Avatrombopag for the Treatment of Adult Patients with Chronic Immune Thrombocytopenia (cITP): Focus on Patient Selection and Perspectives

Galina Tsykunova,1– 3 Waleed Ghanima2– 4 1Department of Hematology, Haukeland University Hospital, Bergen, Norway; 2Institute of Clinical Medicine, University of Oslo, Oslo, Norway; 3Department of Hemato-Oncology, Østfold Hospital, Grålum, Norway; 4Department of hematology, Oslo University Hospital and Institute of Clinical Medicine, University of Oslo, Oslo, NorwayCorrespondence: Waleed Ghanima, Department of Hemato-Oncology, Østfold Hospital, Kalnesveien 300, Grålum, 1714, Norway, Tel +47 69860000, Email Waleed.ghanima@so-hf.noAbstract: Immune thrombocytopenia (ITP) is an autoimmune disorder characterized by a reduced number of circulating platelets due to immune-mediated destruction and decreased platelet production in the bone marrow. Thrombopoietin receptor agonists (TPO-RAs) are highly effective and widely used in the treatment of patients with steroid treatment failure or dependency. Avatrombopag represents a new supplement to the TPO-RAs family. It was originally approved for the treatment of thrombocytopenia in patients with chronic liver disease who are scheduled to undergo an invasive procedure. However, labeled indications for avatrombopag have been relatively recently expanded to include treatment of chronic ITP in adults with insufficient response to the previous treatments. In this article, we provide an overview of pharmacodynamics and pharmacokinetics of avatrombopag as well as results of the clinical trials related to safety and efficacy of avatrombopag with a perspective on current clinical use. Available data so far suggests that avatrombopag can be effectively used in ITP patients and has a favorable safety profile. Though further studies are needed to affirm the efficacy and safety, avatrombopag has the potential to become a TPO agonist of choice for many patients with ITP.Keywords: ITP, thrombopoietin receptor agonists, avatrombopag