OBJECTIVES: Novel therapies for managing vaso-occlusive crises in pediatric sickle cell disease (SCD) have emerged in recent years. However, evidence of their cost-effectiveness is lacking. This study performed a cost-effectiveness analysis of off-label hydroxyurea, hydroxyurea (Siklos), L-glutamine, crizanlizumab and voxelotor, as compared to chronic blood transfusions as standard of care for managing SCD in adolescents.

METHODS: Applying a decision tree model, we compared economic and clinical outcomes of 4 novel FDA-approved products including off-label hydroxyurea over one-year time horizon in a hypothetical cohort of adolescent SCD patients from a US payer perspective. The main outcome is incremental cost per SCD-related hospitalizations averted (ICER). Model inputs were derived from drug clinical trials, published literature and average wholesale prices (AWP) from Wolters Kluwer Medi-Span®. Costs were converted to 2019 US dollars using the consumer price index.

RESULTS: The incremental costs and incremental SCD-related hospitalizations averted were –($7,951) and 0.4/year for hydroxyurea, $23,793 and 0.4/year for L-glutamine, $35,986 and 0.2/year Siklos, $107,283 and 0.3/year crizanlizumab, and $141,883 and 0.5/year for Voxelotor. Generic hydroxyurea dominated standard of care, Siklos and Crizanlizumab. The ICER of L-glutamine and Voxelotor, when compared to generic hydroxyurea was $1,368,627 and $1,615,456 respectively per SCD hospitalization averted. L-glutamine dominated Siklos and Crizanlizumab, but not standard of care. At a willingness-to-pay threshold of $100,000 per SCD-related hospitalization averted, only L-glutamine was deemed cost-effective compared to standard of care with ICER of $55,816 per averted hospitalization.

CONCLUSIONS: Generic hydroxyurea and L-glutamine may be cost-effective interventions for preventing hospitalizations in adolescents with sickle cell disease.