Findings from two international, randomized, double-blind, phase III, placebo-controlled trials published in the September issue of Gastroenterology revealed that the oral second-generation thrombopoietin agonist avatrombopag (Doptelet) when given once a day, notably decreased the requirement for platelet transfusion and rescue therapy for as many as 7 days post the patients with chronic liver disease and thrombocytopenia underwent procedures.

The ADAPT-1 trial reported results showing 66% of patients in the 60-mg section achieved this primary endpoint, and also 88% of patients who received 40 mg for less severe thrombocytopenia, contrasting 23% and 38% of the placebo sections, respectively. In the ADAPT-2 trial, 69% of the 60-mg group met the primary endpoint, as also 88% of the 40-mg group did, versus 35% and 33% of the respective placebo groups.

These observations caused the FDA to approve avatrombopag in May 2018 under its priority review process. The new therapy “may be a safe and effective alternative to platelet transfusions” that could make the clinical management of patients with chronic liver disease and thrombocytopenia easier, Norah Terrault, MD, MPH, and her associates wrote in Gastroenterology.

Avatrombopag and placebo showed similar rates of treatment-caused adverse events. These mostly included abdominal pain, dyspepsia, nausea, pyrexia, dizziness, and headache. Merely three avatrombopag patients’ platelet counts rose above 200 x 10⁹ per liter, showing no symptoms, the investigators noted.