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December 8, 2025Blood

Correlation between efficacy, clonal dynamics evolution, and development of bone marrow failure in PNH/AA syndrome treated with complement inhibitors: A single-center cohort study

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Authors

BHBing Han

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Overview

Retrospective analysis shows 89% hemolysis control with complement inhibitors, suggesting persistent challenges in bone marrow failure.

Key Points

  • 89% of patients achieved hemolysis control, but only 64% achieved a complete hematologic response during treatment.
  • Retrospective analysis assessed 92 PNH/AA patients receiving eculizumab, crovalimab, or iptacopan over 29 months.
  • New somatic mutations linked to bone marrow failure emerged in 20% of patients, highlighting clonal dynamics impact.
  • Residual hemolysis and expanding PNH clone sizes were strongly correlated with bone marrow failure progression.

Cite This Study

Bing Han (2025) studied this question.

synapsesocial.com/papers/69362f6c4fa91c937236e00chttps://doi.org/10.1182/blood-2025-4987
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