Objective To evaluate the effectiveness, safety, and economy of hydroxyurea for the treatment of transfusion⁃dependent β⁃thalassemia. Methods A retrospective study was conducted involving 80 patients with transfusion⁃dependent β⁃thalassemia as the research subjects, therein the hydroxyurea group (40 cases) received conventional blood transfusion combined with hydroxyurea for treatment, while the conventional treatment group (40 cases) received conventional blood transfusion for treatment. The efficacy and safety were compared between patients of the two groups. A Markov model was constructed for cost⁃utility analysis, and economy was compared between the two groups. Results After treatment, the hemoglobin level in the hydroxyurea group was higher than that before treatment and in the conventional treatment group (P<0.05). However, no statistically significant difference was observed between the two groups in terms of total effective rate of treatment, transfusion frequency, transfusion volume (P>0.05), and safety was similar. The results of cost⁃utility analysis revealed that compared with the conventional treatment group, the hydroxyurea group had an incremental cost of 1559.13 RMB per patient and an incremental utility of 0.20 quality adjusted life year (QALY), resulting in an incremental cost effectiveness ratio of 7522.47 RMB/QALY. The results indicated that hydroxyurea demonstrated cost⁃utility advantage to a certain extent when the willingness⁃to⁃pay value exceeded 7522.4 RMB. Probabilistic sensitivity analysis confirmed the stability of the findings of cost⁃utility analysis. Conclusion For patients with transfusion⁃dependent β⁃thalassemia, the addition of hydroxyurea to conventional blood transfusion therapy offers clinical and cost⁃utility advantages compared with conventional treatment regimen to a certain extent.

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