Work

Switching patients with PNH from pegcetacoplan to iptacopan: a case series

Year: 2026

Type: article

Abstract: OBJECTIVES: Limited published data exist on switching therapy from pegcetacoplan to iptacopan in patients with paroxysmal nocturnal hemoglobinuria (PNH). METHODS: Three patient cases were collected from the Mayo Clinic, Cleveland Clinic, and Levine Cancer Institute. RESULTS: Patient 1 is a 57-year-old woman with PNH. She experienced extravascular hemolysis (EVH) while on C5 inhibitors and breakthrough hemolysis (BTH) after switching to pegcetacoplan. Patient 2 is a 37-year-old woman seeking care for PNH, who was initiated on C5 inhibitor therapy and switched to pegcetacoplan. EVH and BTH were observed while she was on C5 inhibitor therapy, and transfusions were required with both C5 inhibitor and pegcetacoplan therapy. Patient 3 is a 58-year-old man with aplastic anemia and PNH who was initiated on C5 inhibitor therapy, then switched to pegcetacoplan. He experienced both EVH and BTH. DISCUSSION: All three patients switched from pegcetacoplan to iptacopan, and none experienced EVH or BTH while on iptacopan. After switching to iptacopan, all three patients improved hemoglobin levels and abating transfusion requirements. CONCLUSION: In this case series, three patients with PNH were successfully transitioned from pegcetacoplan to iptacopan monotherapy. No patients exhibited laboratory evidence of BTH, EVH, or intravascular hemolysis after switching to iptacopan.

Source: Hematology

DOI: 10.1080/16078454.2026.2618403

Authors: Mark Orland, Carmelo Gurnari, Srinivasa Sanikommu, Jaroslaw Maciejewski, David Dingli

Institutions: Cleveland Clinic, University of Rome Tor Vergata, Levine Cancer Institute, Mayo Clinic

Language: English

Topic: Complement system in diseases

Subfield: Immunology

Field: Immunology and Microbiology

Domain: Life Sciences

SDG: Good health and well-being

Open Access status: gold

Estimated APC paid: $2,557