In immune thrombocytopenia (ITP), immunoglobulin G (IgG) platelet autoantibodies accelerate platelet clearance and impair platelet production. IgG homeostasis is regulated by the neonatal Fc receptor (FcRn). Efgartigimod (EFG) is a natural ligand of FcRn engineered to competitively bind to FcRn with high affinity and prevent recycling of endogenous IgG, thereby reducing IgG levels including IgG autoantibody levels. Catherine Broome, MD, Georgetown University, Washington, DC, presents the results of ADVANCE IV (NCT04188379), a Phase III randomized clinical trial evaluating the efficacy and safety of intravenous EFG in adults with primary ITP. 131 participants with long-standing, severe ITP were randomized to receive either 10 mg/kg EFG or placebo (PBO) for 24 weeks. Results demonstrated that a sustained platelet count (PLT) response was reached in more EFG-treated participants (21.8%) than PBO (5.0%). Furthermore, EFG achieved all PLT-related secondary endpoints. The study also showed that more participants on EFG achieved International working group (IWG) response criteria than those on PBO (51.2% versus 20%, respectively). Mean IgG levels in EFG-treated participants decreased steadily over the first 4 weeks of treatment, after which the mean maximum reductions from baseline remained greater than 60% throughout the trial. EFG was also well tolerated with no new safety signals. The most frequent adverse events (AEs) included bruising, headache, hematuria, and petechiae. Overall, the study indicated high efficacy and safety of EFG in patients with ITP. This press briefing took place at the 64th ASH Annual Meeting and Exposition congress in New Orleans, LA.
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