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

Background: Porokeratosis ptychotropica (PP) is a severe inherited skin disorder resistant to traditional treatments. Topical statins are being explored as a potential alternative.

Objective: To evaluate the efficacy and safety of topical simvastatin and simvastatin/cholesterol in treating PP.

Methods: 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.

Results: 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.

Limitations: This trial had a relatively small sample size.

Conclusion: 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.

Keywords: cholesterol; mevalonate pathway; placebo-controlled; porokeratosis; porokeratosis ptychotropica; simvastatin; split-body; statins; topical therapy.