ArticleDrugs - real world outcomes2025
The Economic Burden, Epidemiological Insights, and Treatment Patterns of Wilson's Disease: A Real-World Study in Italy.
Article in Drugs - real world outcomes, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.
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Who cites it
2 citing papers in PubMed.
- Evaluation of Ultrasound-Based Parameters for the Assessment of Hepatic Steatosis and Fibrosis in Hungarian Wilson's Disease Patients.Diagnostics (Basel, Switzerland) · 2026Article
- [Research progress of inherited liver disease in 2025].Zhonghua gan zang bing za zhi = Zhonghua ganzangbing zazhi = Chinese journal of hepatology · 2026Review
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Authors and funding
16 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundWilson's disease (WD) is an autosomal recessive disorder characterized by abnormal copper accumulation, leading to multi-organ damage. The economic impact of WD in Italy has not been comprehensively studied.
aimsThe objectives were to determine the economic burden of WD, describe the demographic and clinical characteristics, and estimate the treatment distribution over time, using real-world data from Italy.
methodsA retrospective multicenter longitudinal chart review study was conducted across six Italian reference centers for WD management. Patients with at least one visit for WD in 2019-2020 were included. Demographic, clinical, and treatment data were collected from medical records, and healthcare resource utilization and related costs were estimated over a 12-month follow-up. Treatment patterns from diagnosis to 2021 were also described.
resultsA total of 243 patients with WD were included (183 adults, 60 minors). Median age at diagnosis was 11 years in adults and 7 years in minors. At enrollment, hepatic involvement was the most frequent clinical manifestation (84.7% of adults; 80% of minors), while 13.1% of adults and 16.7% of minors were asymptomatic. In adults, use of D-penicillamine and zinc decreased, while trientine tetrahydrochloride use increased over time. In minors, treatment remained stable. The average annual cost per patient was €10,394 for adults (mainly driven by pharmacological treatment) and €1351 for minors. Costs increased with the number of disease manifestations.
conclusionThe economic burden of WD in Italy varies with disease severity and treatment strategy, highlighting the need for optimized management practices to mitigate costs while enhancing patient care.
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