托珠单抗治疗白塞病：30 例患者的多中心研究。
Tocilizumab in Behçet Disease: A Multicenter Study of 30 Patients.
机构信息
M.Y. Khitri, MD, A. Bartoli, MD, G. Maalouf, MD, M. Vautier, MD, P. Cacoub, MD, PhD, D. Saadoun, MD, PhD, Department of Internal Medicine and Clinical Immunology France, Centre National de Référence Maladies Autoimmunes Systémiques Rares, Centre National de Référence Maladies Autoinflammatoires et Amylose, and Inflammation-Immunopathology-Biotherapy Department (DMU i3), Sorbonne Université, AP-HP, Hôpital Pitié Salpetrière, Paris, France.
A. Deroux, MD, Department of Internal Medicine, University Hospital of Grenoble, Grenoble, France.
出版信息
J Rheumatol. 2023 Jul;50(7):916-923. doi: 10.3899/jrheum.221106. Epub 2023 Mar 1.
OBJECTIVE
To evaluate tocilizumab (TCZ) efficacy in patients with refractory Behçet disease (BD).
METHODS
This is a multicenter study of 30 patients fulfilling the international criteria for BD and treated with TCZ at different European referral centers. The clinical response was evaluated at 6 months from TCZ initiation.
RESULTS
Ninety percent of patients with BD were refractory or intolerant to anti-tumor necrosis factor (anti-TNF) agents. Overall, TCZ was effective in 25 (83%) patients with BD of whom 18 (60%) and 7 (23%) were complete and partial responders, respectively. The complete response was 67%, 60%, and 42% in patients with uveitis (18/30), neurological manifestations (5/30), and mucocutaneous and/or articular (7/30) manifestations, respectively. TCZ had a significant steroid-sparing effect allowing patients to decrease their median daily prednisone dose from 20 (IQR 10-40) mg/day to 9 (IQR 5-13) mg at 6 months ( < 0.001). The number of patients with BD needing concomitant disease-modifying antirheumatic drug therapy fell from 7 (23%) to 4 (13%) at 6 months. Mild to moderate side effects were observed in 6 (20%) patients, and 3 (10%) presented with serious adverse events (pneumonia, intestinal perforation, and septicemia) requiring therapy discontinuation in 2 cases.
CONCLUSION
TCZ seems to be an effective alternative to anti-TNF agents in treating BD-related uveitis and neurological manifestations.
目的
评估托珠单抗（TCZ）在难治性贝赫切特病（BD）患者中的疗效。
方法
这是一项多中心研究，共纳入 30 例符合 BD 国际标准且在不同欧洲转诊中心接受 TCZ 治疗的患者。从 TCZ 起始 6 个月时评估临床反应。
结果
90%的 BD 患者对肿瘤坏死因子（anti-TNF）拮抗剂耐药或不耐受。总体而言，25 例（83%）BD 患者对 TCZ 有效，其中 18 例（60%）和 7 例（23%）分别为完全和部分应答者。在有葡萄膜炎（18/30）、神经系统表现（5/30）和黏膜皮肤和/或关节（7/30）表现的患者中，完全缓解率分别为 67%、60%和 42%。TCZ 具有显著的激素节省作用，可使患者在 6 个月时将每日泼尼松中位数剂量从 20（IQR 10-40）mg 降至 9（IQR 5-13）mg（<0.001）。需要同时使用疾病修正抗风湿药物治疗的 BD 患者数量从 7（23%）降至 6 个月时的 4（13%）。6（20%）例患者出现轻度至中度不良反应，3（10%）例出现严重不良事件（肺炎、肠穿孔和败血症），2 例需要停止治疗。
结论
TCZ 似乎是治疗 BD 相关葡萄膜炎和神经系统表现的抗 TNF 拮抗剂的有效替代药物。
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