Lusutrombopag Is Safe and Efficacious for Treatment of Thrombocytopenia in Patients With and Without Hepatocellular Carcinoma.
Patients with hepatocellular carcinoma (HCC) secondary to chronic liver disease often require invasive procedures, but frequently have thrombocytopenia. Lusutrombopag is an agonist of the thrombopoietin receptor that activates platelet production.We performed an integrated analysis of data from 2 phase 3 trials (L-PLUS 1, Japan, October 2013 to May 2014 and L-PLUS 2, global, June 2015 to April 2017) that compared efficacy and safety of lusutrombopag to placebo in patients with chronic liver disease, with and without HCC. Our analysis included patients with ECOG grades of 0 or 1, Child-Pugh classes A or B, and platelet count below 50 x 109/L who were scheduled to undergo invasive procedures in 9 to 14 days. Patients received lusutrombopag (3 mg) or placebo daily for 7 days or less before an invasive procedure. Imaging studies assessed treatment-emergent adverse events, including asymptomatic portal vein thrombosis. The primary endpoint was no requirement for platelet transfusion before the invasive procedure or rescue therapies for bleeding 7 days or less after the invasive procedure.The per protocol population included 270 patients (95 with HCC). A significantly higher proportion of patients with HCC who received lusutrombopag achieved the primary endpoint (68.0%) vs patients who received placebo (8.9%) (P