What question did this study set out to answer?

To analyze the efficacy and safety of the oral factor B inhibitor iptacopan in patients with paroxysmal nocturnal hemoglobinuria (PNH).

December 8, 2025BloodOpen Access

Efficacy and safety analysis of iptacopan in paroxysmal nocturnal hemoglobinuria: A real world retrospective study of China

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Retrospective study shows iptacopan improves hemoglobin and reduces adverse events in patients with PNH, indicating effective treatment options.

To analyze the efficacy and safety of the oral factor B inhibitor iptacopan in patients with paroxysmal nocturnal hemoglobinuria (PNH).
Retrospective study of 42 PNH patients receiving iptacopan
Evaluated hemoglobin levels, red-cell transfusion independence, and other clinical outcomes
Statistical analysis conducted using SPSS20.0
86.6% of complement inhibitor-naive patients met hemoglobin response criteria after one week
100% of complement inhibitor-naive patients achieved LDH normalization
47.6% of patients experienced mild treatment-related adverse events

Niu et al. (2025) studied this question.

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Retrospective study shows iptacopan improves hemoglobin and reduces adverse events in patients with PNH, indicating effective treatment options.