Dataset Information

Economic Evaluation of Rituximab + Recombinant Human Thrombopoietin vs. Rituximab for the Treatment of Second-Line Idiopathic Thrombocytopenic Purpura in China.

ABSTRACT: Objective: This study aimed to compare the economic evaluation of recombinant human thrombopoietin+rituximab (rhTPO + RTX) vs. RTX as second-line treatment for adult patients with immunologic thrombocytopenic purpura in China. Methods: The Markov model was used in our research. The response rate and relapse rate data were derived from two clinical trials and one retrospective study. Cost and utility values were derived from published literature, a third-party database, and healthcare documents. In addition, one-way sensitivity analysis and probabilistic sensitivity analysis were performed to observe the stability of the model and data source. Results: In the Markov model, compared with RTX, rhTPO+RTX yielded an additional 0.04 QALYs, with an incremental cost of 2,802 USD. The ICER was 69,097 USD/QALY. According to the results from the one-way sensitivity analysis, complete response of rhTPO+RTX, utility of complete response and response of RTX were the main drivers in the model. The results from the probabilistic sensitivity analysis demonstrated that there was a 100% probability that rhTPO+RTX was not cost-effective vs. RTX alone at a threshold of $10,805/QALY and an 84% probability at a threshold of $32,415/QALY. Conclusion: RTX+rhTPO was not more cost-effective than RTX alone as second-line treatment for adult patients with immunologic thrombocytopenic purpura in China.

SUBMITTER: Rui M

PROVIDER: S-EPMC8012846 | biostudies-literature | 2021

REPOSITORIES: biostudies-literature

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Economic Evaluation of Rituximab + Recombinant Human Thrombopoietin vs. Rituximab for the Treatment of Second-Line Idiopathic Thrombocytopenic Purpura in China.

Rui Mingjun M Wang Yingcheng Y Fei Zhengyang Z Shang Ye Y Ma Aixia A Li Hongchao H

Frontiers in medicine 20210318

<b>Objective:</b> This study aimed to compare the economic evaluation of recombinant human thrombopoietin+rituximab (rhTPO + RTX) vs. RTX as second-line treatment for adult patients with immunologic thrombocytopenic purpura in China. <b>Methods:</b> The Markov model was used in our research. The response rate and relapse rate data were derived from two clinical trials and one retrospective study. Cost and utility values were derived from published literature, a third-party database, and healthca ...[more]

PMID: 33816534

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Project description:BackgroundChronic idiopathic thrombocytopenic purpura (ITP) is an acquired autoimmune disorder that is characterized predominantly by a low platelet count. Thrombopoietin (TPO) receptor agonists increase production of platelets by stimulating the TPO receptor in people with chronic ITP.ObjectivesTo determine the efficacy and safety of TPO receptor agonists in chronic ITP patients.Search strategyWe searched MEDLINE (from 1950 to March 2011), EMBASE (from 1974 to March 2011), and the Cochrane Central Register of Controlled Trials (CENTRAL) (The Cochrane Library 2011, Issue 3) to identify all randomized trials in chronic ITP. We also contacted authors of included studies and TPO receptor agonists manufacturers.Selection criteriaRandomized controlled trials (RCTs) comparing TPO receptor agonists alone, or in combination with other drugs, to placebo, no treatment, other drugs, splenectomy or another TPO receptor agonist in patients with chronic ITP.Data collection and analysisTwo review authors independently screened papers, extracted data and assessed the risk of bias in the included studies.Main resultsSix trials with 808 patients were included. Five studies compared TPO receptor agonists with placebo (romiplostim: 100, eltrombopag: 299, placebo: 175); one study compared TPO receptor agonists with standard of care (SOC) (romiplostim: 157; SOC: 77). SOC included a variety of therapies, such as glucocorticoid, anti-D immune globulin, intravenous immune globulin, rituximab, azathioprine, and so on. Overall survival, one of our primary outcomes, was not studied by these RCTs and we could not estimate number needed to treat (NNT). Another primary outcome, improving significant bleeding events, did not reveal any significant differences between the TPO receptor agonists group and the control group (placebo or SOC) (versus placebo risk ratio (RR) 0.48, 95% confidence interval (CI) 0.20 to 1.15; versus SOC RR 0.49, 95% CI 0.15 to 1.63).For secondary outcomes, TPO receptor agonists statistically significantly improved overall platelet response (versus placebo RR 4.06, 95% CI 2.93 to 5.63; versus SOC RR 1.81, 95% CI 1.37 to 2.37), complete response (versus placebo RR 9.29, 95% CI 2.32 to 37.15) and durable response (versus placebo RR 14.16, 95% CI 2.91 to 69.01). There was a significant reduction in overall bleeding events (WHO grades 1 to 4) when compared to placebo (RR 0.78, 95% CI 0.68 to 0.89), but not when compared to SOC(RR 0.97, 95% CI 0.75 to 1.26).Total adverse events (Grades 1 to 5) were not statistically significantly different between the treatment and control groups(both placebo and SOC) (versus placebo RR 1.04, 95% CI 0.95 to 1.15; versus SOC RR 0.97, 95% CI 0.75 to 1.26). Total serious adverse events (Grade 3 and higher adverse events) were increased when patients receiving treatment with SOC (RR 0.61, 95% CI 0.40 to 0.92), but not receiving treatment with placebo (RR 0.92, 95% CI 0.61 to 1.38).There are selective and performance biases because of open-label and inadequate allocation.Authors' conclusionsThere was currently no evidence to support that TPO receptor agonists are effective in chronic ITP. Compared to placebo or SOC, despite significantly increased platelet response, there was no evidence to demonstrate that TPO receptor agonists did improve significant bleeding events in chronic ITP. The effect on overall survival awaits further analysis. Although long-term studies are lacking, current data demonstrated adverse effects of TPO receptor agonists were similar to that of placebo and SOC.

Dataset Information

Economic Evaluation of Rituximab + Recombinant Human Thrombopoietin vs. Rituximab for the Treatment of Second-Line Idiopathic Thrombocytopenic Purpura in China.

Publications

Economic Evaluation of Rituximab + Recombinant Human Thrombopoietin vs. Rituximab for the Treatment of Second-Line Idiopathic Thrombocytopenic Purpura in China.

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