Thrombopoietin receptor agonist is more effective than platelet transfusion for chronic liver disease with thrombocytopenia, shown by propensity score matching

2020 ◽  
Vol 50 (9) ◽  
pp. 1062-1070 ◽  
Author(s):  
Yoshihiro Furuichi ◽  
Hirohito Takeuchi ◽  
Yuu Yoshimasu ◽  
Yoshitaka Kasai ◽  
Masakazu Abe ◽  
...  
Keyword(s):  
Liver Disease ◽  
Propensity Score ◽  
Chronic Liver Disease ◽  
Propensity Score Matching ◽  
Receptor Agonist ◽  
Platelet Transfusion ◽  
Chronic Liver ◽  
Thrombopoietin Receptor ◽  
Thrombopoietin Receptor Agonist

scholarly journals Avatrombopag and lusutrombopag for thrombocytopenia in people with chronic liver disease needing an elective procedure: a systematic review and cost-effectiveness analysis

2020 ◽  
Vol 24 (51) ◽  
pp. 1-220
Author(s):  
Nigel Armstrong ◽  
Nasuh Büyükkaramikli ◽  
Hannah Penton ◽  
Rob Riemsma ◽  
Pim Wetzelaer ◽  
...  
Keyword(s):  
Systematic Review ◽  
Liver Disease ◽  
Cost Effectiveness ◽  
Receptor Agonist ◽  
Platelet Transfusion ◽  
Rescue Therapy ◽  
Effectiveness Analysis ◽  
Thrombopoietin Receptor ◽  
Thrombopoietin Receptor Agonist ◽  
The Cost

Background There have been no licensed treatment options in the UK for treating thrombocytopenia in people with chronic liver disease requiring surgery. Established management largely involves platelet transfusion prior to the procedure or as rescue therapy for bleeding due to the procedure. Objectives To assess the clinical effectiveness and cost-effectiveness of two thrombopoietin receptor agonists, avatrombopag (Doptelet®; Dova Pharmaceuticals, Durham, NC, USA) and lusutrombopag (Mulpleta®; Shionogi Inc., London, UK), in addition to established clinical management compared with established clinical management (no thrombopoietin receptor agonist) in the licensed populations. Design Systematic review and cost-effectiveness analysis. Setting Secondary care. Participants Severe thrombocytopenia (platelet count of < 50,000/µl) in people with chronic liver disease requiring surgery. Interventions Lusutrombopag 3 mg and avatrombopag (60 mg if the baseline platelet count is < 40,000/µl and 40 mg if it is 40,000–< 50,000/µl). Main outcome measures Risk of platelet transfusion and rescue therapy or risk of rescue therapy only. Review methods Systematic review including meta-analysis. English-language and non-English-language articles were obtained from several databases including MEDLINE, EMBASE and Cochrane Central Register of Controlled Trials, all searched from inception to 29 May 2019. Economic evaluation Model-based cost-effectiveness analysis. Results From a comprehensive search retrieving 11,305 records, six studies were included. Analysis showed that avatrombopag and lusutrombopag were superior to no thrombopoietin receptor agonist in avoiding both platelet transfusion and rescue therapy or rescue therapy only, and mostly with a statistically significant difference (i.e. 95% confidence intervals not overlapping the point of no difference). However, only avatrombopag seemed to be superior to no thrombopoietin receptor agonist in reducing the risk of rescue therapy, although far fewer patients in the lusutrombopag trials than in the avatrombopag trials received rescue therapy. When assessing the cost-effectiveness of lusutrombopag and avatrombopag, it was found that, despite the success of these in avoiding platelet transfusions prior to surgery, the additional long-term gain in quality-adjusted life-years was very small. No thrombopoietin receptor agonist was clearly cheaper than both lusutrombopag and avatrombopag, as the cost savings from avoiding platelet transfusions were more than offset by the drug cost. The probabilistic sensitivity analysis showed that, for all thresholds below £100,000, no thrombopoietin receptor agonist had 100% probability of being cost-effective. Limitations Some of the rescue therapy data for lusutrombopag were not available. There were inconsistencies in the avatrombopag data. From the cost-effectiveness point of view, there were several additional important gaps in the evidence required, including the lack of a price for avatrombopag. Conclusions Avatrombopag and lusutrombopag were superior to no thrombopoietin receptor agonist in avoiding both platelet transfusion and rescue therapy, but they were not cost-effective given the lack of benefit and increase in cost. Future work A head-to-head trial is warranted. Study registration This study is registered as PROSPERO CRD42019125311. Funding This project was funded by the National Institute for Health Research (NIHR) Health Technology Assessment programme and will be published in full in Health Technology Assessment; Vol. 24, No. 51. See the NIHR Journals Library website for further project information.

