Hydroxyurea Therapy in Sickle Cell Disease: Evaluating Healthcare Costs and Utilization in a Brazilian Tertiary Setting Open Access

• Hydroxyurea use in patients with sickle cell disease reduced acute care utilization but did not decrease total healthcare costs.

• Total health costs were likely higher due to persistent chronic care management, decreased adherence and later initiation of hydroxyurea.

Patients with sickle cell disease (SCD) treated with hydroxyurea, a disease-modifying agent, experience reduced hospitalizations, blood utilization, opioid use, and mortality, but how the reduced acute care utilization translates to cost-savings is understudied. This study aimed to test whether hydroxyurea reduces overall care costs for SCD patients in Brazil. Healthcare costs in Brazilian Reais (R$) were collected from patients with SCD who had at least one encounter between January 1, 2018, and June 30, 2018. The total median healthcare resource utilization included ambulatory clinic, emergency department (ED), and inpatient care. Patients were stratified by hydroxyurea use and medication adherence (MPR >50% vs. ≤50%). A total of 3331 active patients were included in the analysis, with 3032 having at least one encounter. 51% were female, and the median age was 15 years (range 0-76). 2212 (73.0%), had HbSS/Sβ°thalassemia, followed by HbSC (n=610, 20%) and HbSβ⁺thalassemia (n=148, 5%). 614 (20.2%) were prescribed hydroxyurea. Hydroxyurea-treated patients had higher median ambulatory visits, fewer ED visits, and fewer hospitalizations than those not on hydroxyurea. While hydroxyurea reduced acute care utilization, costs remained comparable to those of patients not using hydroxyurea suggesting that disease severity, use of expensive drugs, and chronic conditions are key factors driving costs requiring further analysis.