04/16/2025
Pemphigus vulgaris (PV) is a severe autoimmune blistering disorder that often starts with painful mucosal erosions and can progress to widespread skin involvement. Standard treatments typically include corticosteroids, rituximab, and steroid-sparing agents, but some patients remain refractory despite aggressive therapy 💊. In this case report by Dr. Saakshi Khattri at the Icahn School of Medicine, upadacitinib, a selective JAK1 inhibitor, was successfully used to achieve remission in a patient with persistent mucosal PV.
🧵https://bit.ly/3ztQ8cz
This 64-year-old patient had failed multiple therapies — including rituximab, IVIG, mycophenolate, and high-dose corticosteroids — yet continued to suffer from painful oral lesions and poor quality of life 🥄. After starting off-label upadacitinib 15 mg daily alongside IVIG and rituximab, the patient's mucosal lesions steadily healed, PO intake improved, and no new lesions developed ✨. After 6 months of therapy, the patient achieved corticosteroid-free remission with no new cutaneous or mucosal lesions 🎯.
This case is important because JAK-STAT signaling is implicated in PV pathogenesis via aberrant Th1/Th2 cytokine activity (including IFN-γ, IL-4, IL-6, IL-10, IL-15) which drive B-cell activation and autoantibody production 🧬. By targeting this pathway, upadacitinib may help break the cycle of inflammation and tissue injury in PV 🔄.
While randomized data are still needed, this report suggests JAK inhibitors could represent a promising adjunct in refractory PV, particularly for difficult-to-treat mucosal involvement where conventional options may fall short 🌟.
Icahn School of Medicine at Mount Sinai
The Mount Sinai Hospital
