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

Hitoshi Yoshiji; Junto Suzuki; Masafumi Imasaki; Eri Tsukimura; Makiko Miyano; Masayuki Kurosaki

doi:10.1111/hepr.13945

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

Hepatol Res. 2023 Nov;53(11):1105-1116. doi: 10.1111/hepr.13945. Epub 2023 Aug 11.

Authors

Hitoshi Yoshiji¹, Junto Suzuki², Masafumi Imasaki², Eri Tsukimura³, Makiko Miyano³, Masayuki Kurosaki⁴

Affiliations

¹ Department of Gastroenterology, Nara Medical University, Kashihara, Nara, Japan.
² Pharmacovigilance Department, Shionogi & Co., Ltd., Chuo-ku, Osaka, Japan.
³ PMS & Pharmacoepidemiology Department, Shionogi Pharmacovigilance Center Co., Ltd., Chuo-ku, Osaka, Japan.
⁴ Department of Gastroenterology and Hepatology, Musashino Red Cross Hospital, Musashino, Tokyo, Japan.

PMID: 37497574
DOI: 10.1111/hepr.13945

Abstract

Aim: Lustrombopag has been approved for the treatment of thrombocytopenia in patients with chronic liver diseases 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.

Methods: 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.

Results: The safety analysis set included 1033 (100.0%), 130 (12.6%), and 14 (1.4%) patients who received one or more, two or more, and three or more treatment cycle(s), and 482 (48.9%), 457 (46.3%), and 43 (4.4%) patients who were Child-Pugh class A, B, and C, respectively. The most common serious adverse drug reactions were portal vein thrombosis, pancytopenia, and white blood cell count decrease, reported in 14 (1.36%), three (0.29%), and two (0.19%) patients, respectively. The incidence of adverse drug reactions was not higher in patients with Child-Pugh class C or those undergoing retreatment cycles compared with other Child-Pugh 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.

Conclusions: This surveillance study further supports the safety and effectiveness of lusutrombopag in a broad range of patients with chronic liver diseases undergoing planned invasive procedures.

Clinical trial registration: JapicCTI-163432.

Keywords: liver diseases; lusutrombopag; post-marketing; product surveillance; thrombocytopenia; thrombopoietin.

Grants and funding

Shionogi & Co., Ltd.