Table 2

Treatment options for tuberous sclerosis complex–associated skin, mucocutaneous and dental manifestations13

ConditionTreatment options Additional considerations
Facial angiofibromaTopical mTOR inhibitors
  • Rapid response in weeks after initiating therapy

  • May be more effective for small early lesions and for preventing recurrence after surgery

  • Well tolerated with no systemic toxicity

  • Continual therapy necessary

Vascular laser surgery
  • Consider pulsed-dye laser for erythematous lesions

  • Effect may be enhanced when combined with 5-aminolevulinic acid

  • Temporary improvement

Ablative laser surgery
  • Consider for fibrotic lesions

  • Sedation or general anaesthesia may be necessary

Surgical excision
  • Consider for symptomatic large lesions

  • Also consider for single or fewer fibrotic lesions

  • Sedation may be necessary

Fibrous cephalic plaqueSurgical intervention
  • Consider if rapid progression and/or disfiguring

  • Sedation may be necessary

  • Surgical risks

Skin tagsSnip excision
  • Treatment usually not necessary, but consider if symptomatic

  • Minimal surgical risk

Hypomelanotic maculesTopical mTOR inhibitors
  • Treatment usually not necessary, but consider for cosmetic sensitive area on the face

  • Long-term treatment may be necessary

  • High cost

Periungual/subungual fibromaAblative laser
  • Consider if lesions symptomatic or >3 mm

  • Repeated treatment may be necessary

Surgical excision
Intraoral fibromaGood oral hygiene
  • To minimise irritation

  • None

Other options
  • If obstructive, consider electrocautery, ablative laser or surgical excision

  • Sedation may be necessary

Dental pitsGood dental hygiene
  • None

  • Consider if at risk for dental caries

  • Sedation may be necessary

Jaw cystsCurettage, surgical excision
  • Consider if at risk for bony destruction

  • Sedation may be necessary

  • Reproduced with permission from JAMA Dermatology 2014;150:1095–1101. Copyright 2014 American Medical Association. All rights reserved.

  • mTOR, mammalian target of rapamycin.