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- Title
Safety and Efficacy of the Sirolimus Gel for TSC Patients With Facial Skin Lesions in a Long-Term, Open-Label, Extension, Uncontrolled Clinical Trial.
- Authors
Wataya-Kaneda, Mari; Nagai, Hiroshi; Ohno, Yuuki; Yokozeki, Hiroo; Fujita, Yasuyuki; Niizeki, Hironori; Yoshida, Kazue; Ogai, Masaaki; Yoshida, Yuichi; Asahina, Akihiko; Fukai, Kazuyoshi; Tateishi, Chiharu; Hamada, Izumi; Takahata, Tatsuro; Shimizu, Kenji; Shimasaki, Shigeki; Murota, Hiroyuki
- Abstract
Introduction: Our previous clinical studies have demonstrated the short-term efficacy and safety of the sirolimus gel for patients with tuberous sclerosis complex (TSC). However, long-term clinical evidence is lacking. Our objective was to assess the safety and efficacy of long-term treatment with the sirolimus gel for the skin lesions of TSC patients. Methods: We conducted a multicenter, open-label, uncontrolled clinical trial in 94 Japanese patients with TSC. Patients applied the 0.2% sirolimus gel on their face or head twice daily for > 52 weeks (maximum 136 weeks for safety). The safety endpoints were the rate of adverse event (AE)-caused discontinuation (primary endpoint) and the incidence of AEs. The efficacy endpoint was the response rate of angiofibromas, cephalic plaques, and hypomelanotic macules. Results: Among 94 enrolled patients (mean age, 21 years; range 3–53 years), the rate of AE-caused discontinuation was 2.1% (2/94 patients). Although application site irritation and dry skin occurred relatively frequently, none of the drug-related AEs were serious; most of the drug-related AEs resolved rapidly. The major drug-related AEs (≥ 5% in incidence) were application site irritation (30.9%), dry skin (27.7%), acne (20.2%), eye irritation (8.5%), pruritus (8.5%), erythema (7.4%), dermatitis acneiform (6.4%), and dermatitis contact (5.3%). The response rates of angiofibromas, cephalic plaques, and hypomelanotic macules were 78.2% [95% confidence interval (CI) 68.0–86.3%], 66.7% (95% CI 51.1–80.0%), and 72.2% (95% CI 46.5–90.3%), respectively. Conclusions: The gel was well tolerated for a long time by patients with TSC involving facial skin lesions and continued to be effective. Trial Registration: ClinicalTrials.gov identifier: NCT02634931.
- Subjects
RAPAMYCIN; TUBEROUS sclerosis; CLINICAL trials; CONTACT dermatitis; COLLOIDS
- Publication
Dermatology & Therapy, 2020, Vol 10, Issue 4, p635
- ISSN
2193-8210
- Publication type
Article
- DOI
10.1007/s13555-020-00387-7