Efgartigimod significantly improved platelet counts in patients with primary immune thrombocytopenia (ITP), compared with placebo. Moreover, the phase 3 ADVANCE IV study showed that the study drug was well tolerated by participants.

The ADVANCE trial randomized 131 patients with chronic or persistent ITP, who had received at least two prior ITP treatments, or one prior and one concurrent treatment, and had platelet counts less than 30×109/L 2:1 to efgartigimod or placebo.1 The primary endpoint was the proportion of patients with a sustained platelet count response, defined as at least 50×109 platelets/L in at least four out of six visits in weeks 19-24 of the study, in the absence of intercurrent events. Dr. Catherine Broome (Georgetown University) presented the findings at the 2022 annual meeting of the American Society of Hematology.

In total, 21.8% of participants in the efgartigimod arm and 5.0% in the placebo arm achieved the primary endpoint, reflecting a significant difference between the arms in favor of the experimental drug (P=0.0316). In addition, the number of cumulative weeks of disease control showed a benefit for the efgartigimod arm over the placebo arm (mean 6.1 vs 1.5; P=0.0009). Dr. Broome added that the results were consistent across subgroups.

The study drug was well tolerated in the study population and results were consistent with previous data published on efgartigimod.2,3 Serious adverse events (AEs) were more prevalent in the placebo arm than in the efgartigimod arm (15.6% vs 8.1%). Dr. Broome commented that this was mostly due to an increased rate of bleeding events in the placebo arm (86.7% vs 70.9%). Finally, the infection rate was numerically slightly higher in the efgartigimod arm than in the placebo arm (29.1% vs 22.2%).

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