Dataset Information

Cost-Effectiveness Analysis of Pharmacokinetic-Guided Prophylaxis Versus Standard Prophylaxis in Adults with Severe Hemophilia A in China.

ABSTRACT:

Introduction

Multiple studies have demonstrated the effectiveness of pharmacokinetic (PK)-guided individualized prophylaxis with human coagulation factor VIII (FVIII) compared with standard prophylaxis, but no studies have evaluated the economics of PK-guided prophylaxis in China. Hence, we conducted this study to assess the cost-effectiveness of PK-guided prophylaxis with recombinant FVIII (rFVIII) versus standard prophylaxis in Chinese adult patients with severe hemophilia A.

Methods

A discrete event simulation model was developed to simulate 10,000 patients with hemophilia A who received rFVIII treatment over a 1-year time horizon. The standard prophylaxis rFVIII dose was 30 IU/kg by intravenous injection. The PK-guided prophylaxis dosage was adjusted for each patient to maintain FVIII trough level at 1-5 IU/dL. Dosing interval for both approaches was kept fixed at 48 h. The health outcomes included annual joint bleed rate (AJBR) and quality-adjusted life years (QALYs). The model considered the costs of drug. Incremental cost-effectiveness ratio (ICER) was estimated and scenario analysis was performed.

Results

A total of 94.3% of patients receiving PK-guided individualized prophylaxis achieved the goal of maintaining the trough concentration at 1-5 IU/dL compared with 62.7% on standard prophylaxis. AJBR and QALYs gained in PK-guided and standard prophylaxis were 1.527 vs 1.601, and 0.8384 vs 0.8383, respectively. Costs of drug prophylaxis and costs of treatment for bleeding events in PK-guided prophylaxis (148,641.47 USD; 4546.43 USD) were lower than those in standard prophylaxis (159,620.93 USD; 4753.39 USD). An average saving of USD 11,186.47 was obtained by the PK-guided approach. The prophylaxis treatment scenarios were the most influential factors.

Conclusion

PK-guided individualized prophylaxis appeared to be a dominant treatment compared with standard prophylaxis, with slightly higher QALYs but lower total costs.

SUBMITTER: Gu C

PROVIDER: S-EPMC9309149 | biostudies-literature | 2022 Aug

REPOSITORIES: biostudies-literature

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Publications

Cost-Effectiveness Analysis of Pharmacokinetic-Guided Prophylaxis Versus Standard Prophylaxis in Adults with Severe Hemophilia A in China.

Gu Congling C Huang Hui H Han Yi Y

Advances in therapy 20220630 8

<h4>Introduction</h4>Multiple studies have demonstrated the effectiveness of pharmacokinetic (PK)-guided individualized prophylaxis with human coagulation factor VIII (FVIII) compared with standard prophylaxis, but no studies have evaluated the economics of PK-guided prophylaxis in China. Hence, we conducted this study to assess the cost-effectiveness of PK-guided prophylaxis with recombinant FVIII (rFVIII) versus standard prophylaxis in Chinese adult patients with severe hemophilia A.<h4>Method ...[more]

PMID: 35768709

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Dataset Information

Cost-Effectiveness Analysis of Pharmacokinetic-Guided Prophylaxis Versus Standard Prophylaxis in Adults with Severe Hemophilia A in China.

Introduction

Methods

Results

Conclusion

Publications

Cost-Effectiveness Analysis of Pharmacokinetic-Guided Prophylaxis Versus Standard Prophylaxis in Adults with Severe Hemophilia A in China.

Similar Datasets

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