Safety and tolerability of adjuvant topical tacrolimus treatment in boys with lichen sclerosus: a prospective phase 2 study.

BACKGROUND

Management of lichen sclerosus (LS) in boys is still controversial. Although in most cases only the prepuce is affected, meatal and urethral involvement may require major surgical reconstruction with substantial morbidity.

OBJECTIVE

Because the frequency of such complicated courses is still unclear, an adjuvant postoperative treatment is highly desirable. Therefore, we addressed safety and tolerability of tacrolimus 0.1% ointment in the postoperative period.

DESIGN, SETTING, AND PARTICIPANTS: Among 222 penile surgeries, in 25 cases LS was confirmed histologically and 20 of those patients participated in the adjuvant treatment study. Moreover, 18 patients of the same cohort showed a lichenoid inflammatory reaction pattern suggestive of early but not fully established LS.

INTERVENTIONS AND MEASUREMENTS

Subsequent to the operation and after explicit information about off-label use, parents applied tacrolimus 0.1% ointment twice daily to the glans and the meatus for 3 wk in cases of proven LS. The 18 patients with possible early LS were followed up only without any treatment. Clinical follow-up was performed up to 13 mo (median).

RESULTS AND LIMITATIONS

All 20 LS patients completed the topical treatment without any relevant side-effects. Two relapses occurred in the treatment group and were clinically cured with an additional 3-wk cycle of topical tacrolimus 0.1% ointment. None of the 18 early LS cases progressed to full-scale LS.

CONCLUSIONS

This is the first study showing that tacrolimus 0.1% ointment applied immediately after surgery of fully established LS is a tolerable and most probably safe adjuvant novel treatment option. Because the therapy led to disease control in all treated individuals for >1 yr (median), this study establishes the groundwork for future trials with expanded treatment and follow-up periods to verify the true clinical benefit of tacrolimus in patients after LS surgery. Lichenoid tissue reactions suggestive of early LS seem to require no adjuvant treatment.