Untreated PNH can lead to early mortality.4 In fact, before the availability of today’s standard of care of C5 inhibitors, ~35% of patients died within 5 years.5,6
For this reason, rapid diagnosis of PNH and urgent initiation of a complement-mediated treatment that can block uncontrolled terminal complement activity – the cause of intravascular haemolysis (IVH), thrombosis and life-threatening consequences in PNH – is essential.1,7
Breakthrough IVH is a return of IVH and its potentially life-threatening consequences due to inadequate complement inhibition, complement-activating conditions (triggers), such as an infection or surgery, or missed treatment dose.1,2,9 Complete and sustained terminal complement inhibition lowers the risk of breakthrough IVH events.1-3,11
Today, targeted C5 inhibition has transformed the natural history of PNH by improving the survival rate to near normal levels, similar to that seen in the general population.12,13
Disclaimer: Survival was not a prespecified endpoint, rather a post-hoc analysis and study was not powered to detect differences in survival. This study and OLE were not designed to assess survival. Death was a post-hoc safety endpoint. Survival analysis was performed using data for 192 complement inhibitor-experienced patients who received weight-based dosing of ravulizumab every 8 weeks for up to 4 years during the open-label extension phase of a Phase 3 randomized study (Study 302; ClinicalTrials.gov NCT03056040).
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