Topical sirolimus gel clinically effective for systemic rare vascular malformations

The study's primary endpoint was calculated as the safety evaluation of sirolimus gel. The main secondary endpoint was the improvement rate evaluated by the Central Judgment Committee at 12 weeks using photographs.

The researchers reported the following findings:

• No adverse events were observed.
• Blood sirolimus was not detected in any patient.
• 50% of the patients had mild improvement, and the remaining 50% of patients showed no change after 12 weeks of treatment.
• Blue rubber bleb nevus syndrome, a generalized venous malformation, showed the greatest response.
• Treatment response varied with different lesions in the same patients.

"We found 0.2% sirolimus gel to be as clinically effective as systemic sirolimus treatment in patients with venous and capillary malformations and more effective for early active lesions, even systemic venous malformations," the researchers concluded.

Reference:

Wataya-Kaneda, M., Maeda, S., Nakamura, A., Hayashi, M., & Fujimoto, M. Verification of the efficacy of topical sirolimus gel for systemic rare vascular malformations: A pilot study. The Journal of Dermatology. https://doi.org/10.1111/1346-8138.16930