A pilot study of topical imiquimod therapy for the treatment of recurrent extramammary Paget's disease

• Conservative therapy is proposed for recurrent EMPD of the vulva.
• Imiquimod, an anti-tumor immune response modifier, can be used off-label for EMPD.
• Complete clinical and histologic responses are achievable using topical imiquimod.

ObjectiveThe objective of this prospective pilot study was to assess the clinical and histologic effects of topical imiquimod therapy on recurrent extramammary Paget's disease of the vulva.MethodsPatients with biopsy-proven recurrent extramammary Paget's disease presenting to the gynecology outpatient services at two participating institutions were recruited for conservative treatment with 5% imiquimod cream from 2007 to 2011. The topical cream was to be applied 3 times per week for 12 weeks. Punch biopsy and photography were performed at baseline and at the 12-week time point.ResultsEight patients from two institutions were enrolled. Complete clinical and histologic response was achieved in 6 (75%) patients by the 12-week follow-up appointment. Of the two remaining patients, one had a complete clinical response but no significant histologic response; the other patient was removed from the study protocol secondary to intolerable local irritation. Two patients continue to have no evidence of disease after a median follow-up of 35 months. Five are alive with disease. No patients progressed to invasive cancer while receiving therapy.ConclusionTopical 5% imiquimod cream is a safe and feasible option for women suffering from recurrent extramammary Paget's disease of the vulva, and should be considered as a viable alternative to surgical management. Given the rare nature of this disease, additional multi-institutional prospective studies should be conducted to explore the efficacy of this treatment regime.