Topical Sirolimus for Facial Angiofibroma in Tuberous Sclerosis Complex: A Case Series

Autor: Gampi Ete, Manjeet Ramteke, Ratnakar R Kamath, Neha Fogla, Aarti V Chavhan
Jazyk: angličtina
Rok vydání: 2024
Předmět:
Zdroj: Journal of Clinical and Diagnostic Research, Vol 18, Iss 11, Pp 01-03 (2024)
Druh dokumentu: article
ISSN: 2249-782X
0973-709X
DOI: 10.7860/JCDR/2024/72846.20280
Popis: Facial angiofibromas (FAs) are a distressing manifestation of Tuberous Sclerosis Complex (TSC), often leading to significant physical and psychological burdens. Traditional treatment modalities have limitations, prompting the exploration of alternative therapies. In the present case series, topical sirolimus, a Mammalian Target of Rapamycin (mTOR) inhibitor, was used to manage FAs associated with TSC. Four cases (1 male and 3 female) of FAs in patients with TSC were treated with 0.1% sirolimus ointment applied once daily. The Facial Angiofibroma Severity Index (FASI) was utilised to assess FA severity at baseline and during follow-up visits, which spanned 3 to 12 months. Patient satisfaction and tolerability were also evaluated. Results demonstrated a consistent reduction in FASI scores across all patients, indicating improved FA severity. Patient satisfaction varied, with higher contentment observed in cases where treatment was initiated at a younger age. Importantly, no significant adverse effects were noted, affirming the safety profile of topical sirolimus. These findings underscore the potential of topical sirolimus as a promising therapeutic avenue for FAs in TSC, offering a cost-effective and well-tolerated alternative to conventional treatments. Further research is warranted to elucidate optimal dosing strategies and long-term outcomes, but current evidence supports its consideration as a viable option in the management of FAs associated with TSC.
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