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December 8, 2025BloodOpen Access

Real-world data on breakthrough hemolysis in patients with paroxysmal nocturnal hemoglobinuria treated with proximal and terminal complement inhibitors.

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Authors

KKK.P.S. KushwahaJLJimmy LeeTBTaha Bat

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Overview

Real-world data shows breakthrough hemolysis impacts anemia in paroxysmal nocturnal hemoglobinuria patients, suggesting management challenges.

Key Points

  • To evaluate the incidence and management of breakthrough hemolysis in patients with paroxysmal nocturnal hemoglobinuria on complement inhibitors.
  • Analyzed real-world U.S. data from patients aged 18-70 with paroxysmal nocturnal hemoglobinuria treated with complement inhibitors from 2007-2025.
  • Defined breakthrough hemolysis events by declines in hemoglobin and corresponding LDH levels within specified timelines.
  • Examined patient demographics, treatment details, and outcomes related to breakthrough hemolysis.
  • 18 of 87 patients experienced breakthrough hemolysis events, highlighting persistent anemia challenges.
  • Median duration on complement inhibitors was approximately 93.9 months, with notable effects on hemoglobin and LDH levels during events.
  • 25 of 49 breakthrough hemolysis events were linked to complement amplifying conditions.

Cite This Study

Kushwaha et al. (2025) studied this question.

synapsesocial.com/papers/69362f6c4fa91c937236dffbhttps://doi.org/10.1182/blood-2025-4986
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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 breakthrough hemolysis patterns across 1,723 patient-years of complement inhibition in paroxysmal nocturnal hemoglobinuria2025
  2. 2Characterization of Breakthrough Hemolysis in Patients With Paroxysmal Nocturnal Hemoglobinuria: An International Multicenter Experience.2025 · 6 citations
  3. 3The effect of breakthrough hemolysis on complement factor h and factor I levels in paroxysmal nocturnal hemoglobinuria2025
  4. 4[New treatment strategies for paroxysmal nocturnal hemoglobinuria: drug selection in the era of novel complement inhibitors].2025
  5. 5Factor bb as a biomarker during breakthrough hemolysis in paroxysmal nocturnal hemoglobinuria2025