Avatrombopag for adults with early versus chronic immune thrombocytopenia

Abstract Avatrombopag is a newer thrombopoietin receptor agonist (TPO‐RA) currently approved to treat chronic ITP (duration >12 months). No studies have yet evaluated the safety and effectiveness of avatrombopag in newly diagnosed ITP (duration <3 months) or persistent ITP (duration 3–12 months), and so its use in these populations is presently off‐label worldwide. We hypothesize that avatrombopag has similar safety and effectiveness irrespective of ITP disease phase. To evaluate this, we performed a multicenter observational cohort study of adults with ITP treated with avatrombopag, comparing patient outcomes by disease phase (newly diagnosed/persistent versus chronic). Seventy‐five patients were included, 23 with newly diagnosed/persistent ITP (17.7 patient‐years of avatrombopag treatment) and 52 with chronic ITP (65.3 patient‐years of avatrombopag treatment). On avatrombopag, 91% of newly diagnosed/persistent patients versus 96% of chronic patients ( p = .58) achieved a platelet response (≥50 × 10 9 /L) and 86% versus 81% of patients ( p = .78) achieved a complete response (≥100 × 10 9 /L). Median platelet counts on avatrombopag were similar between the two groups (165 × 10 9 /L vs. 129 × 10 9 /L, p = .57). Response durability was high and similar in both groups. No patients in the newly diagnosed/persistent group had a major bleeding event, thromboembolic event or avatrombopag discontinuation for adverse events, compared with 4, 1, and 2, respectively, in the chronic group. Thrombocytosis (platelets ≥400 × 10 9 /L) incidence was similar in the two groups. No other drug‐related adverse events occurred in either group. Avatrombopag was safe and effective in patients with newly diagnosed and persistent ITP, with outcomes numerically, statistically, and clinically similar to patients receiving avatrombopag for chronic ITP.