The following is a summary of the “Avatrombopag is effective in patients with chemoradiotherapy-induced aplastic anemia: a single-center, retrospective study,” published in the January 2023 issue of Hematology by Chi, et al.

The results can be challenging to manage when a malignant tumor is combined with aplastic anemia (AA). Therefore, they set out to determine if thrombopoietin receptor agonist avatrombopag (AVA) may help individuals who had developed AA due to chemoradiation. Patients with malignant tumors diagnosed with AA following radiation and chemotherapy and consented to AVA at Peking Union Medical College Hospital between September 2020 and October 2021 were included in this retrospective analysis. 

The median age was 60 (20-71) years, with 34 total participants, 13 male (38.2%). Overall response rates (ORRs) at 1, 3, and 6 months were 32.4 percent, 55.9 percent, and 58.8 percent, respectively, with corresponding complete response rates (CRRs) of 5.9 percent, 14.7 percent, and 23.5 percent at a median of 8 (6-18) months of follow-up. Around 3 months (from 1 to 6) was the most common response time. No clonal evolution was observed, despite the fact that 15.0% of patients had a recurrence while followed. Around 17 percent of individuals experienced mild side effects without stopping medication treatment. 

There was a recurrence of tumors in 17.6% of patients by the end of the follow-up period. Around 3 individuals had a recurrence of their tumor, and 1 patient suffered a brain hemorrhage and died. The ORR and CRR did not correlate with eltrombopag before AVA (P > 0.05), but they did rise with increasing total exposure to AVA (P = 0.011), and the threshold for response to AVA was a cumulative dosage of > 3,000 mg (P = 0.013). Patients with AA who received chemoradiotherapy also responded well to AVA, and there may be a correlation between a larger dose of AVA and a better response.