艾曲波帕治疗异基因造血干细胞移植后难治性血小板减少的临床研究  

Eltrombopag for refractory thrombocytopenia in patients with allogeneic hematopoietic stem cell transplantation

作　　者：马艳茹[1] 黄晓军[1] 莫晓冬[1] 韩伟[1] 闫晨华[1] 陈瑶[1] 纪宇[1] 陈育红[1] 王昱[1] 张晓辉[1] 刘开彦[1] 许兰平[1] 

机构地区：[1]北京大学人民医院、北京大学血液病研究所,100044

出　　处：《中华血液学杂志》2016年第12期1065-1069,共5页Chinese Journal of Hematology

基　　金：国家自然科学基金重点项目（81530046）;国家自然科学基金面上项目（81370666）

摘　　要：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l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≥50×10^9/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× 10^9/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、84d,PLT均≥100×10^9/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.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× 10^9/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.

关 键 词：造血干细胞移植 血小板减少 艾曲波帕 治疗 临床研究性 

分 类 号：R558.2[医药卫生—血液循环系统疾病] R457.7[医药卫生—内科学]