Clinical outcomes of patients with giant cell arteritis treated with tocilizumab in real-world clinical practice: decreased incidence of new visual manifestations.
Clinical outcomes of patients with giant cell arteritis treated with tocilizumab in real-world clinical practice: decreased incidence of new visual manifestations.
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DOI：
10.1186/s13075-020-02377-8
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发表时间：
2021-01-06
影响因子：
4.9
通讯作者：
Stone JH
中科院分区：
文献类型：
--
作者：
Unizony S;McCulley TJ;Spiera R;Pei J;Sidiropoulos PN;Best JH;Birchwood C;Pavlov A;Stone JH
安慰剂对照的临床试验已经证明了托珠单抗（TCZ）在巨细胞动脉炎（GCA）患者中维持缓解和糖皮质激素保留的疗效。然而，关于TCZ在现实临床实践中治疗GCA的有效性和安全性的数据有限。这是一项对接受静脉或皮下TCZ治疗的GCA患者进行的回顾性、单中心分析（2010-2018）。TCZ治疗开始前后评价的结局包括发作发生率、至发作时间、年发作率、发作特征（即，风湿性多肌痛[PMR]症状、颅骨表现）、泼尼松使用和安全性。发作定义为需要强化治疗的明确GCA表现复发。对GCA诊断时有PMR或视觉表现的患者进行亚组分析。纳入60例GCA患者。TCZ治疗前和治疗后的中位（IQR）病程分别为0.6（0.2-1.6）年和0.5（0.3-1.4）年。TCZ治疗开始前43例（71.7%）和治疗开始后18例（30.0%）患者至少观察到1次发作。TCZ治疗前至发作的中位（IQR）时间为0.5（0.3-0.7）年，TCZ治疗开始后为2.1（0.6-2.6）年（HR 0.22; 95% CI 0.10-0.50; p = 0.0003）。使用TCZ后，年发作率显著降低（TCZ前1.4 [95%CI 1.0-2.1]; TCZ后0.6 [95%CI 0.3-1.0]事件/年; p < 0.001）。在GCA诊断时有视觉表现或PMR症状的患者中观察到类似的改善。TCZ降低了新的视觉表现的发生率，并且当患者接受TCZ时，没有发生与永久性视力丧失相关的闪光。TCZ开始时和随访结束时的平均（SD）泼尼松剂量分别为30（18.3）和5（6.9）mg/天（p < 0.0001）。TCZ开始后，46.6%的患者成功停用泼尼松。主要归因于糖皮质激素的不良事件的发生率在TCZ开始之前和之后相似。在这种现实环境中，TCZ显著改善了GCA的临床结局，并在GCA诊断时具有PMR症状和GCA相关视觉表现的患者亚组中证明了有效性。TCZ启动后未发生新的失明病例。不良事件，许多归因于糖皮质激素，TCZ治疗前后相当。
Placebo-controlled clinical trials have demonstrated the efficacy of tocilizumab (TCZ) for remission maintenance and glucocorticoid sparing in patients with giant cell arteritis (GCA). However, limited data exist on the effectiveness and safety of TCZ for GCA in real-world clinical practice. This was a retrospective, single-center analysis of patients with GCA treated with intravenous or subcutaneous TCZ (2010–2018). Outcomes evaluated before and after TCZ initiation included occurrence of flare, time to flare, annualized flare rate, flare characteristics (i.e., polymyalgia rheumatica [PMR] symptoms, cranial manifestations), prednisone use, and safety. Flare was defined as the recurrence of unequivocal GCA manifestations requiring treatment intensification. Subgroup analyses of patients with PMR or visual manifestations at GCA diagnosis were performed. Sixty patients with GCA were included. The median (IQR) disease duration before and after the start of TCZ was 0.6 (0.2–1.6) and 0.5 (0.3–1.4) years, respectively. At least 1 flare was observed in 43 patients (71.7%) before and in 18 (30.0%) after TCZ initiation. Median (IQR) time to flare was 0.5 (0.3–0.7) years before TCZ treatment and 2.1 (0.6–2.6) years after TCZ initiation (HR 0.22; 95% CI 0.10–0.50; p = 0.0003). The annualized flare rate significantly decreased following TCZ use (before TCZ 1.4 [95% CI 1.0–2.1]; after TCZ 0.6 [95% CI 0.3–1.0] events/year; p < 0.001). Similar improvements were observed in patients with visual manifestations or PMR symptoms at GCA diagnosis. TCZ reduced the incidence of new visual manifestations, and no flares associated with permanent vision loss occurred while patients were receiving TCZ. Mean (SD) prednisone dose at TCZ onset and at the end of follow-up was 30 (18.3) and 5 (6.9) mg/day, respectively (p < 0.0001). After TCZ initiation, 46.6% of patients successfully discontinued prednisone. The incidence of adverse events, primarily attributed to glucocorticoids, was similar before and after TCZ initiation. In this real-world setting, TCZ improved GCA clinical outcomes significantly and demonstrated effectiveness in the subgroups of patients with PMR symptoms and GCA-related visual manifestations at GCA diagnosis. No new cases of blindness occurred after TCZ initiation. Adverse events, many attributable to glucocorticoids, were comparable before and after TCZ treatment.
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影响因子：
158.5
作者：
Stone, J. H.;Tuckwell, K.;Collinson, N.
通讯作者：
Collinson, N.
影响因子：
1.6
作者：
Restuccia, Giovanna;Boiardi, Luigi;Salvarani, Carlo
通讯作者：
Salvarani, Carlo
影响因子：
5.5
作者：
Yates, Max;MacGregor, Alex J.;Watts, Richard A.
通讯作者：
Watts, Richard A.
影响因子：
1.6
作者：
Alba MA;García-Martínez A;Prieto-González S;Tavera-Bahillo I;Corbera-Bellalta M;Planas-Rigol E;Espígol-Frigolé G;Butjosa M;Hernández-Rodríguez J;Cid MC
通讯作者：
Cid MC
影响因子：
4.7
作者：
Unizony, S.;Arias-Urdaneta, L.;Stone, J. H.
通讯作者：
Stone, J. H.