Abstract：
OBJECTIVE: 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.
BACKGROUND: ClinicalTrials.gov, NCT05394909.
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.
BACKGROUND: ClinicalTrials.gov, NCT05394909.
摘要：
目的：评估托珠单抗（TCZ）单药治疗在大血管GCA（LV-GCA）停药后一年的疗效维持。
方法：17例活动性LV-GCA患者先前接受3次静脉注射甲基强的松和每周皮下TCZ单药治疗52周。第52周无复发临床缓解的患者停止TCZ并进入第二部分，这是26周的观察性随访期。在26周观察期结束时（第78周）对所有患者进行PET/CT检查。终点是与基线和第52周相比，第78周的PET血管活动评分（PETVAS）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，在第78周观察到PETVAS显著降低,平均(95%CI)变化-6.6(-9.5~-3.7).然而,与第52周相比，PETVAS在TCZ停药后6个月（第78周）显着增加，平均（95%CI）变化4.6（0.7-8.5）。在第78周和随访结束时（从TCZ停药148周开始的中位时间）无复发临床缓解的患者比例为11/17（65％，95%CI38-86)和8/17(47%，95%CI23-72），分别。PETVAS每增加一个单位，经年龄和性别调整的HR（95％CI）表明随后的复发为1.36（0.92-2.00）。
结论：TCZ单药治疗一年可有效维持LV-GCA的无药临床缓解。TCZ停药后早期PETVAS的变化可能预测随后的复发。
背景：ClinicalTrials.gov，NCT05394909。
方法：17例活动性LV-GCA患者先前接受3次静脉注射甲基强的松和每周皮下TCZ单药治疗52周。第52周无复发临床缓解的患者停止TCZ并进入第二部分，这是26周的观察性随访期。在26周观察期结束时（第78周）对所有患者进行PET/CT检查。终点是与基线和第52周相比，第78周的PET血管活动评分（PETVAS）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，在第78周观察到PETVAS显著降低,平均(95%CI)变化-6.6(-9.5~-3.7).然而,与第52周相比，PETVAS在TCZ停药后6个月（第78周）显着增加，平均（95%CI）变化4.6（0.7-8.5）。在第78周和随访结束时（从TCZ停药148周开始的中位时间）无复发临床缓解的患者比例为11/17（65％，95%CI38-86)和8/17(47%，95%CI23-72），分别。PETVAS每增加一个单位，经年龄和性别调整的HR（95％CI）表明随后的复发为1.36（0.92-2.00）。
结论：TCZ单药治疗一年可有效维持LV-GCA的无药临床缓解。TCZ停药后早期PETVAS的变化可能预测随后的复发。
背景：ClinicalTrials.gov，NCT05394909。
