Efficacy and Safety of Topical Timolol for the Treatment of Facial Angiofibroma in Children with Tuberous Sclerosis Complex

OBJECTIVE: This study aimed to assess the efficacy and safety of topical timolol in treating facial angiofibromas (FAs) in pediatric patients with tuberous sclerosis complex (TSC). METHODS: A prospective clinical trial was conducted involving 15 children diagnosed with TSC and presenting with FAs. The participants were administered topical timolol gel 0.5% twice daily. Prior to the intervention, the severity of FAs in each patient was evaluated using the FA severity index (FASI), which assessed erythema, size, and extent of lesions. Clinical response was assessed at weeks 2 and 4 during the intervention period as well as 1 month after discontinuation of treatment. FINDINGS: Four weeks after discontinuing topical timolol 0.5%, statistically significant reductions were observed in the mean FASI score, erythema, size, and extent of lesions (P < 0.0001, P < 0.0001, P = 0.012, P = 0.008, respectively). FASI scores at 4 and 12 weeks postintervention, as well as 4 weeks after treatment cessation, demonstrated a significant decrease compared to baseline (P < 0.001). Erythema and extension scores also exhibited a significant decrease 1 month after treatment cessation compared to baseline (P < 0.05), while the mean size of lesions before and after the intervention did not show a statistically significant difference (P = 0.004). CONCLUSION: Topical timolol 0.5% represents a cost-effective and readily available treatment option for pediatric patients with FAs associated with tuberous sclerosis.