Real-world analysis and cost-effectiveness of iron chelation therapy adherence in transfusion-dependent thalassemia

Background Adherence to iron chelation therapy (ICT) is essential for preventing iron overload complications and optimizing health outcomes in transfusion-dependent thalassemia (TDT) patients. This study evaluates the cost-effectiveness of ICT adherence in Malaysia. Methods A cross-sectional analysis was conducted at a tertiary hospital. Adherence was measured using the Malaysia Medication Adherence Assessment Tool, and health utility values were assessed with SF-6D. Based on the societal perspective, costs and quality-adjusted life years (QALYs) were compared between adherent and non-adherent patients, with incremental cost-effectiveness ratio (ICER) calculations and sensitivity analyses. Results 162 adult TDT patients were recruited. Adherent patients (n = 76) achieved higher QALYs (0.783 vs. 0.733) but incurred slightly higher annual costs ($7,773.26 vs. $7,643.33). The ICER of $2,598.90 per QALY remained below Malaysia's willingness-to-pay threshold ($5,441.16), confirming cost-effectiveness. Sensitivity analyses indicated that cost variations significantly influenced the ICER, while utility values had minimal impact. Conclusions The findings underscore the economic and clinical benefits of ICT adherence, advocating for targeted strategies to enhance compliance. Future research should explore long-term cost implications and intervention strategies to improve adherence, ensuring sustainable thalassemia management.