What question did this study set out to answer?

This study aims to evaluate the efficacy and safety of iptacopan in patients with paroxysmal nocturnal hemoglobinuria (PNH).

December 8, 2025BloodOpen Access

Efficacy and safety of iptacopan in Chinese patients with paroxysmal nocturnal hemoglobinuria: A multicenter retrospective study

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Retrospective analysis shows iptacopan improves hematologic responses in PNH patients, suggesting safe treatment effectiveness.

This study aims to evaluate the efficacy and safety of iptacopan in patients with paroxysmal nocturnal hemoglobinuria (PNH).
Retrospective analysis of 20 PNH patients treated with iptacopan across four Chinese centers.
Patients included various PNH subtypes: classic PNH, PNH-aplastic anemia, and PNH-myelodysplastic syndrome.
Assessment of hematologic response, symptom relief, and adverse events.
71% of patients achieved significant hemoglobin increases at week 2; 70% at month 1.
No transfusion requirements reported post-treatment, indicating sustained efficacy.
15% experienced mild adverse events, all manageable without treatment discontinuation.

Xu et al. (2025) studied this question.

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