Objective: To evaluate the efficacy and safety of eltrombopag in post-HSCT thrombocytopenia. Methods: A total of 10 patients who underwent post-HSCT thrombocytopenia at Peking University center, who had been treated with eltrombopag, were retrospectively evaluated. Results: Of the 10 cases, 5 males and 5 females with a median of 34 years old (range, 17-54 years), 5 patients were acute myeloid leukemia, 3 with acute lymphoid leukemia and 2 with severe aplastic anemia. Nine patients had undergone haplo-identical donor transplantation, and one patient was a matched related recipient. All patients had failed prior treatment for thrombocytopenia before eltrombopag started. The median time when eltrombopag started was 221 days (range, 73-917 days) after transplantation. Five patients (50%) had achieved CR. The cumulative incidence of 30-day CR was 35.7%. The median time to platelet recovery ≥ 50 × 109/L without transfusion support was 16 days (range, 10-56 days). At the last follow-up, three of the patients with CR had withdrawal eltrombopag and remained normal platelet counts. No patients experienced drug-related adverse events. Conclusion: Eltrombopag is effective and well tolerated in patients with refractory post-HSCT thrombocytopenia.
目的: 观察艾曲波帕治疗异基因造血干细胞（allo-HSCT）移植后难治性血小板减少的疗效及安全性。
方法: 2015年5月1日至2016年5月1日，应用艾曲波帕治疗10例allo-HSCT后难治性血小板减少（糖皮质激素等治疗无效）患者。10例患者中，男、女各5例，中位年龄34(17~54）岁。急性髓系白血病5例，急性淋巴细胞白血病3例，重型再生障碍性贫血2例。同胞HLA配型全相合移植1例，单倍型相合移植9例。allo-HSCT至接受艾曲波帕治疗的中位时间为221(73~917) d。艾曲波帕剂量为50~75 mg/d（口服）。
结果: 5例（50%）患者获得完全有效（PLT≥50×109/L且脱离血小板输注），用药30 d累积完全有效率为35.7%，治疗后达到第1次PLT≥50×109/L的中位时间为16(10~56) d。至随访截止，3例完全有效患者已分别停药39、342、84 d, PLT均≥100×109/L。5例患者无效。未发生药物相关不良反应。
结论: 艾曲波帕对于部分allo-HSCT后难治性血小板减少患者有效且耐受性良好，值得进一步研究。