Avatrombopag (AVA), a 2nd generation thrombopoietin receptor agonist, improves outcomes and carries a favorable toxicity profile compared with placebo among patients with chronic immune thrombocytopenic purpura (ITP), according to a poster that will be presented at the 59th American Society of Hematology (ASH) Annual Meeting in Atlanta, Georgia.1

For this phase 3 study, researchers randomly assigned 49 patients with chronic ITP refractory to immunoglobulins, splenectomy, or corticosteroids to receive AVA or placebo over 6 months. The primary outcome was the cumulative weeks during which a patient sustained a platelet response.

Nearly 70% of patients who received AVA completed the study compared with just 6% of patients who received placebo; the most common cause for discontinuation was lack of therapeutic effect.

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Patients who received AVA had a mean platelet response duration of 12.4 weeks versus 0 weeks for patients in the placebo arm (P < .0001). Patients treated with AVA had a platelet response of 65.6%  on day 8 vs 0% in the placebo group (P < .0001).

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Almost 97% of patients treated with AVA reported treatment-emergent adverse events (AE) compared with 58.5% of patients who received placebo. The most frequently reported AEs included headache, upper respiratory tract infection, contusion, epistaxis, and arthralgia.

The authors concluded that “AVA may potentially provide an additional treatment option for patients with refractory chronic ITP.”


  1. Jurczak W, Chojnowski K, Mayer J, Krawczyk K, Bibbiani F. Avatrombopag, a novel oral thrombopoietin receptor agonist, demonstrates superiority to placebo for the treatment of chronic immune thrombocytopenic purpura in a phase 3, multicenter, randomized, double-blind, placebo-controlled trial. Poster presentation at: American Society of Hematology (ASH) 58th Annual Meeting & Exposition; December 9-12, 2017; Atlanta, GA.