The impact of adherence to iron chelation therapy on the health-related quality of life and cost-utility in transfusion-dependent thalassemia patients in Malaysia

Abstract

Adherence to iron chelation therapy (ICT) is a vital component of the management strategy for transfusion-dependent thalassemia (TDT) patients as inconsistent use of ICT may cause in prolonged iron overload, consequently leading to health risks and affecting the health-related quality of life (HRQoL). The study systematically reviewed the global impact of ICT adherence on TDT patients’ health outcomes following Preferred Reporting Items for Systematic Reviews and Meta-Analysis (PRISMA). A cross-sectional analysis in Malaysia was conducted to examine the rate of adherence to ICT and its impact on the HRQoL using self-reported measures. The cost-utility analysis (CUA) of iron chelators was performed from societal perspectives. The systematic review included 20 studies, revealing that ICT adherence rates ranged from 20.93% to 75.3% globally. According to the systematic review, patients on oral chelators had higher adherence rates (100% and 51.6%) than those on injectables (36.4%), but no significant association between ICT type and adherence was found (p=0.074). Better adherence correlated with lower serum ferritin levels, reduced risks of liver, cardiac, endocrine complications, and enhanced HRQoL. Meanwhile, the cross-sectional study found that adherence was suboptimal at 46.91% among 162 TDT participants. Adherent patients demonstrating significantly higher HRQoL (mean SF- 36 score 79.21) compared to nonadherent counterparts (mean SF-36 score 69.47) (p=0.00). Additionally, female gender (p=0.032), employment status (p=0.003), and age (p=0.050) correlated positively with HRQoL. The average annual cost of TDT management per adherent patient of $7,773.26 (MYR34,202.35) was slightly higher but not significantly different compared to non-adherent patient of $7,643.33 (MYR33,630.59). Adherent patients exhibited a higher SF-6D utility value (0.783), while nonadherent patients had a lower value (0.733), although not statistically significant. In comparison to the nonadherent group, the adherent group exhibited an ICER of $2,598.90 (MYR11,435.20) per QALY gained, suggesting adherent group are potentially cost-effective strategy. The study emphasized the need for interventions, such as counselling and support programs and highlighted the economic impact of adherence to ICT to optimize the cost-effectiveness of ICT. In summary, it provided practical insights that assist policymakers, healthcare providers, and researcher in developing focused initiations and optimizing the allocation of resources for TDT management in Malaysia