Abstract | OBJECTIVE: DATA SOURCES: STUDY SELECTION AND DATA EXTRACTION: Articles written in English and relevant to the topic were included. DATA SYNTHESIS: In the phase 2 trial, the mean improvement factor, a composite measure of improved tumor size and redness, was achieved in all patient groups (P < 0.001) with significant responses among the adult and pediatric subgroups at week 12. There were no serious adverse events recorded. In the phase 3 trial, 60% of participants responded to treatment in the sirolimus group compared with 0% in the placebo group with different response rates between the adult and pediatric subgroups at week 12. Sirolimus gel had no serious adverse events, and dry skin was the most common adverse reaction. Patients who had completed the 12-week trials were then enrolled in a long-term trial; angiofibromas had response rates of 78.2% to 0.2% sirolimus gel. RELEVANCE TO PATIENT CARE AND CLINICAL PRACTICE IN COMPARISON TO EXISTING DRUGS: CONCLUSIONS: Topical sirolimus 0.2% gel is a moderately effective treatment for TSC-associated facial angiofibromas with an adequate safety profile.
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Authors | Diem-Phuong D Dao, Jessica N Pixley, Zeynep M Akkurt, Steven R Feldman |
Journal | The Annals of pharmacotherapy
(Ann Pharmacother)
Pg. 10600280231182421
(Jun 29 2023)
ISSN: 1542-6270 [Electronic] United States |
PMID | 37386842
(Publication Type: Journal Article, Review)
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