益气养血方联合西药治疗再生障碍性贫血有效患者临床特征分析

Clinical Characteristics of Effective Treatment of Aplastic Anemia Patients with Yiqi Yangxue Prescription Combined with W estern Medicine

目的 探讨补气养血法治疗再生障碍性贫血(Aplastic anemia, AA)有效患者的临床特征。方法 采用前瞻性、随机、双盲、安慰剂对照、多中心研究设计。用分层区组随机方法将患者分为三组,补肾生血组、益气养血组、对照组,中药治疗分别予补肾填精颗粒、益气养血颗粒、安慰剂(50%补肾填精颗粒),均联合口服西药环孢素及雄激素。每组治疗以3个月为一疗程,连续观察两个疗程。观察治疗前后血常规、白细胞计数、血红蛋白含量、血小板计数、中性粒细胞计数,输血情况,中医证候积分、生活质量评分,安全性指标,并记录不良反应事件,观察期结束后进行疗效评价。选取2018年9月—2021年3月就诊于中国中医科学院西苑医院、中国中医科学院广安门医院等19个分中心的164例运用补气养血法治疗的AA患者,根据疗效标准将164例AA患者进行疗效评定,其中有效患者131例,无效患者33例;采集有效和无效患者的临床资料,并运用SPSS 20.0统计学软件进行分析,总结有效患者的临床特征。结果 独立样本t检验结果显示,治疗前血小板、中性粒细胞越高的AA患者,益气养血方联合西药治疗更易获得疗效,差异具有统计学意义(P<0.05);χ^(2)检验结果显示,无过敏史、治疗前血小板≥40×10^(9)/L的AA患者,益气养血方联合西药治疗更易获得疗效,差异具有统计学意义(P<0.05);Fisher检验结果显示,病程<1个月、治疗前白细胞≥0.5×10^(9)/L、中性粒细胞≥2.0×10^(9)/L、染色体核型正常的AA患者,益气养血方联合西药治疗更易取得疗效,差异具有统计学意义(P<0.05);多因素Logistic回归分析结果显示,病程越短,益气养血方联合西药治疗AA获得的疗效越好,差异具有统计学意义(P<0.05);ROC曲线图表明,多因素Logistic回归分析所建立的临床特征模型来预测益气养血方联合西药治疗AA有效的这一方法是可取的。结论 益气养血方联合西药为主治疗AA有效率达79.9%,无过敏史、病程<1个月且越短,血小板≥40×10^(9)/L且血小板越高,白细胞≥0.5×10^(9)/L,中性粒细胞≥2.0×10^(9)/L且越高,染色体核型正常的AA患者采用益气养血方联合西药治疗更易获得疗效。

Objective To investigate the clinical characteristics of effective treatment of aplastic anemia(AA)by tonifying qi and nourishing blood.Methods A prospective,randomized,double-blind,placebo-controlled,multicenter trial was conducted.The AA patients were randomly assigned by stratified block randomization into three groups:a kidney tonifying and blood generating group,a qi replenishing and blood nourishing group,and a control group,which were treated with Bushen Tianjing Granules,Yiqi Yangxue Granules,and placebo(50%Bushen Tianjing Granules),respectively.In addition,the patients in all the three groups orally took cyclosporine and androgen.Each course of treatment lasted for 3 months,and the patients were observed for two courses.The blood routine,white blood cell count(WBC),hemoglobin con-tent,platelet count,neutrophil count,blood transfusion,TCM syndrome score,quality of life score,and safety indicators were observed before and after treatment.Adverse events were recorded,and efficacy was evaluated at the end of the observation period.A total of 164 AA patients treated with qi tonifying and blood nourishing method in 19 medical institutions including Xiyuan Hospital and Guang′anmen Hospital of China Academy of Chinese Medical Sciences from September 2018 to March 2021 were selected.The treatment efficacy for the 164 patients was evaluated according to the efficacy criteria,which showed effective treatment in 131 patients and ineffective treatment in 33 patients.The clinical data of all the patients were collected and analyzed by SPSS 20.0,and the clinical characteristics of effective patients were summarized.Results The independent sample t test results showed that Yiqi Yangxue Prescription combined with western medicine was more likely to exert curative effect on the AA patients with higher levels of platelets and neutrophils before treatment(P<0.05).Chi-square test results showed that Yiqi Yangxue Prescription combined with western medicine was more effective for the AA patients with no history of allergy and platelets≥40×10^(9)/L before treatment(P<0.05).Fisher′s test showed that Yiqi Yangxue Prescription combined with western medicine was more effective for the AA patients with the disease course<1 month,WBC≥0.5×10^(9)/L,neutrophils≥2.0×10^(9)/L,and normal chromosome karyotype before treatment(P<0.05).Multivariate Logistic regression analysis showed that Yiqi Yangxue Prescription combined with western medicine had better efficacy for the patients with shorter course of disease(P<0.05).The receiver operator characteristic(ROC)curve showed that the clinical characteristic model established by Logistic regression was feasible to predict the efficacy of Yiqi Yangxue Prescription combined with western medicine in treating AA.Conclusion The effective rate of Yiqi Yangxue Prescription combined with western medicine in the treatment of AA reached 79.9%.This therapy is more effective for the patients with no history of allergy,course of disease<1 month and shorter course of disease,platelets≥40×10^(9)/L and higher platelet count,WBC≥0.5×10^(9)/L,neutrophils≥2.0×10^(9)/L and higher platelet count,and normal chromosome karyotype.