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Topical everolimus for facial angiofibromas in the tuberous sclerosis complex. A first case report
Patricia E Dill1, Gaston De Bernardis2, Peter Weber3
1Department of Pediatric Neurology and Developmental Medicine, University Children's Hospital Basel, University of Basel, Basel, Switzerland; INSERM Unité 1511, Paris, France.
Background:
Facial angiofibromas are present in most of the patients with the tuberous sclerosis complex and may cause severe disfiguration of the face. The tumor growth in tuberous sclerosis complex is promoted by the disinhibition of the mammalian target of rapamycin pathway. Thus, the systemic treatment with mammalian target of rapamycin inhibitors such as sirolimus and everolimus has recently been established to treat specific tuberous sclerosis complex-associated lesions. For patients who suffer from disfiguring facial angiofibromas only, there is a need for a topical use of mammalian target of rapamycin inhibitors. Sirolimus has been shown to be beneficial in treating facial angiofibromas. But the topical use of everolimus, which has the approval to treat tuberous sclerosis complex-associated tumors, namely giant cell astrocytomas and renal angiofibromas, has not been reported.
Patients And Results:
We present a 10-year-old girl whose facial angiofibromas were successfully treated with an everolimus ointment without relevant side effects. In addition, we provide a short pharmacological overview of sirolimus and everolimus with focus on the topical use.
Conclusions:
Topical everolimus seems to be a favorable and safe option for patients with facial angiofibromas who do not require systemic treatment.
Insights
Topical everolimus ointment effectively treated facial angiofibromas in a young patient. This mammalian target of rapamycin inhibitor offers a safe, localized treatment option for tuberous sclerosis complex patients.
Area of Science:
- Dermatology
- Oncology
- Pharmacology
Background:
- Facial angiofibromas are common in tuberous sclerosis complex (TSC), causing disfigurement.
- TSC tumor growth is linked to the mammalian target of rapamycin (mTOR) pathway.
- Systemic mTOR inhibitors (sirolimus, everolimus) treat some TSC lesions, but topical options are limited.
Observation:
- A 10-year-old girl with facial angiofibromas was treated.
- Treatment involved a topical everolimus ointment.
- The treatment was successful with no significant side effects.
Findings:
- Topical everolimus demonstrated efficacy in treating facial angiofibromas.
- The treatment was well-tolerated, with no relevant adverse events reported.
- This case highlights the potential of topical everolimus for localized TSC lesions.
Implications:
- Topical everolimus presents a promising, safe alternative for TSC patients with facial angiofibromas needing localized treatment.
- This approach avoids systemic side effects associated with oral mTOR inhibitors.
- Further research into topical mTOR inhibitors for dermatological manifestations of TSC is warranted.
