Background Recognition of the burden of sickle cell anaemia (SCA) in sub-Saharan African (SSA) countries is increasing, with few therapies available for clinical management. Hydroxyurea is the only disease-modifying therapy that has proven feasible and clinically efficacious in low-income countries in SSA; however, the health economic implications of its use in this region have not been quantified. Thus, we examined the incremental cost-effectiveness of hydroxyurea given as a fixed-dose regimen or at the maximum tolerated dose (MTD).

Methods We estimated the cost of outpatient treatment at a specialized sickle cell clinic in Kampala, Uganda, from a provider’s perspective. These estimates were used in a discrete-event simulation model to project mean costs (US$), disability-adjusted life years (DALYs), and consumption of blood products per patient (450 ml units). We calculated cost-effectiveness as the ratio of incremental costs over incremental DALYs averted, discounted at 3% annually.

Findings For Ugandan patients under the age of 18, we predicted that hydroxyurea at the MTD would avert an expected 1.38 DALYs and save US$ 111 per patient compared to standard care, while hydroxyurea at a fixed dose would avert 0.81 DALYs per patient at an incremental cost of US$ 21. Additionally, we predicted that the fixed-dose alternative would save 9.2 (95% CI 9.0–9.3) units of whole-blood equivalents per patient, while the MTD strategy saved 11.3 (95% CI 11.1–11.4) units of blood per patient.