Safety and effectiveness of lusutrombopag in Japanese chronic liver disease patients with thrombocytopenia undergoing invasive procedures: Interim results of a postmarketing surveillance

Abstract

Aim Lusutrombopag is approved for the treatment of thrombocytopenia in chronic liver disease patients undergoing invasive procedures. This real‐world surveillance assesses the safety and effectiveness of lusutrombopag in Japan. Methods This ongoing, multicenter, prospective, real‐world surveillance is collecting data from case report forms between October 2016 and May 2021. Interim data up to September 2018 were used to evaluate safety (adverse events and adverse drug reactions [ADRs]) and effectiveness (proportion of patients avoiding preoperative platelet transfusion and change in platelet count from baseline). Results The safety analysis set included 331 patients. The mean baseline platelet count was 46.2 ± 13.7 × 109/L. Of 377 invasive procedures, radiofrequency ablation (110 procedures, 29.2%) was the most frequent. The mean time from starting lusutrombopag treatment to invasive procedure was 12.3 days. Incidences of serious adverse events and ADRs were 8.76% and 3.32%, respectively. Six cases (1.81%) of portal vein thrombosis were considered serious adverse events; of these, four cases (1.21%) were classified as serious ADRs. Of 300 patients who underwent an invasive procedure (excluding those with platelet transfusion refractoriness), 282 (94.0%) avoided preoperative platelet transfusion. In patients with platelet measurements before and after lusutrombopag administration who did not undergo platelet transfusion, the mean maximum change in platelet count from baseline was 41.7 ± 31.4 × 109/L (range, −6 to 276; n = 286). All patients receiving second (n = 20) and third (n = 1) treatments avoided preoperative platelet transfusion without developing any ADRs. Conclusions This real‐world surveillance further supports the safety and effectiveness of lusutrombopag in patients with chronic liver disease undergoing invasive procedures.

Effect of three or more treatments with lusutrombopag in patients with cirrhotic thrombocytopenia: A retrospective single‐center study

“…16 The effect of three treatments with lusutrombopag was shown in the interim results of a post-marketing surveillance study. 17 However, to the best of our knowledge, studies in which lusutrombopag was given three or more times have not been reported, and this is the first report that describes the administration of lusutrombopag more than three times in patients with CLD.…”

Is platelet monitoring during 7‐day lusutrombopag treatment necessary in chronic liver disease patients with thrombocytopenia undergoing planned invasive procedures? A phase IIIb open‐label study

“…The time course of PC in the non-naïve patients was similar to the profiles in lusutrombopag-naïve patients from previous reports. 19,20 The incidence of TEAEs was 75.0%. There were no thrombosis-related or bleeding-related AEs in non-naïve patients.…”

“…18 Furthermore, only a few reports describe the effect of lusutrombopag in patients being retreated with lusutrombopag. 19,20 This open-label study aimed to determine whether platelet monitoring and the discontinuation criteria could be applied at a reduced frequency without affecting clinical outcomes. Additionally, the efficacy, safety, and pharmacokinetics (PK) were evaluated in lusutrombopag-naïve and lusutrombopag retreatment (non-naïve) CLD patients with thrombocytopenia undergoing planned invasive procedures.…”

Efficacy of Lusutrombopag for Thrombocytopenia in Patients with Chronic Liver Disease Scheduled to Undergo Invasive Procedures

“…Lusutrombopag induces platelet production by acting on the transmembrane domain of the human TPO receptor and activating the signaling pathway in the same way as endogenous TPO (7). Lusutrombopag treatment efficiently increases platelet counts and makes it possible for patients with CLD and thrombocytopenia to receive invasive procedures (8)(9)(10)(11)(12). In this study, we assessed the efficacy of repeated lusutrombopag treatment and the factors associated with a response to lusutrombopag.…”