Abstract Number: PB1349
Meeting: ISTH 2020 Congress
Theme: Platelet Disorders and von Willebrand Disease » Acquired Thrombocytopenias
Background: Immune thrombocytopenia (ITP) is an acquired bleeding disorder caused by immune-mediated platelet destruction and insufficient platelet production. AVA is an oral thrombopoietin receptor agonist (TPO-RA) approved for the treatment of chronic ITP when a patient has had insufficient response to a prior therapy. A better understanding of the efficacy profile for AVA will help clinicians make treatment decisions for their patients.
Aims: The objective of these analyses was to evaluate the efficacy of AVA treatment in ITP patients using alternative measurements that may be clinically meaningful.
Methods: A 6-month, multicenter, randomized, double-blind, Phase 3 study enrolled 32 AVA and 17 placebo-treated patients with ITP. The primary endpoint was the median number of cumulative weeks of platelet count (PC) response (achieving a PC ≥50,000/µL) over the course of the study without rescue medication. Patients receiving any rescue medication during the study were deemed to be non-responders for the remainder of the study. These post-hoc analyses evaluate the ability to achieve a PC of ≥50,000/µL once or twice during the course of the study and consecutive weeks of PC ≥50,000/µL.
Results: 87.5% of AVA versus 5.9% of placebo-treated patients attained a PC ≥50,000/µL at least once during the study. 81.3% of AVA-treated patients were able to achieve this PC response level at least twice in comparison to 0.0% with placebo. 53.3% of patients had a PC ≥50,000/µL for at least 4 consecutive weeks, as opposed to 0.0% with placebo. The mean number of weeks of continuous platelet response was 6.5 for AVA versus 0.1 for placebo-treated patients.
Conclusions: AVA effectively increases PCs in patients with ITP and the majority of treated patients achieved a sustained platelet response. Results of this analysis may be more meaningful for HCPs who understand that treatment of chronic ITP requires constant monitoring and dose modifications to manage PCs.
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To cite this abstract in AMA style:
Maitland H, Aggarwal K, Vredenburg M, Tian W, Gabrail N. Response to Avatrombopag (AVA) in Chronic Immune Thrombocytopenia: Alternative Efficacy Measures [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/response-to-avatrombopag-ava-in-chronic-immune-thrombocytopenia-alternative-efficacy-measures/. Accessed October 2, 2023.« Back to ISTH 2020 Congress
ISTH Congress Abstracts - https://abstracts.isth.org/abstract/response-to-avatrombopag-ava-in-chronic-immune-thrombocytopenia-alternative-efficacy-measures/