Zhang Xiwen, Wang Yiyi, Tan Ping, Zhou Xingli, Xiao Yue, Feng Xun, Li Jishu, Wei Mintong, Zou Min, Kim Gyeongah, Jiang Lu, Li Xiaohong, Wang Jinqiu, Wang Mi, Li Wei
Department of Dermatology & Rare Disease Center, West China Hospital, Sichuan University, Chengdu, Sichuan, China.
West China School of Nursing, Sichuan University, Chengdu, Sichuan, China.
Front Immunol. 2025 Jul 25;16:1537334. doi: 10.3389/fimmu.2025.1537334. eCollection 2025.
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).
To evaluate the effectiveness and safety of ofatumumab in patients with PV.
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.
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%, =0.012) and MT (68.8% versus 25.0%, =0.009). Furthermore, patients in OFA group took lower cumulative glucocorticoid doses by week 52 (6186 [SD: 1177]mg versus 9317 [SD: 1579]mg, <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.
Ofatumumab combined with glucocorticoids demonstrated favorable effectiveness compared with GC group, without increasing severe adverse events.
奥法妥木单抗是一种皮下注射的全人源抗CD20单克隆抗体，被批准用于治疗多发性硬化症，理论上可能对寻常型天疱疮（PV）患者有效。
评估奥法妥木单抗治疗PV患者的有效性和安全性。
本队列研究基于华西医院自身免疫性大疱性疾病注册数据库（AIBD-WCH），分为两组。一组为奥法妥木单抗（OFA）组，包括接受奥法妥木单抗皮下注射（2×20mg，间隔2周）及全身应用糖皮质激素（联合或不联合免疫抑制剂）的患者。糖皮质激素对照组（GC）采用倾向评分匹配法，按照1:2的比例根据性别、年龄和体重指数进行匹配。两组均进行为期52周的定期随访。主要终点为第52周时治疗期间达到完全缓解（CRDT）的患者比例。次要终点包括每日泼尼松剂量<0.2mg/kg/d的维持治疗（MT）、复发率、天疱疮疾病面积指数变化及糖皮质激素累积剂量。同时收集安全性结果。
OFA组和GC组分别纳入16例和32例患者。在第52周时，OFA组达到CRDT的患者更多（31.2%对3.12%，P=0.012），且MT患者更多（68.8%对25.0%，P=0.009）。此外，至第52周时，OFA组患者的糖皮质激素累积剂量更低（6186[标准差：1177]mg对9317[标准差：1579]mg，P<0.001）。OFA组有1例患者发生胃出血，判定与奥法妥木单抗无关，而GC组有2例患者发生肺部感染。
与GC组相比，奥法妥木单抗联合糖皮质激素显示出良好的有效性，且未增加严重不良事件。
