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Iptacopan Real-World Data in Chinese PNH Show Rapid Hemoglobin Gains Without Transfusions

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At the 2026 EHA congress, two Chinese real-world studies of iptacopan in paroxysmal nocturnal hemoglobinuria (PNH) were presented. In IMPACT-PNH China, 34 treatment-naive patients received iptacopan for 3 months: mean hemoglobin rose 4.3 g/dL, half reached 12 g/dL or more, 90.9% saw LDH fall to within 1.5 times the upper limit of normal, and all patients became transfusion-free. A second study followed 15 patients who switched from failed C5 inhibitors to iptacopan or a similar drug; hemoglobin rose from 8.2 to 12.2 g/dL, with LDH, bilirubin and reticulocyte counts all significantly improved. The findings add real-world Chinese evidence that iptacopan can raise hemoglobin and eliminate transfusion dependence in both newly treated and switch patients.

Written by a model from the reports · updated 2 hours ago

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Oct 22
  1. mp.weixin.qq.com
    Fu Rong and Yuan Hengjie Interpret Chinese PNH Real-World Studies: Iptacopan Raised Hb and Freed Patients From Transfusion

    Two Chinese PNH real-world studies were presented at the 2026 EHA congress. IMPACT-PNH China showed that in 34 treatment-naive patients, iptacopan for 3 months raised mean Hb by 4.3 g/dL, with 50% reaching ≥12 g/dL, 90.9% reducing LDH to within 1.5 times normal, and all patients transfusion-free. Another study of 15 patients who switched from failed C5 inhibitors to iptacopan or a similar drug showed Hb rising from 8.2 to 12.2 g/dL, with LDH, bilirubin, and reticulocytes all significantly improved.

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