阿伐曲泊帕治疗肝肾功能异常的严重型再生障碍性贫血8例分析

Avatrombopag for the treatment of 8 cases of SAA with abnormal hepatic and renal function

目的评估因肝肾功能异常不耐受强化免疫抑制治疗(IST)联合艾曲泊帕治疗的严重型再生障碍性贫血(SAA)患者,换用阿伐曲泊帕治疗的疗效及耐受性。方法对2020年10月至2021年7月间江苏省人民医院收治的8例SAA,因肝肾功能异常不耐受IST联合艾曲泊帕,改用阿伐曲泊帕20 mg/d治疗,评估治疗前后血象变化、血栓事件、肝肾功能等指标。结果阿伐曲泊帕中位治疗时间4(1~6)个月,1例完全缓解,3例部分缓解,2例血红蛋白及血小板改善,1例中性粒细胞及血小板改善,1例患者因治疗时间短未纳入疗效评估。中位起效时间68.5(28~107)d,自身对照显示中性粒细胞计数(P=0.004)、血红蛋白水平(P=0.005)及血小板计数(P=0.016)均较治疗前显著升高。阿伐曲泊帕治疗期间,所有患者无血栓事件发生,肝肾功能指标均维持正常范围内。结论阿伐曲泊帕对IST联合艾曲泊帕不耐受的SAA患者有一定疗效,耐受性良好。

Objective To evaluate the efficacy and tolerance of avatrombopag in patients with severe aplastic anemia(SAA),who failed to immunosuppressive therapy(IST)combined with eltrombopag due to abnormal hepatic and renal function.Methods Eight cases of SAA patients with hepatic and renal insufficiency from October 2020 to July 2021 in Jiangsu Province Hospital were enrolled,treated with avatrombopag(20 mg/d).Complete blood counts,biochemical indexes and adverse events during avatrombopag treatment were recorded.Results At a median follow-up of 4 months(range 1~6 months),1 achieved complete response and 3 achieved partial response.2 cases reached erythroid/platelet response and 1 case reached neutrophil/platelet response.1 patient were not included in efficacy evaluation due to short treatment time.The median response time was 68.5 days(range 28-107 days).Neutrophil count(P=0.004),hemoglobin level(P=0.005)and and platelet count(P=0.016)increased significantly after treatment with avatrombopag.No thrombotic events and hepatic or renal insufficiency happened during use of avatrombopag.Conclusions Avatrombopag has certain efficacy and good tolerance in SAA patients with hepatic or renal function insufficiency,which is worthy of further exploration.