論文

査読有り 国際誌
2020年5月8日

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

Dermatology and therapy
  • 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

記述言語
英語
掲載種別
研究論文(学術雑誌)
DOI
10.1007/s13555-020-00387-7

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.

リンク情報
DOI
https://doi.org/10.1007/s13555-020-00387-7
PubMed
https://www.ncbi.nlm.nih.gov/pubmed/32385845

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