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The following is a summary of “Long-term outcome of Eltrombopag with first-line immunosuppressive therapy for newly diagnosed severe Aplastic Anemia,” published in the August 2024 issue of Hematology by Yokota et al.

Researchers conducted a retrospective study verifying if adding eltrombopag (EPAG) to rabbit anti-thymocyte globulin (ATG)-based immunosuppressive therapy (IST) for newly diagnosed severe aplastic anemia (SAA) improves patient outcomes and influences the risk of clonal evolution (CE). 

They collected and reviewed data from 101 patients aged 15-65 undergoing initial IST.

The results showed that no significant difference in age, sex, or severity was found between the EPAG (n-20) and non-EPAG (n=81) groups. The median EPAG treatment duration was 16.1 months (range: 0.6-41.1 months). Half a year after initiating IST, the complete response rate significantly improved in the EPAG group (P<0.01). However, allogeneic stem cell transplantation rates at 2 years and overall survival (OS) were similar between groups (allo-SCT, P=0.31; OS, P=0.64). There was no increase in grade 3-4 adverse events or cumulative incidence of CE (P=0.96).

Investigators concluded that initial efficacy was significantly better in the EPAG group. While adding EPAG didn’t decrease the need for allo-SCT, there was no rise in the incidence of clonal evolution with long-term use of EPAG.

Source: thejh.org/index.php/jh/article/view/1289/850