Objectives To assess the maintenance of efficacy of one year of tocilizumab (TCZ) monotherapy after its discontinuation in large vessel-GCA (LV-GCA). Methods 17 patients with active LV-GCA were previously treated with 3 boluses of intravenous methylprednisone and weekly subcutaneous TCZ in monotherapy for 52 weeks. Patients in relapse-free clinical remission at week 52 discontinued TCZ and entered part two, which was a 26-week observational follow-up period. PET/CT was performed in all patients at the end of the 26-week observational period (week 78). End points were the variation in PET vascular activity score (PETVAS) at week 78 compared with baseline and with week 52, and the proportion of patients with relapse-free clinical remission at week 78 and at the end of the follow-up. Results Compared with baseline, a significant reduction in PETVAS was observed at week 78, mean (95% CI) change -6.6 (-9.5 to -3.7). However, compared with week 52, PETVAS significantly increase 6 months after TCZ discontinuation (week 78), mean (95% CI) change 4.6 (0.7–8.5). The proportion of patients with relapse-free clinical remission at weeks 78 and at the end of the follow-up (median time from TCZ discontinuation 148 weeks) was 11/17 (65%, 95% CI 38–86) and 8/17 (47%, 95% CI 23–72), respectively. Age and sex–adjusted HR (95% CI) for each unit increase of PETVAS indicating subsequent relapse was 1.36 (0.92–2.00). Conclusions One year of TCZ monotherapy was effective in maintaining drug-free clinical remission in LV-GCA. Changes in PETVAS early after TCZ discontinuation may predict subsequent relapses. Clinical trial registration ClinicalTrials.gov, NCT05394909

中文翻译：

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用超短效糖皮质激素和托珠单抗治疗巨细胞动脉炎：TOPAZIO 研究扩展的结果


目的 评估大血管-GCA (LV-GCA) 托珠单抗 (TCZ) 单药治疗停药后一年的疗效维持情况。方法 17 例活动性 LV-GCA 患者先前接受 3 次静脉推注甲基强的松和每周皮下注射 TCZ 单药治疗 52 周。第 52 周达到无复发临床缓解的患者停用 TCZ 并进入第二部分，即为期 26 周的观察随访期。在 26 周观察期结束时（第 78 周），所有患者均进行了 PET/CT 检查。终点是第78周时PET血管活动评分（PETVAS）与基线和第52周相比的变化，以及第78周和随访结束时无复发临床缓解的患者比例。结果 与基线相比，第 78 周时观察到 PETVAS 显着降低，平均 (95% CI) 变化为 -6.6（-9.5 至 -3.7）。然而，与第 52 周相比，TCZ 停用后 6 个月（第 78 周）PETVAS 显着增加，平均 (95% CI) 变化 4.6 (0.7–8.5)。第 78 周和随访结束时（TCZ 停药中位时间 148 周）达到无复发临床缓解的患者比例为 11/17（65%，95% CI 38-86）和 8/17 (47%, 95% CI 23–72)。 PETVAS 每增加一个单位，表明随后复发的年龄和性别调整 HR (95% CI) 为 1.36 (0.92–2.00)。结论 一年的 TCZ 单药治疗可有效维持 LV-GCA 的无药临床缓解。 TCZ 停用后早期 PETVAS 的变化可能预测随后的复发。临床试验注册ClinicalTrials.gov，NCT05394909