目的 以雙黃升白口服液Ⅱ期臨床試驗(yàn)設(shè)計(jì)方案為例，探索中成藥治療肺癌化療后中性粒細(xì)胞（ANC）減少的臨床試驗(yàn)方案的設(shè)計(jì)，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(yàn)設(shè)計(jì)方案：以晚期肺癌或肺癌術(shù)后化療導(dǎo)致1、2級(jí)ANC減少的精氣兩虧證患者為研究對(duì)象，采用隨機(jī)、雙盲、安慰劑平行對(duì)照、多中心的臨床試驗(yàn)方法，將240例患者以1∶1比例分到試驗(yàn)組和對(duì)照組，試驗(yàn)組予雙黃升白口服液、對(duì)照組予雙黃升白口服液模擬劑，療程為21 d。以ANC維持與復(fù)常為主要療效指標(biāo)；將ANC和白細(xì)胞（WBC）計(jì)數(shù)減少的發(fā)生率、ANC曲線下面積、粒細(xì)胞減少性發(fā)熱（FN）的發(fā)生率、重組人粒細(xì)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(píng)分變化作為次要療效指標(biāo)；以不良事件作為安全性指標(biāo)。結(jié)果 雙黃升白口服液Ⅱ期臨床試驗(yàn)設(shè)計(jì)方案將疾病共性規(guī)律與患者個(gè)體特征相結(jié)合，在方法學(xué)上病證結(jié)合評(píng)定療效，客觀評(píng)價(jià)藥物的有效性與安全性，具備實(shí)用性和科學(xué)性。結(jié)論 該方案既體現(xiàn)了中醫(yī)學(xué)治療思維和療效優(yōu)勢，又適應(yīng)了循證理念，成果具有外推性，為規(guī)范臨床試驗(yàn)方法和推動(dòng)中醫(yī)藥走向國際公認(rèn)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(xiàng)項(xiàng)目-重大新藥創(chuàng)制專項(xiàng)（2019ZX09201004-002-001）;國家中醫(yī)藥管理局高水平中醫(yī)藥重點(diǎn)學(xué)科建設(shè)項(xiàng)目中醫(yī)血液病學(xué)（zyyzdxk-2023268）;北京中醫(yī)藥大學(xué)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(diǎn)項(xiàng)目（DZMG-ZJXY-23014）;北京中醫(yī)藥大學(xué)揭榜掛帥重點(diǎn)項(xiàng)目（2024-JYB-JBZD-007）