Tofacitinib for refractory ocular mucous membrane pemphigoid.

ABSTRACT

PURPOSE: To report the successful use of tofacitinib in the treatment of refractory ocular mucous membrane pemphigoid (MMP). OBSERVATIONS Two patients with ocular MMP presented with refractory disease after failure of multiple therapies. Treatment with tofacitinib led to durable control of conjunctival inflammation within 8 weeks and no apparent progression of sub-conjunctival fibrosis. One patient maintained absence of apparent disease activity over 16 months of follow-up. Cessation of tofacitinib in the other patient led to disease relapse which was reversed by re-initiation of therapy. CONCLUSIONS AND IMPORTANCE Small molecule inhibitors of Janus kinases, such as tofacitinib, may offer an effective treatment option for refractory ocular MMP.