目的 以雙黃升白口服液Ⅱ期臨床試驗設計方案為例，探索中成藥治療肺癌化療后中性粒細胞（ANC）減少的臨床試驗方案的設計，為治療腫瘤化療后出現(xiàn)ANC減少的中藥新藥研發(fā)提供參考。方法 雙黃升白口服液Ⅱ期臨床試驗設計方案：以晚期肺癌或肺癌術后化療導致1、2級ANC減少的精氣兩虧證患者為研究對象，采用隨機、雙盲、安慰劑平行對照、多中心的臨床試驗方法，將240例患者以1∶1比例分到試驗組和對照組，試驗組予雙黃升白口服液、對照組予雙黃升白口服液模擬劑，療程為21 d。以ANC維持與復常為主要療效指標；將ANC和白細胞（WBC）計數(shù)減少的發(fā)生率、ANC曲線下面積、粒細胞減少性發(fā)熱（FN）的發(fā)生率、重組人粒細胞集落刺激因子（rhG-CSF）注射液使用量、中醫(yī)證候療效改變以及FACT-L量表評分變化作為次要療效指標；以不良事件作為安全性指標。結果 雙黃升白口服液Ⅱ期臨床試驗設計方案將疾病共性規(guī)律與患者個體特征相結合，在方法學上病證結合評定療效，客觀評價藥物的有效性與安全性，具備實用性和科學性。結論 該方案既體現(xiàn)了中醫(yī)學治療思維和療效優(yōu)勢，又適應了循證理念，成果具有外推性，為規(guī)范臨床試驗方法和推動中醫(yī)藥走向國際公認化做出了有益探索。

Objective To explore the design of the clinical trial protocol of Chinese traditional medicine for the treatment of absolute neutrophil count (ANC) reduction after chemotherapy for lung cancer by taking the design protocol of phase II clinical trial of Shuanghuang Shengbai Oral Liquid as an example, so as to provide reference for the development of new Chinese medicine for the treatment of neutropenia after chemotherapy. Methods The design scheme of phase II clinical trial of Shuanghuang Shengbai Oral Liquid: Patients with advanced lung cancer or postoperative chemotherapy for lung cancer resulting in reduction of grade 1 or 2 ANC with deficiency of both essence and qi were taken as the study subjects, and a total of 240 patients were divided into the experimental group and the control group in a ratio of 1∶1 by adopting a randomized, double-blind, placebo-parallel-controlled, multicentre clinical trial method, and the experimental group was given Shuanghuang Shengbai Oral Liquid, while the control group was given the simulant Shuanghuang Shengbai Oral Liquid, and the course of treatment was 21 days. ANC maintenance and recurrence were the main therapeutic indexes. The incidence of ANC and decreased white blood cell count, the area under ANC curve, the incidence of granulocytopenic fever, the amount of rhG-CSF injection, the change of TCM syndrome effect and the change of FACT-L scale score were taken as the secondary efficacy indicators. Adverse events were used as safety indicators. Results The design of phase II clinical trial of Shuanghuang Shengbai Oral Liquid combines the common law of disease with the individual characteristics of patients, and evaluates the therapeutic effect by combining disease and syndrome in the methodology to objectively evaluate the effectiveness and safety of the drug, which is practical and scientific. Conclusion The protocol embodies the therapeutic thinking and efficacy advantages of Chinese medicine while adapting to evidence-based concepts, and the results are extrapolated, making a useful exploration for standardizing the methodology of clinical trials and promoting Chinese medicine towards international recognition.

國家科技重大專項項目-重大新藥創(chuàng)制專項（2019ZX09201004-002-001）;國家中醫(yī)藥管理局高水平中醫(yī)藥重點學科建設項目中醫(yī)血液病學（zyyzdxk-2023268）;北京中醫(yī)藥大學東直門醫(yī)院（國家中醫(yī)藥管理局下?lián)埽R床研究和成果轉(zhuǎn)化能力提升試點項目（DZMG-ZJXY-23014）;北京中醫(yī)藥大學揭榜掛帥重點項目（2024-JYB-JBZD-007）