Sirolimus efficacy and safety
1 evidence topic · 2 sources
Summary
SELVA enrolled 51 participants, of whom 50 initiated QTORIN rapamycin treatment. The primary efficacy population comprised 49 participants aged at least 6 years. At week 24, the mean change on the mLM-IGA was +2.13 points, with p<0.001. Among 43 participants aged at least 6 years who completed the efficacy evaluation period, 41 experienced at least a 1-point improvement.
Treatment-related adverse events occurred in 17 of 50 participants (34%). Four participants (8%) experienced serious adverse events assessed as unrelated to study treatment. SELVA was a single-arm, baseline-controlled study without a concurrent placebo or active-treatment comparator.