Greenebaum Comprehensive Cancer Center, University of Maryland School of Medicine, Baltimore, MD, USA.
H. Lee Moffitt Cancer Center and Research Institute, Tampa, FL, USA.
Leuk Lymphoma. 2020 Aug;61(8):1901-1907. doi: 10.1080/10428194.2020.1751841. Epub 2020 Apr 19.
Thrombocytopenia occurs frequently in patients with myelodysplastic syndromes (MDS), and the survival of patients after failure of hypomethylating agents (HMAs) is poor. We conducted a trial of eltrombopag in patients with MDS, MDS/myeloproliferative neoplasm (MPN) or acute myeloid leukemia (AML) with 20-30% myeloblasts after HMA failure and mean baseline platelet count ≤ 50 × 10/L. Eltrombopag was escalated from 50 mg daily up to 200 mg daily. The primary objective was to determine the maximally tolerated dose (MTD). 37 patients were enrolled, and MTD was not reached. Responses were observed in 9 patients (24%), 2 achieving marrow CR with hematologic improvement (HI), 1 marrow CR without HI, and 6 HI. Median overall survival was 7.5 months. Eltrombopag was well-tolerated and yielded modest responses in heavily treated, predominantly higher-risk MDS patients after HMA failure. Future studies should focus on determining characteristics that predict response.
血小板减少症在骨髓增生异常综合征（MDS）患者中经常发生，且在低甲基化药物（HMAs）治疗失败后患者的生存率较差。我们在 HMAs 治疗失败且骨髓原始细胞比例为 20-30%、平均基线血小板计数 ≤ 50×10/L 的 MDS、MDS/骨髓增殖性肿瘤（MPN）或急性髓系白血病（AML）患者中开展了艾曲泊帕的试验。艾曲泊帕从 50mg 每日增加至 200mg 每日。主要目标是确定最大耐受剂量（MTD）。共纳入 37 例患者，未达到 MTD。9 例患者（24%）出现应答，2 例患者达到骨髓完全缓解伴血液学改善（HI），1 例患者达到骨髓完全缓解不伴 HI，6 例患者仅 HI。中位总生存期为 7.5 个月。艾曲泊帕耐受良好，在 HMAs 治疗失败后的、经大量治疗且主要为高风险 MDS 患者中产生了适度的应答。未来的研究应重点确定预测应答的特征。
