Autoinflammatory & rare systemic diseases
Efficacy and safety of mechanistic target of rapamycin (mTOR) inhibition in cutaneous sarcoidosis: a single-centre trial
Authors' conclusion
Short-term treatment with systemic sirolimus might be an effective and safe treatment option for patients with persistent glucocorticoid-refractory sarcoidosis with a long-lasting disease-modulating effect.
Study context
- Population
- Patients with persistent, active, histologically proven, GC-refractory cutaneous sarcoidosis, Vienna (Vienna) General Hospital, Austria14 randomised in topical phase
- Participants
- 16 patients
- Comparison
- Topical sirolimus 0.1% vs placebo, then single-arm oral sirolimus
- Primary endpoint
- Change in Cutaneous Sarcoidosis Activity and Morphology Index (CSAMI) after topical or systemic treatment
Methodological detailssingle-centre, n-of-1 crossover, placebo-controlled, double-blind topical treatment period, subsequent single-arm systemic treatment phase
- Single-centre
- n-of-1 crossover
- Placebo-controlled
- Double-blind topical treatment period
- Subsequent single-arm systemic treatment phase
Primary result
As reported in the abstract
Daily topical treatment did not improve cutaneous lesions
effect estimate −1.213 [95% CI −2.505 to 0.079], p=0.066 (95% CI, p)
Systemic treatment targeting trough serum concentrations of 6 ng/mL: clinical and histological improvement of skin lesions
seven (70%) of ten participants
Systemic treatment, change in Cutaneous Sarcoidosis Activity and Morphology Index (CSAMI) (median, 95% CI, p)
median −7.0
95% CI −16.5 to −3.0p=0.018
Further findings
Durability of response to systemic sirolimus
- Various morphologies responded with a long-lasting effect for more than 1 year after treatment stopped
Safety
- No serious adverse events and no deaths
- All participants included in safety analyses
Original publication
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