Avatrombopag for adults with early versus chronic immune thrombocytopenia

作者：Zain M. Virk, Rebecca Karp Leaf, David J. Kuter, Katayoon Goodarzi, Nathan T. Connell, Jean M. Connors, Hanny Al‐Samkari · 发表于：American Journal of Hematology · 年份：2023 · DOI：10.1002/ajh.27080 · 被引用次数：28 · 研究领域：Platelet Disorders and Treatments、Blood groups and transfusion、Autoimmune Bullous Skin Diseases

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 grou...