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FRIDAY, June 8, 2018 (HealthDay News) — Topical rapamycin seems effective for tuberous sclerosis complex (TSC)-related facial angiofibromas, according to a study published online May 23 in JAMA Dermatology<\/i>.<\/p>\n
Mary Kay Koenig, M.D., from The University of Texas Health Science Center at Houston, and colleagues examined the efficacy and safety of topical rapamycin for TSC-related facial angiofibromas in a study involving 179 patients. Participants were randomized in a 1:1:1 ratio to topical formulation containing 1 percent rapamycin, 0.1 percent rapamycin, or vehicle alone (59, 63, and 57 patients, respectively). The formulation was applied daily to designated areas at bedtime.<\/p>\n
The researchers observed clinically meaningful and statistically significant improvement in facial angiofibromas for 1 and 0.1 percent rapamycin versus vehicle only, and for 1 versus 0.1 percent rapamycin; most of the improvement was seen in the first month. The Angiofibroma Grading Scale mean improvement at six months was 16.7, 11.0, and 2.1 points for 1 and 0.1 percent rapamycin and vehicle only, respectively (P < 0.001 for 1 and 0.1 percent versus vehicle only). End-of-treatment photos were rated better than baseline for 81.8, 65.5, and 25.5 percent of patients in the 1 and 0.1 percent rapamycin groups and the vehicle group, respectively (P < 0.001 for all three pairwise comparisons).<\/p>\n
“Topical rapamycin appears effective and safe for treatment of TSC-related facial angiofibromas,” the authors write. “In this trial, the preferred dose was 1 percent once daily.”<\/p>\n
Several authors disclosed financial ties to Novartis; one author disclosed ties to MedStudy. Several authors have a provisional patent pending.<\/p>\n
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