Access

Real-world breakthrough hemolysis patterns across 1,723 patient-years of complement inhibition in paroxysmal nocturnal hemoglobinuria

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VVValeria Visconte

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Overview

Analysis shows eculizumab treatment leads to breakthrough hemolysis in paroxysmal nocturnal hemoglobinuria patients, indicating a need for improved therapies.

Key Points

  • Assess the incidence and characteristics of breakthrough hemolysis during complement inhibition in PNH patients.
  • Reviewed data over 1,723 patient-years from 208 patients on CI therapy from 2004 to 2025.
  • Defined breakthrough hemolysis using specific hemoglobin and LDH criteria.
  • Analyzed BTH rates based on treatment protocols and therapy transitions.
  • Documented 153 BTH episodes with varying rates across different complement inhibitors.
  • Noted that eculizumab had significant BTH occurrences in non-responders compared to complete responders.
  • Iptacopan showed the lowest BTH incidence among treatments, suggesting promising therapeutic advantages.

Cite This Study

Valeria Visconte (2025) studied this question.

synapsesocial.com/papers/69362f444fa91c937236d5c0https://doi.org/10.1182/blood-2025-3205
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Also Consider

Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

  1. 1Real-world data on breakthrough hemolysis in patients with paroxysmal nocturnal hemoglobinuria treated with proximal and terminal complement inhibitors.2025
  2. 2Successful switch from pegcetacoplan to iptacopan after repeated severe breakthrough hemolysis events - case report.2025 · 1 citations
  3. 3Characterization of Breakthrough Hemolysis in Patients With Paroxysmal Nocturnal Hemoglobinuria: An International Multicenter Experience.2025 · 6 citations
  4. 4Impact of iptacopan, a complement inhibitor, on Chinese patients with paroxysmal nocturnal hemoglobinuria: A multicenter retrospective cohort study2025
  5. 5Complement inhibition in paroxysmal nocturnal hemoglobinuria: From biology to therapy2024 · 28 citations