Cost analysis of the impact of caplacizumab in the treatment of acquired thrombotic thrombocytopenic purpura from a US hospital perspective

Pollissard, Laurence; Leinwand, Brian I.; Fournier, Marie; Pham, Huy P.

doi:10.1080/13696998.2021.1992413

Cited by 3 publications

(1 citation statement)

References 38 publications

(38 reference statements)

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“…Owing to its high price (12), In clinical trials, the use of caplacizumab is strictly controlled, adheres to strict dosing and withdrawal protocols, and is limited to use in trial hosting centers (9). Currently, there is a great controversy about the safety and efficacy of caplacizumab in the treatment of TPP, Therefore, we conducted this systematic review and meta-analysis of all available randomized controlled trials (RCTs) and summarized their results to evaluate the efficacy and safety of caplacizumab in patients with TTP.…”

Section: Introductionmentioning

confidence: 99%

Comparison of the efficacy and safety of caplacizumab versus placebo in thrombotic thrombocytopenic purpura: a meta-analysis and systematic review based on randomized controlled trials

Chen¹,

Li²,

Xiao³

et al. 2022

Ann Transl Med

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Background: We conducted this meta-analysis to investigate the efficacy and safety of caplacizumab in patients with thrombotic thrombocytopenic purpura (TTP). TTP is a potentially fatal disorder characterized by systemic microvascular thrombosis.Methods: Randomized controlled trials (RCTs) were conducted from PubMed, Embase, Cochrane Library and Web of Science, China National Knowledge Infrastructure (CNKI), VIP and Wanfang databases. RCTs of caplacizumab treatment for TPP were mainly included. Data from eligible studies were extracted and analyzed using relative effect sizes versus placebo use. The Cochrane bias assessment tool was used to assess the risk of bias of included studies, and the assessment results were presented graphically in Revman5.3.Results: Four RCTs with a total of 416 patients were included, all of which were of high quality.Caplacizumab was associated with improvements in platelet counts normalization time [weighted mean difference (WMD) −1.18, 95% confidence interval (CI): −2.55 to 0.19, I 2 =69.9%, P=0.036], plasma exchange (PE) time (WMD −2.97, 95% CI: −4.44 to −1.50, I 2 =8.2%, P=0.163) and hospital stay (WMD −2.88, 95% CI: −4.56 to −1.21, I 2 =48.7%, P=0.036). In addition, the occurrence of adverse events was also investigated.The difference in mortality between the two groups was not statistically significant [relative risk (RR) 0.56, 95% CI: 0.18 to 1.72, I 2 =22.7%, P=0.275], relapse (RR 0.68, 95% CI: 0.13 to 3.49, I 2 =78.3%, P=0.01), or major thrombotic events (RR 1.01, 95% CI: 0.65 to 1.57, I 2 =43.4%, P=0.151).Conclusions: Caplacizumab shortens the platelet normalization time, PE time, and hospital stay in patients with TTP, and did not significantly increase the risk of adverse events. These results indicate that caplacizumab treatment provides significant benefits to patients with TTP. Even though this is evidence from RCTs, few original studies were included, so more multicenter RCTs are required.

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Section: Introductionmentioning

confidence: 99%

Comparison of the efficacy and safety of caplacizumab versus placebo in thrombotic thrombocytopenic purpura: a meta-analysis and systematic review based on randomized controlled trials

Chen¹,

Li²,

Xiao³

et al. 2022

Ann Transl Med

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Snakebite-associated thrombotic microangiopathy: a spotlight on pharmaceutical interventions

Noutsos

2023

Expert Review of Clinical Pharmacology

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Global Health Resource Utilization and Cost-Effectiveness of Therapeutics and Diagnostics in Immune Thrombotic Thrombocytopenic Purpura (TTP)

Butt

Allen

Purcell

et al. 2023

JCM

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In this review, we examine the current landscape of health resource utilization and cost-effectiveness data in the care of patient populations with immune thrombotic thrombocytopenic purpura. We focus on the therapeutic (therapeutic plasma exchange, glucocorticoids, rituximab, caplacizumab) and diagnostic (ADAMTS13 assay) health technologies employed in the care of patients with this rare disease. Health resource utilization and cost-effectiveness data are limited to the high-income country context. Measurement of TTP-specific utility weights in the high-income country context and collection of health resource utilization data in the low- and middle-income country settings would enable an evaluation of country-specific quality-adjusted life expectancy and cost-effectiveness of these therapeutic and diagnostic health technologies. This quantification of value is one way to mitigate cost concerns where they exist.

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Cost analysis of the impact of caplacizumab in the treatment of acquired thrombotic thrombocytopenic purpura from a US hospital perspective

Cited by 3 publications

References 38 publications

Comparison of the efficacy and safety of caplacizumab versus placebo in thrombotic thrombocytopenic purpura: a meta-analysis and systematic review based on randomized controlled trials

Comparison of the efficacy and safety of caplacizumab versus placebo in thrombotic thrombocytopenic purpura: a meta-analysis and systematic review based on randomized controlled trials

Snakebite-associated thrombotic microangiopathy: a spotlight on pharmaceutical interventions

Global Health Resource Utilization and Cost-Effectiveness of Therapeutics and Diagnostics in Immune Thrombotic Thrombocytopenic Purpura (TTP)

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