Apremilast Versus Placebo for Genital Erosive Lichen Planus in Women: A Randomized Controlled Trial.

Abstract

Objectives: We set out to evaluate the efficacy and safety of phosphodiesterase-4 inhibitor apremilast for moderate-to-severe genital erosive lichen planus (GELP) in women.

Materials and methods: In this randomized, double-blinded, placebo-controlled trial, women with moderate-to-severe GELP received either 30-mg apremilast tablets twice daily, or placebo tablets, for 24 weeks. According to pretrial power calculations, the study needed 40 participants to demonstrate a meaningful clinical effect. Efficacy was evaluated using a clinical scoring tool (GELP score). The primary endpoint was GELP scores at week 24 assessed by an Analysis of covariance model adjusted for baseline values. Secondary endpoints included Female Sexual Distress Scale-revised and Dermatology Life Quality Index scores.

Results: A total of 37 women with mean age 57.5 years and a mean GELP score of 14.2 were randomized to receive either apremilast (n = 19) or placebo (n = 18). In an intention-to-treat analysis at week 24, mean GELP score was 11.6 in the apremilast group and 11.6 in the placebo group with mean difference between the 2 groups being -0.4 (95% CI = -2.7 to 1.9; p = .71). No significant differences in the secondary endpoints, including Female Sexual Distress Scale-revised and Dermatology Life Quality Index scores, were observed. The per protocol analyses with 14 and 15 participants in each group revealed similar results. Adverse events were registered in 36 women, including serious adverse events in 2 women in the apremilast group.

Conclusions: Although having a small sample size, this randomized controlled trial did not reveal any signals indicating a clinical effect from apremilast treatment in women with moderate-to-severe GELP.