Wangshi Baochi Pill for functional dyspepsia syndrome in infants and toddlers: a multicenter randomized...

Gastrointestinal discomfort in infants and toddlers is common, yet many cases may not meet the Rome IV diagnostic criteria, posing a challenge to clinical management. To address this gap, Chinese experts have proposed the novel diagnostic entity "functional dyspepsia syndrome in infants and toddlers" (FDSIT). Therapeutic options for functional gastrointestinal disorders in infants and toddlers remain considerably limited, particularly with regard to conventional Western pharmacotherapies. Wangshi Baochi Pill (WBP), a traditional Chinese medicine with a long history of pediatric use, has shown efficacy in older children (≥4 years) with functional dyspepsia. However, its efficacy and safety for FDSIT remain unclear. This study aimed to assess the efficacy and safety of WBP in infants and toddlers diagnosed with FDSIT according to the Chinese expert consensus.

This multicenter, randomized, double-blind, placebo-controlled trial was conducted across 15 hospitals in China. Eligible participants aged 1-36 months were randomly assigned (1:1, block size 4) to receive WBP (2 mg/kg twice daily) or a matching placebo for 7 days, with follow-up through day 14. The primary endpoint was the combined symptom effectiveness rate (CSER) at day 7, defined as the proportion of participants achieving a ≥50% reduction in total symptom score across five prespecified symptom domains, each graded on a 4‑point scale. Secondary endpoints included CSER at days 3 and 14, individual symptom remission rates, growth parameters, and adverse events (AEs), which were systematically monitored via diary cards and clinical assessments.

There were 240 infants and toddlers randomized; 229 were included in the full analytical set (WBP, n=113; placebo, n=116). Baseline characteristics were well balanced. At day 7, CSER was significantly higher in the WBP group than in the placebo group [69.03% vs. 33.62%; absolute difference 35.41%, 95% confidence interval (CI): 23.30% to 47.51%; χ²=28.72, P<0.001]. Significant improvements with WBP were also observed at day 14 (82.30% vs. 43.97%; absolute difference 38.33%, 95% CI: 26.89% to 49.79%; χ²=36.04, P<0.001). Symptom remission rates for decreased appetite, belching/abdominal distension/increased flatus, and abnormal defecation were significantly higher in the WBP group at both time points. AEs occurred in 19 (16.81%) WBP-treated participants and 17 (14.53%) placebo recipients (P=0.63). One mild episode of diarrhea (0.88%) in the WBP group was deemed a treatment-related adverse drug reaction. No serious AEs were reported.

In this trial, WBP demonstrated notable efficacy and a favorable short-term safety profile for the treatment of FDSIT. These findings suggest that WBP may represent a potential therapeutic option, although further confirmatory studies are warranted.

Chinese Clinical Trial Registry (ChiCTR), ChiCTR2300072543. Registered on 16 June 2023.