Hydroxyurea Is Associated With Lower Costs of Care of Young Children With Sickle Cell Anemia

Abstract

BACKGROUND AND OBJECTIVE: In the BABY HUG trial, young children with sickle cell anemia randomized to receive hydroxyurea had fewer episodes of pain, hospitalization, and transfusions. With anticipated broader use of hydroxyurea in this population, we sought to estimate medical costs of care in treated versus untreated children. METHODS: The BABY HUG database was used to compare inpatient events in subjects receiving hydroxyurea with those receiving placebo. Unit costs were estimated from the 2009 MarketScan Multi-state Medicaid Database for children with sickle cell disease, aged 1 to 3 years. Inpatient costs were based on length of hospital stay, modified by the occurrence of acute chest syndrome, splenic sequestration, or transfusion. Outpatient expenses were based on the schedule required for BABY HUG and a “standard” schedule for 1- to 3-year-olds with sickle cell anemia. RESULTS: There were 232 hospitalizations in the subjects receiving hydroxyurea and 324 in those on placebo; length of hospital stay was similar in the 2 groups. Estimated outpatient expenses were greater in those receiving hydroxyurea, but these were overshadowed by inpatient costs. The total estimated annual cost for those on hydroxyurea ($11 072) was 21% less than the cost of those on placebo ($13 962; P = .038). CONCLUSIONS: Savings on inpatient care resulted in a significantly lower overall estimated medical care cost for young children with sickle cell anemia who were receiving hydroxyurea compared with those receiving placebo. Because cost savings are likely to increase with age, these data provide additional support for broad use of hydroxyurea treatment in this population.

Greater number of perceived barriers to hydroxyurea associated with poorer health‐related quality of life in youth with sickle cell disease

“…The drug's induced dose‐dependent increase in fetal hemoglobin (HbF) is largely responsible for its impact . Hydroxyurea markedly reduces symptoms, morbidity and mortality, improves HRQL, decreases healthcare cost, and may protect against cumulative disease burden. Despite these therapeutic benefits, adherence in adolescents and young adults with SCD is often poor …”

Health‐related quality of life and adherence to hydroxyurea in adolescents and young adults with sickle cell disease

“…Hydroxyurea, the only medication approved by the U.S. Food and Drug Administration for SCD, reduces morbidity and mortality and lowers healthcare utilization . Despite these benefits, hydroxyurea adherence is suboptimal and underutilization remains a challenge .…”

Update on the use of hydroxyurea therapy in sickle cell disease

“…All but one RCT 15 and its secondary analyses [16][17][18][19][20] enrolled patients who had previous sickle cell-related symptoms or complications. Thirty studies enrolled children, 13 enrolled adults, and 5 enrolled both.…”