DataSheet1_Efficacy and Safety of Shenqu Xiaoshi Oral Liquid Compared With Domperidone Syrup in Children With Functional Dyspepsia.docx

Background: Treatment of functional dyspepsia (FD) in children is generally symptomatic and unsatisfactory. Traditional Chinese medicines, such as Shenqu Xiaoshi Oral Liquid (SXOL), have been recommended to alleviate dyspeptic symptoms. However, evidence of their safety and efficacy remains limited to date. AIM: To assess whether 2 weeks of therapy with SXOL was non-inferior to domperidone syrup in children with FD.
Methods: In this randomized, double-blind, double-simulated, non-inferiority, multi-center clinical trial, we recruited children (3–14 years) with FD according to the Rome IV criteria from 17 tertiary medical centers across China. Patients were randomly allocated (1:1) to receive SXOL or domperidone syrup for 2 weeks. We compared the participants’ clinical scores from both groups based on the severity and frequency of dyspepsia symptoms according to Rome IV criteria (0, 1, 2, and 4 weeks after randomization). The primary endpoint was the total response rate, which was defined as the proportion of patients with a decrease of 30% or more in the FD symptoms clinical score from baseline, at the end of the 2-weeks treatment. A non-inferiority margin of -10% was set. Secondary endpoints and adverse events were assessed. This trial is registered with www.Chictr.org.cn, number ChiCTR1900022654.
Results: Between February 2019 and March 2021, a total of 373 patients were assessed for eligibility, and 356 patients were enrolled and randomized. The clinical response rate at week two was similar for SXOL [118 (83.10%) of 142] and domperidone [128 (81.01%) of 158]; difference 2.09; 95% CI −6.74 to 10.71, thereby establishing non-inferiority. The total FD symptom scores were significantly improved in the two groups at 1-, 2-, and 4-weeks follow-up periods (p < 0.005). The decrease in symptom score compared with the baseline were similar between these two groups. Over the total study period, 10 patients experienced at least one treatment-related adverse event [six (3.37%)] in the SXOL group, four [(2.25%) in the domperidone group], although no serious adverse event was noted.
Conclusion: Treatment with SXOL effectively improves dyspeptic symptoms and is well tolerated. In addition, it is not inferior to domperidone syrup and leads to sustained improvement in Chinese children with FD.

背景：儿童功能性消化不良（functional dyspepsia, FD）的治疗多以对症支持为主，临床疗效不尽如人意。中医药如神曲消食口服液（Shenqu Xiaoshi Oral Liquid, SXOL）虽被推荐用于缓解消化不良症状，但目前其安全性与有效性的相关证据仍较为有限。
研究目的：评估为期2周的SXOL治疗方案用于儿童FD患者是否不劣于多潘立酮混悬液。
方法：本项随机、双盲、双模拟、非劣效性多中心临床试验，从中国17家三级医疗中心招募符合罗马IV标准（Rome IV criteria）的3~14岁FD患儿。按1:1比例将患者随机分配至SXOL组或多潘立酮混悬液组，接受为期2周的治疗。分别于随机化后第0、1、2、4周，依据罗马IV标准评估两组患儿消化不良症状的严重程度与发作频率，以此比较两组的临床评分。本研究的主要终点为治疗2周结束时的总应答率，即较基线FD症状临床评分降低30%及以上的患者占比，研究设定非劣效界值为-10%。同时评估次要终点与不良事件。本试验已在中国临床试验注册中心（www.Chictr.org.cn）注册，注册号为ChiCTR1900022654。
结果：2019年2月至2021年3月期间，共计373例患者接受入组资格筛查，最终356例患者入组并完成随机分组。治疗2周时，SXOL组（142例中118例，83.10%）与多潘立酮组（158例中128例，81.01%）的临床应答率相近，组间差异为2.09%，95%置信区间为-6.74%~10.71%，由此证实SXOL的非劣效性。两组患儿在随访1周、2周及4周时的FD总症状评分均较基线显著改善（P<0.005），且两组症状评分较基线的降幅无统计学差异。整个研究周期内，SXOL组共有6例（3.37%）患者出现至少1次治疗相关不良事件，多潘立酮组为4例（2.25%），未观察到严重不良事件。
结论：SXOL治疗可有效改善儿童FD患者的消化不良症状，且耐受性良好；其疗效不劣于多潘立酮混悬液，能够使中国FD患儿的症状获得持续改善。