Effectiveness and Safety of Oral Compound Glycyrrhizin Followed by Phototherapy for the Treatment of Progressive Vitiligo in Children

ABSTRACT Childhood vitiligo, distinct from its adult counterpart, presents unique treatment challenges. Glycyrrhizin inhibits the release of high‐mobility group box 1 (HMGB1) protein from keratinocytes, preventing melanocyte apoptosis and autophagy. Furthermore, the orally administered compound glycyrrhizin (OCG) effectively treats various autoimmune disorders, demonstrating long‐term efficacy, safety, and tolerability. This study compared the efficacy of OCG and oral prednisone (OP), followed by phototherapy, in patients with progressive childhood vitiligo at 52 weeks' follow‐up. Fifty children with vitiligo were randomized into two groups according to treatment: OCG (50–150 mg/day) followed by phototherapy ( n = 25) and OP (5–10 mg/day) followed by phototherapy ( n = 25). At Week 24, a halt in disease progression (HDP) was observed in 20 (80%) patients in the OCG group and 21 (84%) in the OP group, with no significant difference ( p > 0.99) . However, the mean time to achieve HDP was significantly shorter in the OP group than in the OCG group (14.73 ± 4.84 vs. 19.13 ± 4.82 weeks; p < 0.01). In addition, serum HMGB1 concentrations were significantly reduced after treatment with OCG at Week 24 (3.02 ± 0.83 vs. 0.95 ± 0.36 ng/mL [ p < 0.01]; OP, 2.79 ± 0.16 vs. 1.03 ± 0.34 ng/mL [ p < 0.01]). The decline in Vitiligo Area Scoring Index (VASI) score at the end of follow‐up (i.e., Week 52) did not show a statistically significant difference between the OCG and OP groups (52.31% ± 14.86% vs. 55.71% ± 21.23%; p = 0.55). The therapeutic response of the clinical markers of progression was good and comparable between the OCG and OP groups. OCG demonstrated similar efficacy to OP followed by phototherapy in controlling disease activity and promoting repigmentation in children with vitiligo at 52 weeks of follow‐up. Trial Registration: ChiCTR2400086844