Treatment of facial angiofibromas with topical application of oral rapamycin solution (1 mg mL −1 ) in two patients with tuberous sclerosis

Funding sources: none. Conflicts of interest: none declared. Madam, Tuberous sclerosis (TS) is an autosomal dominant, multiorgan system disease caused by mutations in the tumour suppressor genes TSC1 and TSC2.1 While benign, the cutaneous manifestations of TS can be disfiguring. This is particularly true of angiofibromas, the most common TS lesions to occur on the face.2 Treatment of angiofibromas has historically been limited to surgically destructive techniques. We report the successful treatment of facial angiofibromas in two patients with TS with topical application of noncompounded, commercially available oral rapamycin solution (1 mg mL−1). Patient 1, a 15‐year‐old girl with TS who had previously tried erbium‐YAG laser ablation, presented for worsening facial angiofibromas (Fig. 1a). She was instructed to apply oral rapamycin solution (1 mg mL−1) to affected areas twice daily. However, she was unable to apply the solution more than once daily due to irritation. After 10 weeks, substantial improvement in the number and size of her angiofibromas was noted (Fig. 1b). More pronounced improvement may have been seen with twice daily application. At 10 weeks, her serum rapamycin level was undetectable.