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Jialian Li


cost-utility analysis, β-thalassemia major, deferoxamine, deferiprone, deferasirox


Background: The four most commonly used chelation regimens for ?-thalassemia major patients in China are a combination therapy of deferoxamine and deferiprone (DFO+DFP), deferoxamine(DFO) monotherapy, deferiprone(DFP) monotherapy and deferasirox(DFX) monotherapy. Such patients use iron chelators their whole lives, resulting in enormous treatment costs. This study analyses the cost-utility of these four regimens from the Chinese healthcare system perspective.

Methods: A Markov decision model was used over a 70-year time horizon and was populated using clinical data from a systematic literature review. We obtained utility data from local and previous research. Costs were estimated using Chinese national sources.

Results: From the base-case analysis results, DFP was the most cost-effective chelation regimen, followed by DFO+DFP, DFO and DFX. DFP had a 99.60%, 78.10% and 64.40% likelihood of being cost-effective versus DFX, DFO and DFO+DFP, respectively, at a payment threshold of 193,932.00 CNY/QALY.

Conclusions: DFP was the most cost-effective chelation regimen for ?-thalassemia major patients, followed by DFO+DFP, DFO and DFX. Using DFP as the primary treatment regimen may potentially result in cost-savings and QALY gains for the Chinese healthcare system. To increase these benefits, the Chinese government and clinicians should lower drug costs, increase drug utility and reduce mortality and morbidity. Changes in influential parameters easily affect the results of DFO+DFP versus DFP and of DFP versus DFO; clinicians should focus on such parameters and adjust the regimens accordingly.


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