Switching patients with PNH from pegcetacoplan to iptacopan: a case series.
Mark Orland, Carmelo Gurnari, Srinivasa Sanikommu et al.
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Case in brief
This case series describes three adults with paroxysmal nocturnal hemoglobinuria (PNH), including one man with aplastic anemia. Each had received C5 inhibitor therapy and then pegcetacoplan; hemolysis occurred during prior treatment, and one patient required transfusions with both C5 inhibitor and pegcetacoplan therapy. All three switched to iptacopan monotherapy. On iptacopan, none had reported extravascular or breakthrough hemolysis, hemoglobin levels improved, and transfusion requirements abated. The authors also reported no laboratory evidence of intravascular hemolysis after the switch.
What made this case unusual
The series focuses on patients who switched specifically from pegcetacoplan to iptacopan after hemolysis on prior complement-inhibitor regimens, with no reported hemolysis on iptacopan.
Diagnostic / clinical pearl
In this three-patient series, switching from pegcetacoplan to iptacopan was followed by improved hemoglobin and reduced transfusion requirements, without reported laboratory evidence of hemolysis; these observations are descriptive and do not establish comparative efficacy.