Wang Mingyang, Qin Bixuan, Liu Cuihong, Liu Honglei, Li Dongmei
Beijing Tongren Eye Center, Beijing Tongren Hospital, Capital Medical University, Beijing 100730, China.
Shaanxi Eye Hospital, Xi'an People's Hospital (Xi'an Fourth Hospital), Affiliated People's Hospital of Northwest University, Xi'an 710199, China.
J Endocr Soc. 2024 Nov 2;8(12):bvae193. doi: 10.1210/jendso/bvae193. eCollection 2024 Oct 29.
The efficacy of tocilizumab (TCZ) in treating Graves orbitopathy (GO) remains uncertain due to the small sample sizes of earlier studies, and there is a lack of research on the drug for juvenile GO.
To evaluate the effectiveness of TCZ in treating GO that is resistant to conventional therapy.
This observational study at a tertiary care center included 79 Chinese GO patients, 15 of whom were pediatric patients, with 52 of these patients having moderate to severe active GO (all adult patients having steroid-resistant GO). Intravenous infusion of TCZ 8 mg/kg was given every 28 days for 4 months. Changes from baseline in visual acuity (VA), intraocular pressure (IOP), proptosis, clinical activity score (CAS), and thyrotropin receptor antibody (TRAb) levels throughout TCZ therapy were assessed at baseline (T0), the fifth month (T4), and follow-up (T5). Additionally, improvements in CAS by at least 2 points and CAS < 4 points at T4 and T5 were evaluated.
Significant improvements were found in VA, IOP, proptosis, CAS, and TRAb levels in the adult group, and proptosis in the pediatric group at T5 ( < .05). Additionally, significant improvements were identified in TRAb levels and CAS (active GO at T0) in the pediatric group at T4 ( < .05). In the adult and pediatric group with active GO at T5, 71.4% and 60% experienced a decrease in CAS by ≥ 2 points, respectively; 89.3% and 60% achieved the response criterion of low activity disease (CAS < 4 points), respectively.
TCZ emerged as a valuable therapeutic option for Chinese patients with active, corticosteroid-resistant, moderate to severe GO.
由于早期研究样本量较小，托珠单抗（TCZ）治疗格雷夫斯眼眶病（GO）的疗效仍不确定，且缺乏针对青少年GO患者使用该药物的研究。
评估TCZ治疗对传统治疗耐药的GO的有效性。
这项在三级医疗中心进行的观察性研究纳入了79例中国GO患者，其中15例为儿科患者，这些患者中有52例患有中度至重度活动性GO（所有成年患者均为激素抵抗性GO）。每28天静脉输注8mg/kg的TCZ，持续4个月。在基线（T0）、第五个月（T4）和随访（T5）时评估整个TCZ治疗过程中视力（VA）、眼压（IOP）、眼球突出度、临床活动评分（CAS）和促甲状腺素受体抗体（TRAb）水平相对于基线的变化。此外，还评估了T4和T5时CAS至少改善2分以及CAS<4分的情况。
在T5时，成年组的VA、IOP、眼球突出度、CAS和TRAb水平以及儿科组的眼球突出度有显著改善（P<0.05）。此外，在T4时，儿科组的TRAb水平和CAS（T0时为活动性GO）有显著改善（P<0.05）。在T5时患有活动性GO的成年组和儿科组中，分别有71.4%和60%的患者CAS降低≥2分；分别有89.3%和60%的患者达到低活动度疾病的反应标准（CAS<4分）。
对于患有活动性、皮质类固醇抵抗性、中度至重度GO的中国患者，TCZ是一种有价值的治疗选择。
