Khái niệm cốt lõi
Sirolimus shows promise in treating cutaneous sarcoidosis.
Tóm tắt
The study explores the effectiveness of sirolimus in treating cutaneous sarcoidosis, highlighting improvements in skin lesions. Key points include:
- Sirolimus treatment led to improvements in skin lesions in 7 out of 10 patients.
- Mechanistic target of rapamycin (mTOR) inhibition is a potential therapeutic avenue for sarcoidosis.
- Previous studies have shown high mTOR activity in sarcoidosis granulomas.
- The study involved a small clinical trial with 16 participants.
- Sirolimus treatment resulted in clinical improvement in skin lesions, with some patients experiencing complete resolution.
- No serious adverse events were reported, but mild skin reactions were noted in some patients.
- The study suggests the need for larger clinical trials to further explore mTOR inhibition in sarcoidosis.
- Molecular analysis combined with clinical response supports the relevance of the mTOR pathway in sarcoid granuloma formation.
Thống kê
7 of 10 patients treated with sirolimus had improvements in skin lesions after 4 months.
70% of patients in the systemic group had clinical improvement in skin lesions.
The median change in Cutaneous Sarcoidosis Activity and Morphology Index was -7.0 points.
42% of patients treated with systemic sirolimus reported mild skin reactions.
Adverse events included hypertriglyceridemia (17%), hyperglycemia (17%), and proteinuria (8%).
Trích dẫn
"Although all reports observed improvement of the disease following the treatment, no clinical trial investigating the efficacy and safety of sirolimus in patients with sarcoidosis had been published" - Georg Stary, MD