Departments of Rheumatology, Internal Medicine and Pathology, Hospital Universitario Marqués de Valdecilla, IDIVAL, Universidad de Cantabria, Santander, Spain.
Department of Rheumatology, Complejo Hospitalario de Navarra, Navarra, Spain.
Semin Arthritis Rheum. 2019 Aug;49(1):126-135. doi: 10.1016/j.semarthrit.2019.01.003. Epub 2019 Jan 5.
Tocilizumab (TCZ) has shown efficacy in clinical trials on giant cell arteritis (GCA). Real-world data are scarce. Our objective was to assess efficacy and safety of TCZ in unselected patients with GCA in clinical practice Methods: Observational, open-label multicenter study from 40 national referral centers of GCA patients treated with TCZ due to inefficacy or adverse events of previous therapy. Outcomes variables were improvement of clinical features, acute phase reactants, glucocorticoid-sparing effect, prolonged remission and relapses. A comparative study was performed: (a) TCZ route (SC vs. IV); (b) GCA duration (≤6 vs. >6 months); (c) serious infections (with or without); (d) ≤15 vs. >15 mg/day at TCZ onset.
134 patients; mean age, 73.0 ± 8.8 years. TCZ was started after a median [IQR] time from GCA diagnosis of 13.5 [5.0-33.5] months. Ninety-eight (73.1%) patients had received immunosuppressive agents. After 1 month of TCZ 93.9% experienced clinical improvement. Reduction of CRP from 1.7 [0.4-3.2] to 0.11 [0.05-0.5] mg/dL (p < 0.0001), ESR from 33 [14.5-61] to 6 [2-12] mm/1st hour (p < 0.0001) and decrease in patients with anemia from 16.4% to 3.8% (p < 0.0001) were observed. Regardless of administration route or disease duration, clinical improvement leading to remission at 6, 12, 18, 24 months was observed in 55.5%, 70.4%, 69.2% and 90% of patients. Most relevant adverse side-effect was serious infections (10.6/100 patients-year), associated with higher doses of prednisone during the first three months of therapy.
In clinical practice, TCZ yields a rapid and maintained improvement of refractory GCA. Serious infections appear to be higher than in clinical trials.
托珠单抗（TCZ）在巨细胞动脉炎（GCA）的临床试验中显示出疗效。真实世界的数据很少。我们的目的是评估 TCZ 在临床实践中未经选择的 GCA 患者中的疗效和安全性。
这是一项来自 40 个 GCA 国家转诊中心的观察性、开放性、多中心研究，纳入了因先前治疗无效或出现不良反应而接受 TCZ 治疗的 GCA 患者。观察指标包括临床症状、急性期反应物、糖皮质激素节省效应、延长缓解期和复发率的改善情况。同时还进行了以下比较研究：（a）TCZ 给药途径（SC 与 IV）；（b）GCA 病程（≤6 个月与＞6 个月）；（c）严重感染（有或无）；（d）TCZ 起始时剂量≤15mg/天与＞15mg/天。
共纳入 134 例患者，平均年龄为 73.0±8.8 岁。从 GCA 诊断到 TCZ 治疗的中位（IQR）时间为 13.5[5.0-33.5]个月。98（73.1%）例患者接受过免疫抑制剂治疗。TCZ 治疗 1 个月后，93.9%的患者临床症状改善。CRP 从 1.7[0.4-3.2]mg/dL 降至 0.11[0.05-0.5]mg/dL（p＜0.0001），ESR 从 33[14.5-61]mm/1h 降至 6[2-12]mm/1h（p＜0.0001），贫血患者比例从 16.4%降至 3.8%（p＜0.0001）。无论给药途径或疾病病程如何，在第 6、12、18、24 个月时，分别有 55.5%、70.4%、69.2%和 90%的患者达到临床缓解。最常见的不良反应是严重感染（10.6/100 患者年），与治疗前 3 个月内较高剂量的泼尼松相关。
在临床实践中，TCZ 可迅速且持续改善难治性 GCA，疗效显著。但严重感染的发生率似乎高于临床试验。
