Mycophenolate Mofetil for Genital and Extragenital Lichen Sclerosus Et Atrophicus

To the Editor,

Lichen sclerosus et atrophicus (LSA) is a chronic inflammatory mucocutaneous disease with an incompletely characterized etiology. ¹ Although topical corticosteroids are appropriate for limited, active disease, some patients may have extensive, rapidly progressive, and/or refractory disease warranting systemic therapy. ^2,3 Systemic corticosteroids’ side effects preclude long-term use, and evidence for steroid-sparing agents such as mycophenolate mofetil (MMF) in LSA is sparse. ⁴ We assessed the response of 11 patients who received MMF for LSA.

Upon Institutional Review Board approval, a retrospective review of patients who received MMF for LSA at Atrium Health Wake Forest Baptist between 2010-2022 was performed. Patients without follow-up evaluation were excluded. Outcomes were classified as response (reduced pruritus, pain, erythema, absence of progression as evidenced by lack of anatomic changes and spread) and no response. We evaluated both genital and extragenital LSA.

The patients were primarily middle-aged (mean 62 ± 8 years) females (91%) who presented with white and/or erythematous (100%) atrophic (73%) plaques on the trunk (73%), vulva (55%), and lower extremities (45%). Eight (73%) had biopsy-proven disease. Patients presented with genital (11%), extragenital (45%), or extragenital and genital (36%) involvement. Most patients reported pruritus (91%) and pain (73%). Medications attempted before MMF included topical corticosteroids (73%), methotrexate (55%), topical tacrolimus (27%), and systemic steroids (27%). Disease existed a median of 13 months before MMF initiation (median daily dose was 1.3 g, median duration 11 months). Ten (91%) patients concomitantly applied topical corticosteroids (Supplemental Table 1).

Ten (91%) patients responded to MMF within a median 4 months (median response dose, 1.5 g). Median time to response and median response dose varied by site of involvement; it was most challenging to achieve response in LSA limited to extragenital sites (extragenital: 8.5 months, 1.5 g; genital: 3.25 months, 1.5 g; combination: 2 months, 1 g). Responders experienced cessation of disease progression and spread (100%), and a decrease in their baseline pruritus (100%), pain (100%), and skin thickening (50%). Skin whitening and atrophy did not resolve with MMF. There was no substantial difference in response between sites.

Six (55%) patients (3 limited extragenital, 3 combination) improved substantially with MMF and attempted tapers. Three responders flared and re-achieved response after MMF dosage increase (dose at flare: 0.25-0.75 g; dose to re-achieve response: 1-1.5 g). Three responders are undergoing tapers at the time of this analysis and have not flared. MMF was generally well-tolerated; side effects included: confusion (18%), constipation and insomnia, tinnitus, pruritus, and throat irritation (9% each) (Supplemental Table 1).

Although this study is limited by its small sample size, subjective outcome measures at various anatomic sites with non-standardized time points, and flares which may represent natural disease fluctuation, it appears MMF may be helpful for patients with resistant anogenital and extragenital LSA.

Supplemental Material

Table S1 - Supplemental material for Mycophenolate Mofetil for Genital and Extragenital Lichen Sclerosus Et Atrophicus

Supplemental material, Table S1, for Mycophenolate Mofetil for Genital and Extragenital Lichen Sclerosus Et Atrophicus by Palak V. Patel, Matthew L. Hrin, Steven R. Feldman and Rita Pichardo in Journal of Cutaneous Medicine and Surgery