Eltrombopag actively promotes hematological response in association with immunosuppressive therapy (IST) as a first-line treatment for severe aplastic anemia (SAA). This study analyzes the cost-effectiveness of the combination of IST plus eltrombopag in the first line in SAA from the Brazilian public healthcare system perspective.

We built a hybrid decision tree comprising three months of treatment and a Markov model, defined by five mutually exclusive health states (free from drug treatment; demanding drug treatment [relapse]; hematopoietic stem cell transplantation [HSCT]; clonal evolution; and death). Life-years gained was adopted as a primary endpoint for effectiveness. Economic endpoints considered direct medical costs only. Resource utilization patterns were extracted from data available and validated by an expert panel. Treatment strategies were compared through the incremental cost-effectiveness ratio (ICER), and results were reported in Brazilian Real (R$).

The combination of eltrombopag plus IST showed a higher cost of treatment than IST alone (R$ 248,964.02 vs. R$ 132,695.59, respectively). However, the combination saved resources related to HSCT, relapse, lack of response, transfusion requirements and end of life. This resulted in an incremental cost of R$ 116.268 per patient treated with IST alone. Moreover, eltrombopag produced an additional survival of approximately 1.4 years per patient, resulting in an ICER of R$ 83.412 per life-year gained.

Despite some technical limitations for cost collection in the Brazilian healthcare, Eltrombopag associated with IST appears cost-effective for the treatment in the first line of SAA from the Brazilian public healthcare system perspective.

Anemia, Aplastic; Eltrombopag; Immunosuppression; Cost-Benefit Analysis.