To observe the effectiveness and hepatorenal safety of Fule Cream alone or in combination with topical corticosteroids (TCS)/calcineurin inhibitors (TCI) in treating pediatric skin diseases.

Children prescribed Fule cream in our hospital were enrolled and categorized into monotherapy cohort (Fule Cream alone) and combination cohort (Fule cream with TCS/TCI) based on medication use. The changes in liver and kidney function indicators, the incidence of drug-induced liver injury (DILI), acute kidney injury (AKI) and clinical effectiveness were compared between the two cohorts. Outcome variables for effectiveness were constructed by extracting efficacy descriptions from medical record texts.

A total of 2 439 children were included for safety analysis and 741 for effectiveness analysis. Effectiveness analysis showed that the combination cohort had a significantly higher response rate of 77.93% (505 cases/648 cases) than the monotherapy cohort 56.99% (53 cases/93 cases) with an odds ratio (OR) of 0.38 (95% CI: 0.24-0.59). Regarding safety, after treatment, The levels of alanine aminotransferase (ALT), aspartate aminotransferase (AST) and alkaline phosphatase (ALP) in cohort 1 and cohort 2 were significantly decreased after treatment (all P<0.05). The incidence of DILI and AKI was low. Data quality analysis revealed a high missing rate for follow-up laboratory tests and a lack of standardized efficacy evaluations.

The Fule Cream combined with TCS/TCI regimen demonstrated not only superior effectiveness but also no increased clinically significant hepatorenal risk in a real-world setting. Although data missingness may affect the precision of effect estimates, sensitivity analyses support the robustness of the primary conclusions. These findings provide real-world evidence supporting the clinical use of Fule Cream and highlight the necessity of enhancing data governance to generate higher-quality real-world evidence.