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Eltrombopag treatment promotes platelet recovery and reduces platelet transfusion for patients with post-transplantation thrombocytopenia.
Annals of Hematology ( IF 3.0 ) Pub Date : 2020-06-09 , DOI: 10.1007/s00277-020-04106-2
Fei Gao 1, 2, 3 , Xiaoyu Zhou 1, 2, 3 , Jimin Shi 1, 2, 3 , Yi Luo 1, 2, 3 , Yamin Tan 1, 2, 3 , Huarui Fu 1, 2, 3 , Xiaoyu Lai 1, 2, 3 , Jian Yu 1, 2, 3 , He Huang 1, 2, 3 , Yanmin Zhao 1, 2, 3
Affiliation  

Post-transplantation thrombocytopenia (PT) is a common and severe complication which usually leads to poor prognosis. Eltrombopag (EPAG), a novel oral thrombopoietin (TPO) receptor agonist, has shown promising effects in thrombocytopenia due to immune thrombocytopenic purpura (ITP) and refractory severe aplastic anemia (rSAA), while the effectiveness of EPAG for PT patients still needs to be evaluated. A total of 32 PT patients receiving EPAG were retrospectively analyzed between September 2017 and July 2019, including 15 patients with poor graft function (PGF) and 17 patients with secondary failure of platelet recovery (SFPR). To date, 21 (65.6%) patients achieved overall recovery (OR) and 14 (43.8%) patients achieved complete recovery (CR). Among responders, 18 (85.7%) patients discontinued or tapered the drug and 16 (76.2%) patients successfully maintained their best response. During the EPAG treatment, responders received much lower median platelet transfusion units than non-responders (11 vs. 95, P < 0.001). After a median follow-up time of 364 days (range, 24–842), the overall survival in these patients was 78.1% (100% for responders and 36.4% for non-responders, P < 0.001). In the univariate and multivariate analysis, PGF was identified as the independent risk factor for OR (P = 0.041, HR = 5.333). Megakaryocyte (Megk) amounts (P = 0.025, HR = 14.638) and splenomegaly (P = 0.042, HR = 11.278) were identified as independent risk factors for CR. Besides, PGF patients tended to take a longer time to achieve PR and CR than SFPR patients. In conclusion, our data suggest that EPAG can promote platelet recovery and reduce platelet transfusion in PT patients.



中文翻译:

Eltrombopag治疗可促进移植后血小板减少症患者的血小板恢复并减少血小板输注。

移植后血小板减少症(PT)是常见且严重的并发症,通常会导致不良的预后。Eltrombopag(EPAG)是一种新型口服血小板生成素(TPO)受体激动剂,由于免疫性血小板减少性紫癜(ITP)和难治性严重再生障碍性贫血(rSAA)而在血小板减少症中显示出令人鼓舞的效果,而EPAG对于PT患者的疗效仍需提高评估。在2017年9月至2019年7月之间,共对32例接受EPAG的PT患者进行了回顾性分析,包括15例移植物功能不良(PGF)和17例继发性血小板恢复失败(SFPR)的患者。迄今为止,共有21(65.6%)的患者实现了总体恢复(OR),有14(43.8%)的患者实现了完全恢复(CR)。在响应者中,有18(85.7%)名患者停用或逐渐减少了该药的使用率,另有16名(76。2%)患者成功地保持了最佳反应。在EPAG治疗期间,反应者的血小板输注中位数要比无反应者低得多(11比95,P <0.001)。在中位随访时间为364天(24-842)之后,这些患者的总生存率为78.1%(有反应者为100%,无反应者为36.4%,P <0.001)。在单变量和多变量分析中,PGF被确定为OR的独立危险因素(P = 0.041,HR = 5.333)。巨核细胞(Megk)的量(P = 0.025,HR = 14.638)和脾肿大(P = 0.042,HR = 11.278)被确定为CR的独立危险因素。此外,与SFPR患者相比,PGF患者倾向于花费更长的时间达到PR和CR。总之,我们的数据表明EPAG可以促进PT患者的血小板恢复并减少血小板输注。

更新日期:2020-06-09
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