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.

Research design and 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: One hundred and sixty-two 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.