目的:我们评估了托珠单抗在日本Takayasu动脉炎(TAK)患者中的真实世界耐受性和有效性。
方法:在过去6个月内未接受托珠单抗的TAK患者纳入ACT-Bridge,第四阶段,观察性研究,来自66所日本机构(入学期间,2017年9月至2020年9月),并每周皮下接受162mg托珠单抗(观察期,52周)。
结果:在纳入的120例患者中(平均年龄,38.4年;平均疾病持续时间,7.7年;治疗复发,50.8%;既往使用过免疫抑制剂,57.5%;基线时使用糖皮质激素,97.5%),49例(40.8%)报告了不良事件(AE)。特别感兴趣的最常见的AE是严重感染(7.5%)。在24例(20.0%)患者中观察到复发(0.8%,2.5%和16.7%报告≥3、2和1复发,分别)。诊断复发的原因包括胸痛和背痛(45.8%),颈部疼痛(25.0%),疲劳(16.7%),发烧和头痛(各12.5%),影像学发现异常(50.0%)和炎症标志物升高(16.7%)。在最后的观察中,83.0%的无复发患者记录伴随糖皮质激素剂量(泼尼松龙当量)<10mg/天。
结论:这项研究证明了托珠单抗在TAK患者中的有效性,没有新的安全问题。Tocilizumab加糖皮质激素可被认为是TAK的治疗选择。
OBJECTIVE: We evaluated the real-world tolerability and effectiveness of tocilizumab in Japanese patients with Takayasu arteritis (TAK).
METHODS: Patients with TAK who had not received tocilizumab in the previous 6 months were enrolled in ACTEMRA® (ACT)-Bridge, a phase 4, observational
study, from 66 Japanese institutions (enrolment period, September 2017 to September 2020) and received weekly subcutaneous tocilizumab 162 mg (observation period, 52 weeks).
RESULTS: Among 120 patients included (mean age, 38.4 years; mean disease duration, 7.7 years; treated for relapse, 50.8%; previous immunosuppressant use, 57.5%; glucocorticoid use at baseline, 97.5%), 49 (40.8%) reported adverse events. The most common adverse event of special interest was serious infection (7.5%). Relapse was observed in 24 (20.0%) patients (0.8%, 2.5%, and 16.7% reporting ≥3, 2, and 1 relapses, respectively). The reasons for diagnosing relapse included chest and back pain (45.8%), neck pain (25.0%), fatigue (16.7%), fever and headache (12.5% each), abnormal imaging findings (50.0%), and elevated inflammatory markers (16.7%). At the last observation, 83.0% of relapse-free patients recorded a concomitant glucocorticoid dose (prednisolone equivalent) <10 mg/day.
CONCLUSIONS: This
study demonstrated the effectiveness of tocilizumab in patients with TAK, with no new safety concerns. Tocilizumab plus glucocorticoids may be considered a treatment option for TAK.