Real-world avatrombopag use for immune thrombocytopenia in the United States: the REAL-AVA 2.0 multisite chart review study

The REAL-AVA 2.0 study was a retrospective multisite chart review evaluating avatrombopag (AVA) effectiveness among patients with primary immune thrombocytopenia (ITP) who underwent routine care in academic- and community-based practices in the United States and initiated AVA between 1 July 2019 and 30 June 2024. Treatment response was defined as achieving platelet counts (PC) ≥30 × 10³/μL, ≥50 × 10³/μL, or ≥100 × 10³/μL. The discontinuation of steroids and/or immunosuppressants after AVA initiation was evaluated. Analyses were performed in the overall population and among 2 stratified subgroups: ITP disease duration (acute, <3 months; persistent, 3-12 months; and chronic, ≥12 months) and prior thrombopoietin receptor agonists exposure status. Among the 177 patients included in the study, 90% achieved or maintained a PC ≥30 × 10³/μL response, 86% achieved or maintained a PC ≥50 × 10³/μL response, and 76% of patients achieved or maintained a PC ≥100 × 10³/μL response, with a median duration of response to AVA of 12.1, 12.4, and 10.0 months, respectively. The median durability of response was 96% at the PC ≥30 × 10³/μL threshold, 93% at the PC ≥50 × 10³/μL threshold, and 76% at the PC ≥100 × 10³/μL threshold. Almost all patients (98%) who used concomitant steroids at AVA initiation subsequently reduced the steroid dose (19%) or discontinued steroid use completely (79%), and 40% of those who used immunosuppressants discontinued them after AVA initiation. Results of the subgroup analyses were consistent with the overall sample. Findings show that AVA is an effective treatment option for patients with ITP, demonstrating durable PC response in a diverse population.