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08.05.2020 | Original Research Open Access

Safety and Efficacy of the Sirolimus Gel for TSC Patients With Facial Skin Lesions in a Long-Term, Open-Label, Extension, Uncontrolled Clinical Trial

Zeitschrift:
Dermatology and Therapy
Autoren:
Mari Wataya-Kaneda, Hiroshi Nagai, Yuuki Ohno, Hiroo Yokozeki, Yasuyuki Fujita, Hironori Niizeki, Kazue Yoshida, Masaaki Ogai, Yuichi Yoshida, Akihiko Asahina, Kazuyoshi Fukai, Chiharu Tateishi, Izumi Hamada, Tatsuro Takahata, Kenji Shimizu, Shigeki Shimasaki, Hiroyuki Murota
Wichtige Hinweise

Digital Features

To view digital features for this article go to https://​doi.​org/​10.​6084/​m9.​figshare.​12162036.

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.
Literatur
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