Abstract
BACKGROUND: Ofatumumab, a fully human anti-CD20 monoclonal antibody administered subcutaneously and indicated for multiple sclerosis, might theoretically be effective for patients with pemphigus vulgaris (PV). OBJECTIVE: To evaluate the effectiveness and safety of ofatumumab in patients with PV. METHODS: This cohort study was based on a registry database of autoimmune bullous diseases at West China Hospital (AIBD-WCH), including two groups. One was ofatumumab (OFA) group, involving patients receiving ofatumumab subcutaneous injections (2×20mg, 2 weeks apart) and systemic glucocorticoids with/without immunosuppressant. The glucocorticoids control (GC) group was matched using propensity score matching in a 1:2 ratio based on sex, age and body mass index. Both groups completed regular follow-up for 52 weeks. The primary endpoint was the proportion of patients achieving complete remission during therapy (CRDT) at week 52. Secondary endpoints included maintaining treatment (MT) with daily prednisone doses <0.2 mg/kg/d, relapse rate, the change of pemphigus disease area index and cumulative glucocorticoid doses. Safety results were also collected. RESULTS: Sixteen and thirty-two patients were included in OFA and GC groups, respectively. At week 52, more patients in OFA group achieved CRDT (31.2% versus 3.12%, p=0.012) and MT (68.8% versus 25.0%, p=0.009). Furthermore, patients in OFA group took lower cumulative glucocorticoid doses by week 52 (6186 [SD: 1177]mg versus 9317 [SD: 1579]mg, p<0.001). A patient in OFA group experienced gastric hemorrhage, which was judged to be unrelated to ofatumumab, while two in GC group developed lung infections. CONCLUSIONS: Ofatumumab combined with glucocorticoids demonstrated favorable effectiveness compared with GC group, without increasing severe adverse events.