Moots Robert J, Fortune Farida, Jackson Richard, Thornburn Tony, Morgan Ann, Carr Daniel F, Murray Philip I, Wallace Graham R, Situnayake Deva
Department of Academic Rheumatology, Liverpool University Hospitals NHS Foundation Trust, Aintree University Hospital, Liverpool, UK.
Faculty of Health, Social Care and Medicine, Edge Hill University, Ormskirk, UK.
Rheumatology (Oxford). 2025 May 1;64(5):2882-2891. doi: 10.1093/rheumatology/keae585.
Whilst biologic therapy is used for Behçet's syndrome of all subtypes refractory to first-line immunomodulation, there has been an absence of high-quality evidence and no predictive biomarkers to optimally inform choice. BIO-BEHÇET'S was a randomized, controlled, head-to-head clinical trial comparing the two most frequently used biologics in active refractory Behçet's.
This was a Bayesian-designed, pragmatic, standard of care, two-arm, parallel head-to-head trial at four UK centres. Patients with active disease were randomized to infliximab or IFN-α2a, and received follow-up with symptom-directed examination at weeks 12 and 24. The primary outcome was the Behçet's Disease Activity Index (BDAI) at 12 weeks. Secondary outcomes included BDAI at 24 weeks and significant improvement in individual organ systems, including ocular symptoms, oral and genital ulcers, arthritis pain, quality of life, disease activity and steroid use. Biomarkers were also investigated but are reported elsewhere.
Seventy-nine patients were recruited. Both treatments were equally effective, with a mean difference of 0.13 in BDAI (80% confidence interval: -0.19, 0.46). No significant differences were observed for secondary outcomes, though there were clinically significant within-group reductions for each over time. A modest steroid-sparing effect was observed, with complete cessation of steroids in 20% and 44% of those randomized to infliximab and IFN-α2a, respectively. There was a trend for minor benefit in favour of infliximab in terms of tolerability and persistence.
In this first reported, high-quality, head-to-head trial of two biologics in Behçet's, both infliximab and IFN-α2a showed comparable short-term clinical efficacy and safety in refractory active disease of all subtypes.
EudraCT: 2014-005390-36; ISRCTN: ISRCTN49793874.
虽然生物疗法用于一线免疫调节难治的所有亚型白塞病，但缺乏高质量证据且没有预测生物标志物来优化治疗选择。BIO-BEHÇET'S是一项随机、对照、直接比较的临床试验，对比了在活动性难治性白塞病中最常用的两种生物制剂。
这是一项在英国四个中心进行的贝叶斯设计、务实、标准治疗、双臂、平行直接比较试验。活动性疾病患者被随机分配接受英夫利昔单抗或干扰素-α2a治疗，并在第周和第24周接受症状导向检查的随访。主要结局是第12周时的白塞病活动指数（BDAI）。次要结局包括第24周时的BDAI以及各个器官系统的显著改善，包括眼部症状、口腔和生殖器溃疡、关节炎疼痛、生活质量、疾病活动和类固醇使用。还对生物标志物进行了研究，但在其他地方报告。
招募了79名患者。两种治疗同样有效，BDAI的平均差异为0.13（80%置信区间：-0.19，0.46）。次要结局未观察到显著差异，尽管随着时间推移每组在临床上均有显著降低。观察到适度的类固醇节省效应，随机接受英夫利昔单抗和干扰素-α2a治疗的患者中分别有20%和44%完全停用了类固醇。在耐受性和持续性方面，有轻微倾向表明英夫利昔单抗有较小益处。
在这项首次报道的、高质量的、两种生物制剂治疗白塞病的直接比较试验中，英夫利昔单抗和干扰素-α2a在所有亚型的难治性活动性疾病中均显示出相当的短期临床疗效和安全性。
EudraCT：2014-005390-36；ISRCTN：ISRCTN49793874。
