Efficacy and safety of pioglitazone in lichen planopilaris in comparison with hydroxychloroquine: Prospective non-inferiority study.

BACKGROUND AND

OBJECTIVES

Pioglitazone is an underused drug in lichen planopilaris (LPP) with limited prospective evidence. This study investigated the therapeutic efficacy and safety profile of pioglitazone compared with hydroxychloroquine.

PATIENTS AND

METHODS

This reviewer-blinded, prospective non-inferiority study was conducted at a tertiary university hospital in Belgrade, Serbia, between October 2023 and January 2025. Patients with LPP affecting < 20 % of the scalp received pioglitazone (exposed cohort), whereas those with > 20 % scalp involvement received hydroxychloroquine (HCQ; control cohort). Patients were evaluated at baseline and after 3 and 6 months. The primary outcome measures were the Lichen Planopilaris Activity Index (LPPAI) and Global Photographic Assessment (GPA).

RESULTS

In total, 82 consecutive patients (42 pioglitazone, 40 HCQ) were enrolled. 66 patients (80.49 %) were analyzed using a per-protocol approach. The mean LPPAI at baseline and last session were 3.32 ± 1.23 and 0.95 ± 0.74 in the pioglitazone group and 3.64 ± 1.46 and 1.13 ± 0.9 in the hydroxychloroquine cohort, respectively. No significant difference in LPPAI reduction (p = 0.66) was observed between cohorts. Pioglitazone prevents disease progression in 87.5 % cases. No statistical difference between cohorts in GPA score was seen (p = 0.98). Both treatment options were well-tolerated (p = 0.73).

CONCLUSIONS

This study provides the largest prospective pioglitazone cohort reported to date in LPP and demonstrates that pioglitazone is non-inferior to hydroxychloroquine as a first-line systemic treatment, while also exhibiting a strong safety profile.