Rheumatology, CHU Strasbourg, Centre National de Référence des maladies auto-immunes et systémiques rares Est/Sud-Ouest (RESO), Strasbourg, Alsace, France.
Rheumatology, Hospital Cavale-Blanche, Brest, Bretagne, France.
Ann Rheum Dis. 2021 Mar;80(3):329-338. doi: 10.1136/annrheumdis-2020-218467. Epub 2020 Nov 18.
No immunomodulatory drug has been approved for primary Sjögren's syndrome, a systemic autoimmune disease affecting 0.1% of the population. To demonstrate the efficacy of targeting interleukin 6 receptor in patients with Sjögren's syndrome-related systemic complications.
Multicentre double-blind randomised placebo-controlled trial between 24 July 2013 and 16 July 2018, with a follow-up of 44 weeks, involving 17 referral centres. Inclusion criteria were primary Sjögren's syndrome according to American European Consensus Group criteria and score ≥5 for the EULAR Sjögren's Syndrome Disease activity Index (ESSDAI, score of systemic complications). Patients were randomised to receive either 6 monthly infusions of tocilizumab or placebo. The primary endpoint was response to treatment at week 24. Response to treatment was defined by the combination of (1) a decrease of at least 3 points in the ESSDAI, (2) no occurrence of moderate or severe activity in any new domain of the ESSDAI and (3) lack of worsening in physician's global assessment on a Visual Numeric Scale ≥1/10, all as compared with enrolment.
110 patients were randomised, 55 patients to tocilizumab (mean (SD) age: 50.9 (12.4) years; women: 98.2%) and 55 patients to placebo (54.8 (10.7) years; 90.9%). At 24 weeks, the proportion of patients meeting the primary endpoint was 52.7% (29/55) in the tocilizumab group and 63.6% (35/55) in the placebo group, for a difference of -11.4% (95% credible interval -30.6 to 9.0) (Pr[Toc >Pla]=0.14).
Among patients with primary Sjögren's syndrome, the use of tocilizumab did not improve systemic involvement and symptoms over 24 weeks of treatment compared with placebo.
NCT01782235.
尚无免疫调节剂获批用于原发性干燥综合征，这是一种影响 0.1%人群的系统性自身免疫性疾病。本研究旨在评估靶向白细胞介素 6 受体治疗干燥综合征相关系统性并发症的疗效。
这是一项 2013 年 7 月 24 日至 2018 年 7 月 16 日进行的、为期 44 周的多中心、双盲、随机、安慰剂对照临床试验，涉及 17 个转诊中心。纳入标准为：符合美国欧洲共识组标准的原发性干燥综合征，EULAR 干燥综合征疾病活动指数（ESSDAI，系统性并发症评分）≥5 分。患者随机接受托珠单抗或安慰剂每 6 个月静脉输注 1 次。主要终点为治疗 24 周时的应答。应答定义为以下 3 项标准同时满足：（1）ESSDAI 评分降低至少 3 分；（2）任何新 ESSDAI 领域均无中度或重度活动；（3）医生整体评估的视觉模拟量表评分≥1/10，较基线无恶化。
共纳入 110 例患者，55 例接受托珠单抗治疗（平均年龄 50.9±12.4 岁，女性占 98.2%），55 例接受安慰剂治疗（平均年龄 54.8±10.7 岁，女性占 90.9%）。治疗 24 周时，托珠单抗组和安慰剂组分别有 52.7%（29/55）和 63.6%（35/55）的患者达到主要终点，差异为-11.4%（可信区间-30.6 至 9.0）（Pr[Toc >Pla]=0.14）。
在原发性干燥综合征患者中，与安慰剂相比，托珠单抗治疗 24 周并不能改善系统性疾病累及和症状。
NCT01782235。
