Safety and efficacy of topical simvastatin plus cholesterol cream versus topical simvastatin cream alone for porokeratosis ptychotropica: A randomized, single-blind, split-body, placebo-controlled, investigator-initiated trial.

Abstract

Porokeratosis ptychotropica (PP) is a severe inherited skin disorder resistant to traditional treatments. Topical statins are being explored as a potential alternative. To evaluate the efficacy and safety of topical simvastatin and simvastatin/cholesterol in treating PP. In this single-blind, split-body, placebo-controlled trial, PP patients were 1:1 randomized to apply either 2% simvastatin or 2% simvastatin/cholesterol cream on 1 randomized side of the buttocks twice daily for 8 weeks. If blinded evaluation determined that the treatment side showed superior efficacy over placebo at week 8, both sides would continue treatment for 40 more weeks. Of 24 participants screened, 18 were enrolled and underwent randomization. One patient withdrew from each group before preliminary assessment. After 8 weeks, 8 of 8 patients in the 2% simvastatin group and 7 of 8 patients in the 2% simvastatin/2% cholesterol group showed better efficacy than placebo. By week 48, severity index and pruritus scores continued to improve, and no difference was seen between the 2 groups. No treatment-related adverse events were observed. This trial had a relatively small sample size. Both topical simvastatin and simvastatin/cholesterol were well tolerated and effective in relieving the erythema, thickness, scaling, and pruritus of PP with relatively long-term use.