Cyproheptadine enhances weight gain and modulates appetite-regulating peptides in children with failure to thrive and food allergies.

Cyproheptadine hydrochloride (CH) possesses both antihistaminic and appetite-stimulating properties, suggesting therapeutic potential for children with failure to thrive (FTT) and food allergies. This study evaluated the effects of CH on growth parameters, clinical manifestations, and serum levels of appetite-regulating peptides Nesfatin-1 and NUCB2 in children aged 1-3 years with FTT and food allergy. In this non-randomized controlled real-world study, participants were assigned to either the treatment group (T-group, n = 25), receiving CH at 0.15 mg/kg twice daily for 12 weeks, or the control group (NT-group, n = 19). Primary outcomes were anthropometric indices: body weight, weight-for-age Z-score (WAZ), height, weight-for-length Z-score (WLZ), body mass index (BMI), and length-for-age Z-score (LAZ). Secondary outcomes included clinical symptom scores and changes in Nesfatin-1 and NUCB2 levels. After 12 weeks, the T-group showed significantly greater improvements in body weight (10.32 ± 0.28 kg vs. 9.05 ± 0.23 kg, P = 0.002), WAZ (P < 0.001), WLZ (P < 0.001), and BMI (P = 0.001) compared to the NT-group. No significant differences were observed in height or LAZ. Within the T-group, significant improvements from baseline to week 12 were observed for all weight-based indices (P < 0.001). CH treatment was also associated with significant reductions in serum Nesfatin-1 (P = 0.000) and NUCB2 (P = 0.043) levels. At baseline, reduced food intake and sleep disturbances were negatively correlated with WAZ and LAZ, respectively. CH treatment significantly enhances weight gain and improves key anthropometric indices in young children with FTT and food allergy, potentially mediated by the downregulation of anorexigenic peptides Nesfatin-1 and NUCB2. These findings suggest that CH may be a promising pharmacotherapeutic option for this complex patient population.