Effectiveness and safety of topical sirolimus in children with angiofibromas and TSC
About this study
This study explored whether a 1% sirolimus cream could safely and effectively treat facial angiofibromas in children with TSC. Angiofibromas, while not dangerous, can be emotionally and socially challenging, especially during adolescence. The goal was to test a non-invasive option that could reduce these lesions without the discomfort and risks of more aggressive treatments like lasers or surgery.
How the study was conducted
Ten children aged 8 to 17 took part. They were given a prepared 1% sirolimus cream. The cream was applied once daily—first to one side of the face for six months, then to the other side for another six months. Each child served as their own comparison, allowing researchers to measure both effectiveness and recurrence. Monthly check-ins were used to monitor progress and any side effects.
Outcomes of the study
All children showed improvement by the third month of treatment. There were fewer angiofibromas, they were smaller, and redness had reduced. Children with smaller and fewer lesions saw the most benefit, experiencing up to 80% improvement. Larger lesions responded more moderately (40–80% improvement). After stopping treatment, some lesions began returning around the fourth month, though not as severely as before. Only two children had mild skin irritation during sun exposure, which was resolved with sunscreen and didn’t require stopping the treatment.
Conclusions of the study
Topical 1% sirolimus appears to be a safe and effective treatment for facial angiofibromas in children with TSC. It’s especially effective when started early and on smaller lesions. The treatment is well tolerated, with very few side effects, and avoids the pain and risks of surgical or laser procedures. While some recurrence was observed, regular treatment cycles could help manage that.
Implications of the study
This study suggests that early treatment of facial angiofibromas seems to give the best results, so monitoring and treating angiofibromas sooner rather than later could make a real difference. More research is still needed to fine-tune how often and how long the cream should be used, but the findings are promising and echo those of other studies regarding topical sirolimus.
DISCLAIMER
This information is intended to provide some insights into recent TSC-related research. It is not intended to, and it should not, constitute medical or other advice. Readers are warned not to take any action without first seeking medical advice.
Lara-Arrieta AF, Puente SG, Flores-Pérez J, Flores-Pérez C, Lomelí-Valdez R, Venegas-Andrade A, Palacios-López C. Effectiveness and safety of topical sirolimus in children with angiofibromas and tuberous sclerosis complex. Bol Med Hosp Infant Mex. 2025;82(2):115-120. English. doi: 10.24875/BMHIM.24000091. PMID: 40393010.
Full paper available at: 10.24875/BMHIM.24000091