Effectiveness of Lusutrombopag in Patients with Mild to Moderate Thrombocytopenia

2019 ◽  
Vol 38 (4) ◽  
pp. 329-334 ◽  
Author(s):  
Yuki Tsuji ◽  
Hideto Kawaratani ◽  
Koji Ishida ◽  
Daisuke Kaya ◽  
Takuya Kubo ◽  
...  
Keyword(s):  
Adverse Reaction ◽  
Platelet Count ◽  
Liver Disease ◽  
Chronic Liver Disease ◽  
Small Molecule ◽  
Receptor Agonist ◽  
Common Complication ◽  
Platelet Counts ◽  
Thrombopoietin Receptor ◽  
Thrombopoietin Receptor Agonist

Aims: Thrombocytopenia is a common complication among patients with chronic liver disease (CLD). To increase platelet counts, lusutrombopag, a small-molecule, second-generation thrombopoietin receptor agonist, was developed in September 2015. Lusutrombopag is mainly used in patients with platelet counts <50,000/µL. However, its usefulness in patients with platelet counts ≥50,000/µL remains unknown. We studied the effectiveness of lusutrombopag administration in patients with platelet counts of ≥50,000/µL. Methods: We evaluated 36 patients who received lusutrombopag for CLD. Changes in platelet counts were evaluated. A treatment response was defined as an increasing platelet count ≥20,000/µL from baseline after drug administration. The differences related to these changes between platelet counts ≥50,000 and <50,000/µL were evaluated. Results: Of the patients, 25 had platelet counts ≥50,000/µL. The increase in platelet count and the date in which it reached a maximum did not significantly differ between the groups. The effectiveness of lusutrombopag did not significantly differ between the groups. In both groups, no adverse reaction was observed during lusutrombopag administration. Conclusion: In this study, we showed the effectiveness of lusutrombopag, which had no complications. This study is the first to report that the effectiveness of lusutrombopag was the same for patients with platelet counts ≥50,000/µL and <50,000/µL.

scholarly journals Eltrombopag Use for Treatment of Thrombocytopenia in a Patient with Chronic Liver Disease and Portal Vein Thrombosis: Case Report

2020 ◽  
Vol 13 (2) ◽  
pp. 863-866
Author(s):  
Sabah E. Mohamed ◽  
Mohamed A. Yassin
Keyword(s):  
Liver Disease ◽  
Drug Use ◽  
Portal Vein ◽  
Chronic Liver Disease ◽  
Portal Vein Thrombosis ◽  
Receptor Agonist ◽  
Off Label ◽  
Vein Thrombosis ◽  
Thrombopoietin Receptor ◽  
Thrombopoietin Receptor Agonist

Off-label drug use refers to drug use beyond the specifications authorized for marketing [J Med Case Rep. 2014 Dec;8(1):303]. Eltrombopag is a thrombopoietin receptor agonist that has been used in treating thrombocytopenia due to chronic liver disease (CLD) as an off-label medication. Treatment of thrombocytopenia in patients with CLD constitute a real dilemma as the options are limited and some of them are invasive. However, thrombopoietin receptor agonist has been increasingly used for this purpose. Here we report a 34-year-old woman who has been diagnosed with CLD due to autoimmune hepatitis 20 years ago. Her condition was complicated with portal vein thrombosis, chronic thrombocytopenia, and variceal hemorrhage, and she has been listed as a candidate for liver transplantation. Given her high risk of bleeding, we started her on low dose of eltrombopag (25 mg daily) in order to maintain a platelet level of ≥50 × 103/µL. However, 1 year after initiation of the therapy, she developed left lower limb deep vein thrombosis.

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