Case report: persistent anemia after eculizumab in paroxysmal nocturnal hemoglobinuria: non-dominantly active intravascular hemolysis - PubMed
6 hours ago
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- #iptacopan
- #PNH
- Paroxysmal nocturnal hemoglobinuria (PNH) is a rare condition marked by intravascular hemolysis (IVH), thrombosis, and organ damage.
- C5 complement inhibitors like eculizumab improve PNH prognosis, but some patients still experience persistent anemia.
- A case report describes a PNH patient who initially responded to eculizumab but became transfusion-dependent after 24 weeks.
- Biomarkers like lactate dehydrogenase (LDH), free hemoglobin, and haptoglobin indicated subclinical yet active IVH.
- Iptacopan monotherapy normalized LDH, free hemoglobin, and haptoglobin levels, eliminating transfusion needs and increasing type III RBCs to 57.3%.
- Persistent anemia post-C5 inhibitor therapy may be linked to subclinical IVH, with proximal complement inhibitors like iptacopan offering effective management.