Blood transfusions are used to treat sickle cell disease (SCD) and other anemias. However, frequent transfusions may lead to iron overload, which can cause organ dysfunction and death. Deferoxamine, deferasirox, and deferiprone are iron chelating therapies (ICTs) used to treat iron overload.

OBJECTIVES:

To evaluate whether oral deferiprone is a cost-effective treatment option in Canada for iron overload in SCD and other anemias compared to oral deferasirox and parenteral deferoxamine.

METHODS:

The cost-utility analysis utilized a semi-Markov structure with 3 health states (On ICT; Not On ICT; and Dead), from the perspective of a Canadian publicly-funded healthcare payer with monthly cycles and lifetime horizon. The model assumed equivalent efficacy for the 3 therapies based on an indirect treatment comparison. The model was structured to track decline in renal function over time using a mixed model for repeated measures analysis of medical record data.

RESULTS:

In the probabilistic base-case analysis, the overall average discounted costs for deferiprone, deferoxamine, and deferasirox in 2022 Canadian dollars were $3,416,325, $2,682,313, and $2,569,891 and overall average discounted quality-adjusted life-years (QALYs) were 17.73, 11.48, and 12.51, respectively, over a lifetime horizon. The pairwise incremental cost-effectiveness ratio (ICER) result for deferiprone compared to deferoxamine was $117,356 per (QALY) gained, with an 99.52% probability of being cost-effective at a willingness-to-pay threshold of $200,000. Comparing deferiprone to deferasirox yielded an ICER of $162,153 per QALY gained, with an 84.68% probability of being cost-effective at a willingness-to-pay threshold of $200,000. All ICERs showed a positive incremental cost benefit. When considering a 10- and 20–year time horizon, deferiprone provided more benefits and fewer costs compared to deferasirox and deferoxamine.

CONCLUSIONS:

Deferiprone contributed to higher life-year gains as well as higher QALY gains and is a cost-effective treatment option for iron overload in SCD and other anemias compared to other approved ICTs in Canada.