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A cost-effectiveness analysis of lusutrombopag for thrombocytopenia in patients with chronic liver disease in Japan.

ABSTRACT:

Background and aim

Thrombocytopenia is a frequent hematological condition in chronic liver disease (CLD) patients increasing the risk of bleeding in patients undergoing invasive procedures. Without an alternative, clinical guidelines recommended the use of platelet transfusion (PT) prior to procedure to prevent this bleeding risk. Lusutrombopag (LUSU), an orally active, small-molecule thrombopoietin receptor agonist, was developed as an alternative to PT. The objective of this study was to evaluate a cost-effectiveness of LUSU as a potential alternative to PT in Japan.

Methods

A cost-effectiveness analysis of LUSU relative to PT was conducted by a simulation model consisting of a decision tree combined to Markov model. Quality-adjusted life years (QALYs) were used as an indicator of efficacy, and the analysis was conducted from the Japanese public healthcare payer's perspective. The time horizon of the analysis was 50 years (a lifetime) and the discount rate was set at 2%.

Results

LUSU gained 6.1803 QALYs with an expected lifetime costs of 2 380 219 JPY compared to PT with 6.1712 QALYs gained and expected lifetime costs of 2 382 908 JPY. Thus, LUSU was deemed dominant compared with PT. Based on probabilistic analyses, the chance of LUSU being dominant and the incremental cost-effectiveness ratio being below 5 million JPY/QALY was estimated at 51.8% and 78.3%, respectively, demonstrating the robustness of the results.

Conclusions

LUSU was evaluated as an efficacious and cost-saving treatment option for Japanese CLD patients with thrombocytopenia who required a planned invasive procedure compared with PT and economically should be considered as an alternative treatment.

SUBMITTER: Miki N

PROVIDER: S-EPMC8341178 | biostudies-literature |

REPOSITORIES: biostudies-literature

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Project description:BackgroundChronic liver disease (CLD) is often complicated by severe thrombocytopenia (platelet count < 50,000/µL). Platelet transfusion has been a gold standard for increasing the platelet count to prevent hemorrhagic events in such patients. Lusutrombopag, a thrombopoietin receptor agonist, can increase the platelet count in such patients when invasive procedures are scheduled. Former studies on lusutrombopag included patients with a platelet count of > 50,000/µL at baseline: the proportions of patients who did not require platelet transfusion were 84-96%, which might be overestimated.MethodsThe efficacy and safety of lusutrombopag were retrospectively investigated in CLD patients with platelet count of < 50,000/µL, a criterion for platelet transfusion, in real-world settings. We examined the proportion of patients who did not require platelet transfusion in 31 CLD patients, which exceeded a minimum required sample size (21 patients) calculated by 80% power at a significance level of 5%. Lusutrombopag, 3 mg once daily, was administered 8-18 days before scheduled invasive procedures.ResultsAmong 31 patients who received lusutrombopag, 23 patients (74.2%) patients showed a platelet count of ≥ 50,000/µL (Group A) and did not require platelet transfusion. The remaining 8 patients (25.8%) did not reached platelet ≥ 50,000/µL (Group B). The means of platelet increase were 38,000/µL and 12,000/µL in groups A and B, respectively. A low platelet count at baseline was a characteristic of patients in group B. Among 13 patients who repeatedly used lusutrombopag, lusutrombopag significantly increased the platelet count as the initial treatment. When all repeated uses of lusutrombopag were counted among these 13 patients, platelet transfusion was not required in 82.1% (23/28) of treatments. Although one patient showed portal thrombosis after lusutrombopag treatment, the thrombosis was disappeared by anticoagulant treatment for 35 days. The degree of platelet increase with lusutrombopag was larger than that in their previous platelet transfusion.ConclusionsThe proportion of patients who did not require platelet transfusion was 74.2%, which is smaller than that in former studies which included CLD patients with a platelet count of > 50,000/µL. However, lusutrombopag is effective and safe for CLD patients with a platelet count of < 50,000/µL.

Project description:IntroductionLusutrombopag is an oral thrombopoietin receptor agonist (TPO-RA). Clinical trials have shown lusutrombopag's efficacy in reducing need for preoperative platelet transfusion in patients with chronic liver disease (CLD) and severe thrombocytopenia. This analysis assessed efficacy and safety of lusutrombopag in patients with severe thrombocytopenia and CLD undergoing planned invasive procedures.MethodsAn electronic database search (through 1 December 2020) identified three randomised, placebo-controlled, double-blind clinical trials comparing lusutrombopag with placebo in patients with CLD and platelet count below 50 × 109/L scheduled to undergo a procedure with a perioperative bleeding risk. A random-effects meta-analysis examined treatment effect, with Cochrane Collaboration's tool assessing risk of bias.ResultsThe meta-analysis included 343 (lusutrombopag 3 mg, n = 173; placebo, n = 170) patients. More patients met the criteria for treatment response (platelet count at least 50 × 109/L and increase of at least 20 × 109/L from baseline anytime during the study) with lusutrombopag versus placebo (risk ratio [RR] 6.39; 95% confidence interval [CI] 3.69, 11.07; p < 0.0001). The primary efficacy outcome, proportion of patients requiring no platelet transfusion and no rescue therapy for bleeding for at least 7 days post procedure, was achieved by more patients treated with lusutrombopag versus placebo (RR 3.42; 95% CI 1.86, 6.26; p = 0.0001). The risk of any bleeding event was significantly lower with lusutrombopag compared to placebo (RR 0.55; 95% CI 0.32, 0.95; p = 0.03); conversely, thrombosis event rates were similar between lusutrombopag and placebo (RR 0.79; 95% CI 0.19, 3.24; p = 0.74).ConclusionThis meta-analysis showed that treatment of severe thrombocytopenia with lusutrombopag in patients with CLD prior to a planned invasive procedure was efficacious and safe in increasing platelet counts, avoiding the need for platelet transfusions, and reducing risk of bleeding, thereby enhancing the certainty of evidence supporting the efficacy and safety of lusutrombopag.