Lin Ying, Zhang Rong-Dong, Lin Zeng-Hua, Chen Qi, Chen Ren-Li
Department of Hematology, Ningde Municipal Hospital of Ningde Normal University, Ningde 352100, Fujian Province, China,E-mail：
Department of Hematology, Ningde Municipal Hospital of Ningde Normal University, Ningde 352100, Fujian Province, China.
Zhongguo Shi Yan Xue Ye Xue Za Zhi. 2023 Jun;31(3):823-829. doi: 10.19746/j.cnki.issn.1009-2137.2023.03.030.
To compare the efficacy of eltrombopag combined with cyclosporine A (CsA) and CsA alone in patients with transfusion-dependent non-severe aplastic anemia (TD-NSAA).
The clinical data of 76 patients with treatment-naive TD-NSAA in Ningde Municipal Hospital of Ningde Normal University and Affiliated Hospital of Nantong University from December 2017 to June 2021 were retrospectively analyzed. Among them, 45 cases were treated with eltrombopag combined with CsA, and 31 patients with compatible baseline characters were treated with CsA alone. The efficacy of patients between the two groups was compared, and the factors affecting the curative effects were also analyzed.
There were significant differences in hematological response (HR) and complete response(CR) rates between the two groups at 3, 6, 12 months, and follow-up endpoint of treatment (<0.05). With the prolongation of eltrombopag treatment time, the curative effect increased gradually, and the patients achieved more CR and HR rates by the end of the follow-up period. Simultaneously, with the increase in the maximum stable dose of eltrombopag, the HR rate increased gradually. The megakaryocyte count in eltrombopag group was higher than that in control at 6 and 12 months (<0.05). Compared with the control group, the median time of platelet transfusion independence in eltrombopag group was more shorter (=0.018), and the median platelets transfusion volume was lower (=0.009). At 3, 6, 12 months after eltrombopag, the change of platelet in eltrombopag group was higher than that in the control group (<0.05). Analysis of related factors affecting the efficacy showed that sex, age, iron overload, platelet count before treatment had no effect on the efficacy, and the median maximum stable dosage and the administration period for eltrombopag were related to the curative effect. The patients of eltrombopag group experienced adverse events of varying degrees, but the reactions were mild and mostly tolerated.
Eltrombopag can effectively improve the hematopoietic response and promote platelet recovery for TD-NSAA patients with relatively more residual hematopoietic cells, and it is safe and well tolerated.
比较艾曲泊帕联合环孢素A（CsA）与单用CsA治疗输血依赖型非重型再生障碍性贫血（TD - NSAA）患者的疗效。
回顾性分析2017年12月至2021年6月在宁德师范学院附属宁德市医院和南通大学附属医院初治的76例TD - NSAA患者的临床资料。其中45例采用艾曲泊帕联合CsA治疗，31例具有匹配基线特征的患者单用CsA治疗。比较两组患者的疗效，并分析影响疗效的因素。
两组在治疗3、6、12个月及随访终点时的血液学缓解（HR）率和完全缓解（CR）率存在显著差异（<0.05）。随着艾曲泊帕治疗时间的延长，疗效逐渐提高，随访期末患者达到更高的CR率和HR率。同时，随着艾曲泊帕最大稳定剂量的增加，HR率逐渐升高。艾曲泊帕组在6个月和12个月时的巨核细胞计数高于对照组（<0.05）。与对照组相比，艾曲泊帕组血小板输注独立的中位时间更短（=0.018），血小板输注量中位数更低（=0.009）。艾曲泊帕治疗后3、6、12个月，艾曲泊帕组血小板变化高于对照组（<0.05）。影响疗效的相关因素分析显示，性别、年龄、铁过载、治疗前血小板计数对疗效无影响，艾曲泊帕的中位最大稳定剂量和给药周期与疗效相关。艾曲泊帕组患者经历了不同程度的不良事件，但反应轻微，大多可耐受。
对于造血细胞残留相对较多的TD - NSAA患者，艾曲泊帕可有效改善造血反应并促进血小板恢复，且安全、耐受性良好。
