A case report of elderly patient with refractory severe aplastic anemia by treatment of avatrombopag in combination with tacrolimus

Objective  To summarize the clinical effect and adverse reactions of avatrombopag combined with tacrolimus in the treatment of elderly patient with refractory severe aplastic anemia.

Methods  An elderly patient with refractory severe aplastic anemia was treated with avatrombopag and tacrolimus, and the literature review on the mechanism of action and safety of avatrombopag and tacrolimus was conducted.

Results  The patient was treated with a combination of cyclosporine, testosterone undecanoate, and itraconazole for 6 months without success, and developed liver and kidney dysfunction. The medication was discontinued, and the patient was treated with a combination of avatrombopag (20 mg, once per day) and tacrolimus (1 mg, twice per day). After 9 months of treatment, the patient's platelet count increased to 54×10⁹/L, the hemoglobin increased to 124 g/L, the white blood cells increased to 8.23×10⁹/L, liver and kidney function also returned to normal level, and no other adverse reactions such as thrombotic events were observed. After treatment, CD4/CD8 decreased, CD8⁺ T cells increased, and the proportion of Treg cells increased as well.

Conclusion  The combination of avatrombopag and tacrolimus is effective and well tolerated in the treatment of elderly patient with refractory aplastic anemia, but the long-term follow-up observation and continued accumulation of cases are still needed in the future.