Supplementary Material for: Long-term Safety and Efficacy of Eltrombopag for Advanced Myelodysplastic Syndromes or Acute Myeloid Leukemia and Severe Thrombocytopenia: Results of the ASPIRE Extension Study

ASPIRE, a three-part, international, phase II trial (ClinicalTrials.gov identifier: NCT01440374), investigated eltrombopag efficacy and safety in patients with advanced myelodysplastic syndrome or acute myeloid leukemia, and grade 4 thrombocytopenia (<25 × 109 platelets/L). Approximately 30–65% of patients in this open-label extension phase experienced clinically relevant thrombocytopenic events; no conclusions could be made regarding long-term efficacy (non-randomized design, no placebo control), and survival rates may simply reflect advanced disease. Long-term safety was consistent with the double-blind phase and contrasted with earlier SUPPORT study findings in higher-risk patients, suggesting that eltrombopag may have a role in treating thrombocytopenia in patients with low-/intermediate-risk myelodysplastic syndrome.

ASPIRE是一项三部分组成的国际性II期临床试验（临床试验.gov标识符：NCT01440374），旨在评估艾曲泊帕（eltrombopag）治疗晚期骨髓增生异常综合征或急性髓系白血病合并4级血小板减少症（血小板计数＜25×10^9/L）患者的疗效与安全性。该开放标签扩展阶段中，约30%~65%的患者出现了临床相关的血小板减少事件；由于本研究采用非随机化设计且未设置安慰剂对照，无法针对长期疗效得出明确结论，且生存率数据仅可反映疾病的晚期进展状态。长期安全性特征与双盲阶段一致，且与此前针对高危患者开展的SUPPORT研究结果存在差异，这提示艾曲泊帕或可用于治疗低/中危骨髓增生异常综合征患者的血小板减少症。