Safety and effectiveness of lusutrombopag in patients who have chronic liver disease with thrombocytopenia and undergoing invasive procedures: Real-world post-marketing surveillance in Japan.

Abstract

Lustrombopag has been approved for the treatment of thrombocytopenia in patients with chronic liver diseases (CLD) who are scheduled to undergo an invasive procedure. Here we report the final results of a post-marketing surveillance assessing the safety and effectiveness of lusutrombopag in Japan. This multicenter, prospective, real-world surveillance collected data from case report forms between October 2016 and May 2021. The observation period was 2 months after the first day of lusutrombopag treatment. Safety and effectiveness (proportion of patients avoiding preoperative platelet transfusion and responders who achieved platelet count increase from baseline) were assessed. The safety analysis set included 1033 (100.0%), 130 (12.6%), and 14 (1.4%) patients received ≥1, ≥2, and ≥3 treatment cycle(s), and 482 (48.9%), 457 (46.3%), and 43 (4.4%) patients were Child-Pugh (CP) class A, B, and C, respectively. The most common serious adverse drug reactions (ADRs) were portal vein thrombosis (PVT), pancytopenia, and white blood cell count decreased, reported in 14 (1.36%), 3 (0.29%), and 2 (0.19%) patients, respectively. The incidence of ADRs was not higher in patients with CP class C or those undergoing retreatment cycles compared with other CP classes or the first treatment cycle, respectively. During the observation period of the first treatment cycle, 94.7% (889/939) of patients avoided preoperative platelet transfusion and 82.8% (741/895) of the patients met the responder criteria. This surveillance further supports the safety and effectiveness of lusutrombopag in a broad range of patients with CLD undergoing planned invasive procedures. This article is protected by copyright. All rights reserved.