Objective: To analyze the influence factors on the efficacy of immunosuppression therapy (IST) combined with eltrombopag and IST alone in the treatment of childhood severe aplastic anemia (SAA). Methods: A retrospective cohort study. A total of 124 children with SAA who were initially treated with IST at Beijing Children's Hospital from March 2017 to May 2020 were enrolled. Clinical characteristics, laboratory examination and prognosis data were collected at the time of enrollment. According to the treatment plan, the children were divided into the eltrombopag combined with IST group (eltrombopag group) and the IST group. Binary Logistic regression model was used to analyze the factors affecting the efficacy of the two groups at 6 months of treatment, and the factors affecting the efficacy of the eltrombopag group at the end of follow-up. Results: There were 75 cases (45 males and 30 females) in the eltrombopag group. The age of diagnosis was 5.9 (3.5, 8.5) years. There were 49 patients in the IST group, including 23 males and 26 females, whose age at diagnosis was 6.2 (4.4, 8.8) years. The absolute lymphocyte count before treatment in the eltrombopag group was significantly lower than that in the IST group (1.1 (0.4, 1.6)×109 vs. 2.1 (1.4, 2.8)×109/L). Absolute reticulocyte count in the eltrombopag group was significantly higher than that of IST group (26.9 (8.7, 54.2)×109 vs. 9.5 (4.0, 19.0)×109/L) (both P<0.05). Influencing factors of 6-month response: a comparison between response and un-response groups in the eltrombopag treated patients showed that, before treatment, hemoglobin (69 (61, 78) vs. 64 (59, 68) g/L), platelet (10 (6, 16)×109 vs. 6 (3, 8)×109/L), absolute reticulocyte count (ARC) (34.0 (15.8, 57.3)×109 vs. 6.5 (4.6, 16.8)×109/L) and the response rate to granulocyte colony stimulating factor (G-CSF) after treatment (82.4% (47/57) vs. 9/18) were significantly different (all P<0.05). Logistic regression model analysis showed that ARC (OR=1.09, 95%CI 1.02-1.18) and absolute neutrophil count were independent influencing factors of 6-month response rate in the eltrombopag group (OR=0.00, 95%CI 0.00-0.89). ARC was also the independent influencing factors of the end of follow-up response rate in the eltrombopag group (OR=1.04, 95%CI 1.01-1.07). Conclusions: Pre-treatment blood count and response to G-CSF were predictors of overall response to eltrombopag combined with IST. The higher the ARC before treatment, the higher the total response rate and complete response.
目的： 分析儿童初治重型再生障碍性贫血（SAA）应用艾曲泊帕联合免疫抑制（IST）和单用IST的疗效及其影响因素。 方法： 回顾性队列研究。选择2017年3月至2020年5月在北京儿童医院初治并应用IST治疗的124例SAA患儿为研究对象，收集入组时临床特征、实验室检查和预后等数据。根据治疗方案是否在IST基础上联用艾曲波帕分为艾曲泊帕联合IST组（简称艾曲泊帕组）和单用IST组，应用二分类Logistic回归模型分析两组患儿治疗6个月时的疗效影响因素，并分析艾曲泊帕组随访末期时的疗效影响因素。 结果： 艾曲泊帕组75例，男45例、女30例，诊断时年龄为5.9（3.5，8.5）岁；单用IST组49例，男23例、女26例，诊断时年龄为6.2（4.4，8.8）岁。艾曲泊帕组治疗前淋巴细胞绝对计数明显低于单用IST组［1.1（0.4，1.6）×109比2.1（1.4，2.8）×109/L］，网织红细胞绝对值计数（ARC）明显高于单用IST组［26.9（8.7，54.2）×109比9.5（4.0，19.0）×109/L］（均P<0.05）。6个月时艾曲泊帕组中有反应组与无反应组相比，治疗前血红蛋白［69（61，78）比64（59，68）g/L］、血小板计数［10（6，16）×109比6（3，8）×109/L］、ARC［34.0（15.8，57.3）×109比6.5（4.6，16.8）×109/L］及治疗后对粒细胞集落刺激因子（G-CSF）的反应率［82.4%（47/57）比9/18］差异均有统计学意义（均P<0.05）。二分类Logistic回归模型分析结果显示ARC（OR=1.09，95%CI 1.02~1.18）和中性粒细胞计数（OR=0.00，95%CI 0.00~0.89）是艾曲泊帕组治疗6个月总反应的独立影响因素，ARC也是艾曲泊帕组随访末期完全反应的独立影响因素（OR=1.04，95%CI 1.01~1.07）。 结论： 治疗前血细胞计数及治疗后对G-CSF的反应为艾曲泊帕联合IST治疗总反应的影响因素。治疗前ARC越高，应用艾曲泊帕联合IST获得反应及完全反应的可能性更高。.