[Effect of Eltrombopag on Response to Immunosuppressive Therapy in Patients with Transfusion-Dependent Non-Severe Aplastic Anemia].

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 (P<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 (P<0.05). Compared with the control group, the median time of platelet transfusion independence in eltrombopag group was more shorter (P=0.018), and the median platelets transfusion volume was lower (P=0.009). At 3, 6, 12 months after eltrombopag, the change of platelet in eltrombopag group was higher than that in the control group (P<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.艾曲泊帕对输血依赖的非重型再生障碍性贫血免疫抑制治疗疗效的影响.比较艾曲泊帕联合环孢素(CsA)和单用CsA治疗输血依赖的非重型再生障碍性贫血(TD-NSAA)的疗效.回顾性分析2017年12月至2021年6月宁德师范学院附属宁德市医院及南通大学附属医院治疗中心76例初治TD-NSAA的临床资料，其中45例使用艾曲泊帕联合CsA治疗(艾曲泊帕治疗组)，31例为同期基线匹配单用CsA治疗患者（对照组），比较两组患者疗效，并分析影响疗效的相关因素.两组在治疗3、6、12个月及随访终点时血液学反应(HR)及完全缓解(CR)均存在明显统计学差异(P<0.05)，随着艾曲泊帕治疗时间的延长疗效进一步提高，至随访终点，获得更多的CR及HR，同时随着艾曲泊帕最大稳定剂量的增加，HR有逐渐增加趋势。治疗6、12个月，艾曲泊帕治疗组骨髓巨核细胞数均明显高于对照组(P<0.05)，其脱离血小板输注中位时间较对照组短(P=0.018)，且中位血小板输注量也明显低于对照组(P=0.009)。艾曲泊帕治疗组在3、6、12个月血小板较基线升高值均高于对照组(P<0.05)。影响疗效的相关因素分析显示，患者性别、年龄、铁超负荷及治疗前血小板数量与疗效无相关性，而艾曲泊帕的中位稳定治疗剂量和中位治疗时间与疗效存在相关性。艾曲泊帕治疗组中部分患者有不同程度的不良反应，但反应轻微，多可耐受.对于残存造血细胞较多的TD-NSAA患者，加用艾曲泊帕可明显改善HR，加快血小板的恢复，安全性及耐受性良好.