Successful treatment of Acquired Amegakaryocytic Thrombocytopenia with eltrombopag and immunosuppressant
Successful treatment of Acquired Amegakaryocytic Thrombocytopenia with eltrombopag and immunosuppressant
复制标题
艾曲波帕和免疫抑制剂成功治疗获得性无巨核细胞性血小板减少症
DOI：
10.1080/09537104.2021.2012140
复制
发表时间：
2021
期刊：
影响因子：
3.3
通讯作者：
Jie Yin
中科院分区：
文献类型：
--
作者：
Hong Tian;Danqing Kong;Yun Li;Chengyuan Gu;Ziqiang Yu;Zhaoyue Wang;Depei Wu;Jie Yin
获得性无巨核细胞血小板减少症（AAMT）是一种极其罕见的血液系统疾病，标准治疗策略尚未建立。我们在此描述了两例对艾曲波帕和免疫抑制剂完全有效的 AAMT 病例。患者 1 对类固醇、IVIG 和重组人血小板生成素 (rhTPO) 耐药。患者 2 对高剂量类固醇、IVIG、rhTPO 和利妥昔单抗没有反应。此外，他的 AAMT 在 5 个月内进展为再生障碍性贫血。两名患者均服用艾曲波帕和免疫抑制剂，随后获得长期缓解，且无明显副作用。我们的研究结果表明，这种组合可以成为 AAMT 中有价值的替代方案。
Acquired amegakaryocytic thrombocypenia (AAMT) is an extremely rare hematologic disorder and standard treatment strategy has not been established. We described herein two cases of AAMT who were fully responded to eltrombopag and immunosuppressant. Patient 1 was refractory to steroid, IVIG and recombinant human thrombopoietin (rhTPO). Patient 2 did not respond to high dosage of steroid, IVIG, rhTPO and rituximab. Moreover, his AAMT progressed to aplastic anemia in 5 months. Both patients took eltrombopag and immunosuppressant, then they achieved long-term remission without obvious side effects. Our findings suggest that this combination can be a valuable alternative in AAMT.