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Updated: Jul 23, 2025
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Cost-Effectiveness of Hydroxyurea for Sickle Cell Anemia in a Low-Income African Setting: A Model-Based Evaluation of
David Teigen1, Robert O Opoka2,3, Phillip Kasirye2,4
1Health Economics, Leadership and Translational Ethics Research Group, Department of Global Public Health and Primary Care, Centre for International Health, University of Bergen, P.O. Box 7804, 5020, Bergen, Norway. david.teigen@student.uib.no.
Insights
Hydroxyurea treatment for sickle cell anemia (SCA) in Uganda is cost-effective, especially at maximum tolerated doses (MTD). This approach improves quality of life and reduces blood product consumption for children with SCA.
Area of Science:
- Hematology
- Pharmacoeconomics
- Public Health in Sub-Saharan Africa
Background:
- Sickle cell anemia (SCA) poses a significant disease burden in sub-Saharan Africa (SSA), with high mortality rates among undiagnosed/untreated children.
- Hydroxyurea is the sole disease-modifying therapy proven effective and feasible for SCA in SSA.
- The economic implications of hydroxyurea use in SCA have not been previously quantified.
Purpose of the Study:
- To estimate the incremental cost-effectiveness of hydroxyurea, administered as a fixed-dose or maximum tolerated dose (MTD) regimen, compared to standard SCA care without hydroxyurea.
- To analyze the health economic impact from a healthcare provider's perspective.
Main Methods:
- A discrete-event simulation model was utilized, incorporating cost data from a pediatric sickle cell clinic in Kampala, Uganda.
- The model projected costs, disability-adjusted life years (DALYs) averted, and blood product consumption for patients aged 9 months to 18 years.
- Cost-effectiveness was calculated as the ratio of incremental costs to incremental DALYs averted, with a 3% annual discount rate. Sensitivity, scenario, and price threshold analyses were performed to ensure robustness.
Main Results:
- Hydroxyurea at MTD averted 1.37 DALYs and saved US$191 per patient compared to no hydroxyurea.
- A fixed-dose regimen averted 0.80 DALYs per patient at an incremental cost of US$2.
- The MTD strategy significantly reduced blood product consumption (11.2 units) compared to the fixed-dose strategy (9.1 units).
Conclusions:
- Hydroxyurea, particularly at MTD, is likely to enhance the quality of life and decrease blood product usage for pediatric SCA patients in Uganda.
- The MTD regimen for hydroxyurea is projected to be a cost-effective treatment for SCA, applicable across SSA.
- Implementing hydroxyurea therapy offers substantial net savings per patient, while simultaneously mitigating SCA-related morbidity and mortality.
Background And Objective:
The disease burden of sickle cell anemia (SCA) in sub-Saharan African (SSA) countries is substantial, with many children dying without an established diagnosis or proper treatment. The global burden of SCA is increasing each year, making therapeutic intervention a high priority. Hydroxyurea is the only disease-modifying therapy with proven feasibility and efficacy suitable for SSA; however, no one has quantified the health economic implications of its use. Therefore, from the perspective of the health care provider, we estimated the incremental cost-effectiveness of hydroxyurea as a fixed-dose regimen or maximum tolerated dose (MTD) regimen, versus SCA care without hydroxyurea.
Methods:
We estimated the cost of providing outpatient treatment at a pediatric sickle cell clinic in Kampala, Uganda. These estimates were used in a discrete-event simulation model to project mean costs (2021 US$), disability-adjusted life years (DALYs), and consumption of blood products per patient (450 mL units), for patients between 9 months and 18 years of age. We calculated cost-effectiveness as the ratio of incremental costs over incremental DALYs averted, discounted at 3% annually. To test the robustness of our findings, and the impact of uncertainty, we conducted probabilistic and one-way sensitivity analyses, scenario analysis, and price threshold analyses.
Results:
Hydroxyurea treatment averted an expected 1.37 DALYs and saved US$ 191 per patient if administered at the MTD, compared with SCA care without hydroxyurea. In comparison, hydroxyurea at a fixed dose averted 0.80 DALYs per patient at an incremental cost of US$ 2. The MTD strategy saved 11.2 (95% CI 11.1-11.4) units of blood per patient, compared with 9.1 (95% CI 9.0-9.2) units of blood per patient at the fixed-dose alternative.
Conclusions:
Hydroxyurea at MTD is likely to improve quality of life and reduce the consumption of blood products for children with SCA living in Uganda. Compared with a fixed dose regimen, treatment dosing at MTD is likely to be a cost-effective treatment for SCA, using realistic ranges of hydroxyurea costs that are relevant across SSA. Compared with no use of the drug, hydroxyurea could lead to substantial net savings per patient, while reducing the disease morbidity and mortality and increasing quality of life.
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