A recent study examined the costs associated with using eltrombopag in first-line treatment of severe aplastic anemia and the results were presented at the 60th American Society of Hematology (ASH) Annual Meeting in San Diego, California.

The study analyzed outcomes and associated costs for a simulated population (1 million patients) comprising an eltrombopag plus immunosuppressive therapy (IST) arm compared with an IST-only arm. The IST consisted of antithymocyte globulin and cyclosporine A. Both arms received first-line treatment for 6 months, at which point patients were assessed for response.

Dosage and treatment duration, as well as rates of response, mortality, and adverse events were drawn from a phase 1/2 trial (ClinicalTrials.gov Identifier: NCT01623167), and cost estimates were derived from a variety of sources.

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Complete or partial response led to 6 months of maintenance therapy with low-dose cyclosporine A, while a lack of response in either cohort resulted in 6 months of second-line eltrombopag monotherapy. 

The response rate for the eltrombopag with IST arm was 94%, while the IST-only arm had a 66% response rate. The annual mortality risk was 0.3% lower for the eltrombopag with IST arm than with IST only.

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The authors reported that eltrombopag first-line therapy cost an additional $77,442 over 3 years. This total included cost savings due to the response rate with this drug precluding some second-line treatment costs.

From this simulated population of 1 million patients, the authors concluded that first-line eltrombopag therapy for severe aplastic anemia imparts a manageable cost increase, from a payer perspective, with improved response and survival outcomes over IST alone.


1. Tremblay G, Said Q, Cai B, et al. Comparing costs and consequences of eltrombopag plus IST versus IST alone for first-line treatment in severe aplastic anemia: results from a responder analysis. Poster presentation at: 60th ASH Annual Meeting and Exposition; December 1-4, 2018; San Diego, CA. Abstract 1041.