Centrala begrepp
Sirolimus shows promise in treating cutaneous sarcoidosis.
Sammanfattning
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.
Statistik
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%).
Citat
"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