目的 初步評價中成藥治療兒童咳嗽變異性哮喘的有效性與安全性,為治療咳嗽變異性哮喘的中藥研發(fā)與疾病的臨床研究提供參考.方法 采用區(qū)組隨機(jī)、雙盲雙模擬、劑量探索、陽性藥平行對照、多中心臨床試驗的方法,將144例患兒以1:1:1:1的比例分到高、低劑量,安慰劑以及陽性藥組,口服小兒咳喘顆粒試驗藥及模擬劑,孟魯司特鈉片及其模擬劑.療程均為4周.以咳嗽嚴(yán)重程度的日平均分為主要療效指標(biāo);把疾病控制情況評估,活動受限、夜間癥狀、緩解藥/急診需求情況,呼氣峰值流速(PEF)日變異率,以及中醫(yī)證候療效作為次要療效指標(biāo);以不良事件作為主要安全性指標(biāo).結(jié)果 建立咳嗽變異性哮喘的評價體系,制定納入、排除、脫落標(biāo)準(zhǔn)以及主、次要評價指標(biāo),并規(guī)范了入組時不同慢性咳嗽的鑒別診斷要求.結(jié)論 通過小兒咳喘顆粒Ⅱ期臨床試驗方案設(shè)計,對中成藥治療兒童咳嗽變異性哮喘的有效性與安全性進(jìn)行了有益的探索,在目前條件下該方案具有可操作性.

Objective To evaluate the effectiveness and safety of Chinese patent medicine (CPM) in the treatment of children's cough variant asthma (CVA), and to provide an example for the clinical study design of CVA. Methods Using block randomized, double-blind, double-dummy, dose exploration, parallel, active-controlled, and multi-center clinical research method, 144 cases of children with CVA were divided into high dose group, low dose group, placebo group, and positive drug group (1:1:1:1). Children are treated with Xiao'er Kechuan Granule, Montelukast, and their placebos for 4 weeks. The average score of cough severity (day and night) was used as the main evaluation index. Assessment of disease control, limited activity, nocturnal symptom, situation of rescue medication/emergency requirement, PEF mutation rate per day, and efficacy of TCM syndrome were detected as secondary indexes. Incidence of adverse events/reaction was used as the main security evaluation index. Results The phase II clinical trials of Xiao'er Kechuan Granule are designed, the effectivity and safety of CPM in the treatment of CVA are explored, and the method has maneuverability under the existing conditions.