真实世界研究和RCT随机对照研究方法评价中西医结合干预慢性乙型肝炎疗效

Comparison of the Advantages of Different Clinical Research Methods of Real-world Study and Randomized Controlled Trial in Evaluating the Efficacy of Traditional Chinese and Western Medicine in the Intervention of Chronic Hepatitis B

[目的]比较基于真实世界(real world,RW)研究的中西医结合干预慢性乙型肝炎(chronic hepatitis B,CHB)临床疗效与同期随机对照研究(random control trial,RCT)结果的异同,为中西医结合治疗方案的疗效评价提供适宜和科学的研究方法。[方法]应用RW研究方法,通过病例相关数据收集,回顾性分析我院肝病科2016年1月至2019年12月行中西医结合干预的CHB病例,系统评估其临床疗效、疾病控制率、不良反应等,并与我科同期RCT结果进行比较和分析。[结果]本次RW研究采取CHB治疗方案较RCT更加个体化和多样化(P<0.05),RW研究得出的临床有效率(82.41%)略高于RCT有效率(80.00%),但其差异无统计学意义(P>0.05);两组干预后总体丙氨酸氨基转移酶(alanine aminotransfease,ALT)复常率、乙型肝炎e抗原(hepatitis Be antigen,HBeAg)转阴率、HBeAg转换率及HBV DNA不可检测率等临床指标改善差异无统计学意义(P>0.05);其中RCT组临床有效率及HBV DNA不可检测比例显著高于RW研究中恩替卡韦+肝苏丸治疗方案(P<0.05),虽RW其余治疗方案较RCT组临床有效率及多数临床指标有所改善,但差异无统计学意义(P>0.05);两组研究方法出现不良反应情况主要以血清肌酐升高、血清肌酸激酶升高等为主,RW研究不良反应率略高,但差异无统计学意义(P>0.05);RW研究中以头痛为主的不良反应主要发生在恩替卡韦+活血化瘀方组,以血清肌酐和肌酸激酶升高为主的不良反应主要发生在恩替卡韦+清利湿热方、疏肝健脾方组。[结论]较RCT方法,RW临床研究所反映的临床疗效及指标更具客观性和临床参考价值,基于中医辨证的个体化和多样化的中西医结合治疗方案能够有效提升患者临床疗效,但在组方用药时应注意药物特点,避免出现不良反应。

[Objective]To compare the similarities and differences between the clinical effects of integrated traditional Chinese and western medicine interventions on chronic hepatitis B(CHB) based on real world(RW) studies and concurrent randomized controlled trials(RCT),and to provide appropriate and scientific research for the evaluation of the efficacy of integrated traditional Chinese and western medicine treatment programs. [Methods] Applying RW research methods,by collecting case-related data,it retrospectively analyzed cases of CHB with integrated traditional Chinese and western medicine during the period between Jan. 2016 and Dec. 2019 in the Department of Liver Diseases of the Sixth People ’s Hospital of Shenyang,and systematically evaluated their clinical efficacy,disease control rate,adverse reactions,etc.,which were compared and analyzed with the results of concurrent RCT.[Results]In RW study,CHB treatment was more individualized and diversified than RCT(P <0.05). The effective rate of clinical efficacy(82.41%) was slightly higher than that of RCT(80.00%),but the difference was not statistically significant(P >0.05). There was no statistically significant difference between the two groups in improving clinical indicators such as the recovery rate of alanine aminotransfease(ALT),conversion rate of hepatitis Be antigen(HBeAg),conversion rate of HBeAg,and undetectable rate of HBV DNA(P>0.05). Among them,the clinical effective rate and undetectable rate of HBV DNA in RCT group were significantly higher than those in RW group(P<0.05). Although the clinical effective rate and most clinical indicators of the other RW treatment regiments were improved compared with RCT group,the difference was not statistically significant(P>0.05). The adverse reactions in the two groups were mainly increased serum creatinine and creatine kinase(CK). The adverse reactions in the RW study were slightly higher,but the difference was not statistically significant(P>0.05). In RW study,the adverse reactions of headache mainly occurred in the Entecavir + blood circulationpromoting and blood stasis-removing prescription group,while the adverse reactions of serum creatinine and CK elevation occurred in the Entecavir +dampness and heat-clearing away prescription group and the liver-soothing and spleen-strengthening prescription group. [Conclusion]Compared with RCT,the clinical efficacy and indicators of RW research are more objective and have more clinical reference value. The individualized and diversified integrated Chinese and western medicine treatment plan based on traditional Chinese medicine syndrome differentiation can effectively improve the patient’s clinical efficacy,but in the preparation of medication should pay attention to the characteristics of the drug to avoid adverse reactions.