Fu L L, Yang B X, Li H M, Wang R X, Chen H, Ma J
Department of Hematology, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, National Key Clinical Discipline of Pediatric Hematology, National Key Discipline of Pediatrics (Capital Medical University),Key Laboratory of Major Diseases in Children, Ministry of Education, Beijing100045, China.
Zhonghua Er Ke Za Zhi. 2024 Dec 2;62(12):1196-1201. doi: 10.3760/cma.j.cn112140-20240731-00536.
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). 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. 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)×10 2.1 (1.4, 2.8)×10/L). Absolute reticulocyte count in the eltrombopag group was significantly higher than that of IST group (26.9 (8.7, 54.2)×10 9.5 (4.0, 19.0)×10/L) (both <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) 64 (59, 68) g/L), platelet (10 (6, 16)×10 6 (3, 8)×10/L), absolute reticulocyte count (ARC) (34.0 (15.8, 57.3)×10 6.5 (4.6, 16.8)×10/L) and the response rate to granulocyte colony stimulating factor (G-CSF) after treatment (82.4% (47/57) 9/18) were significantly different (all <0.05). Logistic regression model analysis showed that ARC (=1.09, 95% 1.02-1.18) and absolute neutrophil count were independent influencing factors of 6-month response rate in the eltrombopag group (=0.00, 95% 0.00-0.89). ARC was also the independent influencing factors of the end of follow-up response rate in the eltrombopag group (=1.04, 95% 1.01-1.07). 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.
分析免疫抑制治疗（IST）联合艾曲泊帕与单纯IST治疗儿童重型再生障碍性贫血（SAA）疗效的影响因素。一项回顾性队列研究。纳入2017年3月至2020年5月在北京儿童医院初治的124例SAA患儿。收集入组时的临床特征、实验室检查及预后数据。根据治疗方案，将患儿分为艾曲泊帕联合IST组（艾曲泊帕组）和IST组。采用二元Logistic回归模型分析治疗6个月时两组疗效的影响因素，以及随访结束时艾曲泊帕组疗效的影响因素。艾曲泊帕组75例（男45例，女30例），诊断年龄为5.9（3.5，8.5）岁。IST组49例，男23例，女26例，诊断年龄为6.2（4.4，8.8）岁。艾曲泊帕组治疗前绝对淋巴细胞计数显著低于IST组（1.1（0.4，1.6）×10².1（1.4，2.8）×10/L）。艾曲泊帕组绝对网织红细胞计数显著高于IST组（26.9（8.7，54.2）×10⁹.5（4.0，19.0）×10/L）（均<0.05）。6个月反应的影响因素：对接受艾曲泊帕治疗的患者中反应组与无反应组进行比较，结果显示，治疗前血红蛋白（69（61，78）⁶⁴（59，68）g/L）、血小板（10（6，16）×10⁶（3，8）×10/L）、绝对网织红细胞计数（ARC）（34.0（15.8，57.3）×10⁶.5（4.6，16.8）×10/L）及治疗后粒细胞集落刺激因子（G-CSF）反应率（82.4%（47/57）⁹/18）差异有统计学意义（均<0.05）。Logistic回归模型分析显示，ARC（=1.09，95% 1.02 - 1.18）和绝对中性粒细胞计数是艾曲泊帕组6个月反应率的独立影响因素（=0.00，95% 0.00 - 0.89）。ARC也是艾曲泊帕组随访结束时反应率的独立影响因素（=1.04，95% 1.01 - 1.07）。治疗前血细胞计数及对G-CSF的反应是艾曲泊帕联合IST总体反应的预测指标。治疗前ARC越高，总反应率及完全缓解率越高。
