ArticleAdvances in therapy2022
Cost-Effectiveness Analysis of Pharmacokinetic-Guided Prophylaxis Versus Standard Prophylaxis in Adults with Severe Hemophilia A in China.
Article in Advances in therapy, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 7 papers, 1 of them a synthesis that pooled it.
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Who cites it
7 citing papers in PubMed, 1 synthesis or guideline pooled it, 8 citations in OpenAlex.
- A Systematic Review of Modelling Approaches in Economic Evaluations of Treatments for Inherited Bleeding Disorders.Applied health economics and health policy · 2026Pooled it
- Pharmacokinetic-guided versus weight-guided coagulation factor replacement in hemophilia patients undergoing arthroplasty: a retrospective real-world study on perioperative bleeding risk and economic outcomes.Frontiers in medicine · 2026Article
- Exploring the Relationship Between Personalization of Care and Participation in Sport Activities Among People with Severe Hemophilia A Across Europe: Post Hoc Analysis of the CHESS II Study.Journal of blood medicine · 2025Article
- Letter to the Editor Regarding "Cost-Effectiveness Analysis of Pharmacokinetic-Guided Prophylaxis Versus Standard Prophylaxis in Adults with Severe Hemophilia A in China".Advances in therapy · 2024Article
- A Response to: Letter to the Editor Regarding "Cost-Effectiveness Analysis of Pharmacokinetic-Guided Prophylaxis Versus Standard Prophylaxis in Adults with Severe Hemophilia A in China".Advances in therapy · 2024Article
- Personalized Prophylaxis with myPKFiTMedicina (Kaunas, Lithuania) · 2023Observational
- In Silico Assessment of Limited Blood Sampling Strategies for Individualised Pharmacokinetic-guided Dosing of Efanesoctocog Alfa in Haemophilia A Patients.Haemophilia : the official journal of the World Federation of HemophiliaArticle
Corrections and comments
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Authors and funding
3 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
introductionMultiple 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.
methodsA 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.
resultsA 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.
conclusionPK-guided individualized prophylaxis appeared to be a dominant treatment compared with standard prophylaxis, with slightly higher QALYs but lower total costs.
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