ArticleFrontiers in pharmacology2025
Cost-effectiveness analysis of enzyme replacement therapy for the treatment of Chinese patients with fabry disease: a Markov model.
Article in Frontiers in pharmacology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
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Who cites it
4 citing papers in PubMed.
- Cost-utility analysis of simultaneous initiation of finerenone and empagliflozin for type 2 diabetes mellitus complicated with chronic kidney disease.Frontiers in pharmacology · 2026Article
- Cost-utility analysis of loxenatide versus DPP-4 inhibitors in Chinese patients with type 2 diabetes at high cardiovascular risk.Frontiers in public health · 2026Article
- Long-Term Enzyme Replacement Therapy and Renal Outcomes in Fabry Disease: A Systematic Review and Meta-Analysis.Biomedicines · 2025Review
- Therapeutic landscape of Fabry disease: advances and challenges from classical strategies to emerging therapies.Frontiers in medicine · 2025Review
Corrections and comments
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Authors and funding
3 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Background: Fabry disease (FD) is an X-chromosome-linked genetic disorder. Currently, the main treatments for FD include disease-nonspecific and disease-specific treatments. Nonspecific treatment involves symptomatic management of organ involvement. On the other hand, disease-specific treatment is to regulate the activity of the corresponding enzymes, which is targeted. Among them, enzyme replacement therapy (ERT) is a classical therapy. Several studies have demonstrated the significant ameliorative effect of agalactosidase-α (ALTA-a) on cardiac and renal function in patients with FD. Despite the excellent clinical performance, there are limited pharmacoeconomic studies on ERT for FD worldwide. Objective: The aim of this study was to analyze the cost-effectiveness of ERT for FD in China from the perspective of the healthcare system. Methods: We constructed a five-state Markov model based on the disease characteristics of FD. The modeling period was 1 month. The time horizon was 3 years. The willingness-to-pay threshold was chosen as 1-3 times the gross national product (GDP) Results: The ICER value is ¥148071.95/QALY, which is between 1-3 times GDP Conclusion: ERT therapy is a cost-effective program compared to "No ERT" therapy.
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Registered trials
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