Ma Y R, Huang X J, Mo X D, Han W, Yan C H, Chen Y, Ji Y, Chen Y Y, Wang Y, Zhang X H, Liu K Y, Xu L P
Institute of Hematology, People's Hospital, Peking University, Beijing 100044, China.
Zhonghua Xue Ye Xue Za Zhi. 2016 Dec 14;37(12):1065-1069. doi: 10.3760/cma.j.issn.0253-2727.2016.12.011.
To evaluate the efficacy and safety of eltrombopag in post-HSCT thrombocytopenia. A total of 10 patients who underwent post-HSCT thrombocytopenia at Peking University center, who had been treated with eltrombopag, were retrospectively evaluated. 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 × 10/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. Eltrombopag is effective and well tolerated in patients with refractory post-HSCT thrombocytopenia.
评估艾曲泊帕治疗异基因造血干细胞移植（HSCT）后血小板减少症的疗效和安全性。回顾性评估了北京大学中心10例接受HSCT后血小板减少症且接受过艾曲泊帕治疗的患者。10例患者中，男性5例，女性5例，年龄中位数为34岁（范围17 - 54岁），5例为急性髓系白血病，3例为急性淋巴细胞白血病，2例为重型再生障碍性贫血。9例患者接受了单倍体相合供者移植，1例患者为全相合相关受体。所有患者在开始使用艾曲泊帕之前，先前治疗血小板减少症均失败。艾曲泊帕开始使用的中位时间为移植后221天（范围73 - 917天）。5例患者（50%）达到完全缓解（CR）。30天CR的累积发生率为35.7%。在无输血支持的情况下，血小板恢复至≥50×10⁹/L的中位时间为16天（范围10 - 56天）。在最后一次随访时，3例达到CR的患者停用了艾曲泊帕，血小板计数仍保持正常。无患者发生药物相关不良事件。艾曲泊帕对难治性HSCT后血小板减少症患者有效且耐受性良好。
