In this episode, Rich and Pip discuss de Latour et al. Oral Iptacopan Monotherapy in Paroxysmal Nocturnal Hemoglobinuria. NEJM 2024. Iptacopan is a proximal complement inhibitor and the first oral treatment for paroxysmal nocturnal haemoglobinuria.
The study randomised patients to either continuation of a C5 inhibitor or switch to ictacopan and found that a significant proportion of patients achieved hemoglobin levels of 120 g/L or higher without the need for transfusions. The treatment was generally well-tolerated, with a safety profile consistent with previous studies, suggesting that iptacopan is a viable alternative to existing therapies.
Join us as we delve into the study's methodology, results, and implications for the future management of PNH.
HaemSTAR is an independent UK-wide network of registrars in clinical haematology, interested in promoting and performing research in classical haematology. Our focus is on collaborative projects across haemostasis & thrombosis, transfusion, general haematology, and obstetric haematology.
This episode was sponsored by Sobi as a hands-off educational grant. Sobi had no editorial input whatsoever.