Real-world Application of the Oral Factor B Inhibitor Iptacopan in Chinese Patients with Paroxysmal Nocturnal Hemoglobinuria: A Four-case Series Analysis

Description

Paroxysmal nocturnal hemoglobinuria (PNH) treatment with iptacopan has demonstrated efficacy in trials, but real-world data from developing countries remain scarce. Economic constraints necessitate cost-sparing strategies, and the impact of iron deficiency on hematopoietic recovery is underrecognized. We report four cases addressing these gaps.

Institutions

• Southern Medical University

  Guangdong, Guangzhou

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