Real-world experience in management of patients with PNH treated with iptacopan: A case series

This case series on managing paroxysmal nocturnal hemoglobinuria (PNH) with iptacopan focuses on scenarios in which the risk of breakthrough hemolysis (BTH) or thrombotic events is heightened. Patient 1 was a 46-year-old woman with PNH and a history of hepatic vein thrombosis. Patient 2 was a 30-year-old woman with PNH and a history of right posterior cerebral artery stroke and cerebral venous sinus thrombosis with extensive right superficial thrombophlebitis. Patient 3 was a 48-year-old woman with PNH who contracted a gastrointestinal infection 3 months after starting iptacopan. Patient 4 was a 39-year-old man with PNH who experienced serous otitis media, a complement-amplifying condition (CAC), concurrent with upper respiratory infection. Patient 5 was a 56-year-old woman with PNH who underwent radiofrequency ablation of the spine while on iptacopan. No patients experienced BTH or hospitalization while on iptacopan. No new thrombotic events were reported after initiating iptacopan. For patients experiencing CACs or surgery, no changes in PNH therapy were required. Notably, BTH did not occur in the patient with gastroenteritis, which can cause drug malabsorption. This case series suggests that iptacopan effectively maintains complement inhibition and prevents PNH-related hemolysis and thrombosis in the setting of past thromboses, gastrointestinal illness, and other CACs.