Discussion
deaths
COMPLICATION
In this study, we evaluated the efficacy and safety of eltrombopag in Chinese patients with r/r SAA. Overall, 70% (14/20) of patients met the primary endpoint of HR rate at Week 26, with 64% (9/14) of these responders having at least a bi-lineage response. The HR at Week 13 was met in 65% (13/20) of patients, and the clinical benefit of reducing the transfusion need was also observed in both studied time points, suggesting that patients receiving eltrombopag experience an early response. The reduction in transfusion need is associated with clinical benefits such as reduced mortality and length of hospitalization, as well as reduced management costs. AEs reported were generally mild, with no treatment-related deaths reported. There were no new safety signals identified throughout this study. A major complication associated with long-term treatment of SAA is clonal evolution, which globally occurs in up to 15% of patients after ISTThe reported results in this study show a similar improvement trend as in previously published data, where the treatment with eltrombopag was associated with multilineage clinical response in patients with r/r SAAIn summary, although this study is limited by the small patient population, it is the first prospective study to demonstrate the safety and efficacy of treatment with eltrombopag in a Chinese adult patient population with r/r SAA. These findings are promising and highlight the potential therapeutic benefit of eltrombopag in this specific population with high unmet need. 