Real-world data on thrombosis predictors and C5 complement inhibitors

Paroxysmal nocturnal haemoglobinuria (PNH) is a rare, acquired disease of haematopoietic stem cells of the bone marrow. GPI deficiency results in the absence of complement-regulating proteins. Thromboembolic complications are the most clinically relevant complication and are the main cause of increased morbidity and mortality. Inhibition of the terminal complement system is a targeted therapeutic strategy. Eculizumab and crovalimab bind the complement factor C5 and have proven to be effective and safe treatment options.