托珠单抗治疗未经治疗的 Takayasu 动脉炎患者：TOCITAKA 法国前瞻性多中心开放标签试验。
Tocilizumab in treatment-naïve patients with Takayasu arteritis: TOCITAKA French prospective multicenter open-labeled trial.
机构信息
Sorbonne Universités AP-HP, Hôpital Saint Antoine, service de médecine interne et Inflammation-Immunopathology-Biotherapy Department (DMU 3iD), Faculté de Médecine Sorbonne Université, F-75012, Paris, France.
Sorbonne Universités AP-HP, Groupe Hospitalier Pitié-Salpêtrière, Département de Médecine Interne et Immunologie Clinique, National center for Autoimmune Systemic rare disease ; National center for Autoinflammatory diseases and amyloidosis, Inflammation-Immunopathology-Biotherapy Department (DMU 3iD), INSERM, UMR_S 959, F-75013, Paris, France.
出版信息
Arthritis Res Ther. 2020 Sep 17;22(1):218. doi: 10.1186/s13075-020-02311-y.
OBJECTIVES
To assess long-term efficacy of tocilizumab in treatment-naive patients with Takayasu arteritis (TAK).
METHODS
Prospective open-labeled trial in naïve patients with TAK who received steroids at the dose of 0.7 mg/kg/day and 7 infusions of 8 mg/kg/month of tocilizumab. The primary endpoint was the number of patients who discontinued steroids after 7 infusions of tocilizumab. Secondary endpoints included disease activity and the number of relapses during 18-month follow-up.
RESULTS
Thirteen patients with TAK were included, with a median age of 32 years [19-45] and 12 (92%) females. Six (54%) patients met the primary end-point. A significant decrease of disease activity was observed after 6 months of tocilizumab therapy: decrease of median NIH scale (3 [3, 4] at baseline, versus 1 [0-2] after 6 months; p < 0.001), ITAS-2010 score (5 [2-7] versus 3 [0-8]; p = 0.002), and ITAS-A score (7 [4-10] versus 4 [1-15]; p = 0.0001)]. During the 12-month follow-up after tocilizumab discontinuation, a relapse occurred among 5 patients (45%) out of 11 in which achieved remission after 6 months of tocilizumab.
CONCLUSION
Tocilizumab seems an effective steroid sparing therapy in TAK, but maintenance therapy is necessary.
TRIAL REGISTRATION
ClinicalTrials.gov NCT02101333 . Registered on 02 April 2014.
目的
评估托珠单抗治疗未经治疗的多发性大动脉炎（TAK）患者的长期疗效。
方法
本研究为前瞻性、开放性、单臂临床试验，纳入未经治疗的 TAK 患者，在给予托珠单抗 8mg/kg/月（7 次）治疗前，患者接受 0.7mg/kg/日的激素治疗。主要终点为 7 次托珠单抗治疗后停用激素的患者比例。次要终点包括 18 个月随访期间的疾病活动度和复发率。
结果
共纳入 13 例 TAK 患者，中位年龄 32 岁（19-45 岁），12 例（92%）为女性。6 例（54%）患者达到主要终点。托珠单抗治疗 6 个月后，疾病活动度显著降低：NIH 评分中位数从基线时的 3 分[3，4]降至 6 个月时的 1 分[0-2]；p<0.001）、ITAS-2010 评分（5 分[2-7] vs 3 分[0-8]；p=0.002）和 ITAS-A 评分（7 分[4-10] vs 4 分[1-15]；p=0.0001）。托珠单抗停药后 12 个月随访期间，11 例患者中的 5 例（45%）复发，其中 6 个月时达到缓解。
结论
托珠单抗似乎是一种有效的 TA K 激素保留治疗方法，但需要维持治疗。
临床试验注册
ClinicalTrials.gov 注册号 NCT02101333 ，于 2014 年 4 月 2 日注册。
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