Tocilizumab monotherapy for polymyalgia rheumatica: A prospective, single-center, open-label study
Tocilizumab monotherapy for polymyalgia rheumatica: A prospective, single-center, open-label study
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DOI：
10.1111/1756-185x.13723
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发表时间：
2019-10-17
影响因子：
2.5
通讯作者：
Amano, Koichi
中科院分区：
文献类型：
--
作者：
Chino, Kentaro;Kondo, Tsuneo;Amano, Koichi
目的风湿性多肌痛（PMR）是一种病因不明的老年全身性炎症性疾病。虽然糖皮质激素是PMR治疗的主要药物，但各种糖皮质激素相关不良事件也很常见。最近，几项研究报道了托珠单抗（TCZ）（一种抗白细胞介素-6受体抗体）治疗PMR的疗效，此外还伴随糖皮质激素的减少，甚至在某些情况下逐渐减少。本研究的目的是阐明TCZ单药治疗在不使用糖皮质激素的情况下对PMR的疗效。方法我们进行了一项为期2年的前瞻性、单中心、开放标签的初步研究，在PMR患者中进行TCZ单药治疗。TCZ（8 mg/kg）在前2个月每两周给药一次，在接下来的10个月每月给药一次。随后，患者又被观察了一年，没有任何治疗。主要终点是第12周和第52周的缓解率，次要终点是104周内的复发率和安全性。结果13例患者纳入本研究。其中4例患者在第12周达到缓解（缓解率31%）。4例患者因不良事件（n = 2）和无效（n = 2）退出研究。在第52周，所有9名完成第一年治疗的患者均获得缓解。8例患者完成了第二年的无药治疗，7例维持缓解。结论TCZ单药治疗耐受性良好，在无糖皮质激素的情况下，大多数PMR患者可获得缓解。
Objectives Polymyalgia rheumatica (PMR) is a systemic inflammatory disease in the elderly of unknown etiology. While glucocorticoids are the mainstay of treatment for PMR, various glucocorticoid-related adverse events are common. Recently, several studies have reported the efficacy of tocilizumab (TCZ), an anti-interleukin-6 receptor antibody, for PMR treatment in addition to an accompanying reduction, or even tapering-off, of glucocorticoids in some cases. The objective of this study was to elucidate the efficacy of TCZ monotherapy in the absence of glucocorticoids for PMR. Method We conducted a 2-year, prospective, single-center, open-label pilot study of TCZ monotherapy in patients with PMR. TCZ (8 mg/kg) was administered at fortnightly intervals for the first 2 months and monthly over the next 10 months. Subsequently, patients were observed for another year without any treatment. The primary endpoints were the remission rates at weeks 12 and 52, and the secondary endpoints were the relapse rate and safety over the total 104 weeks. Results Thirteen patients were included in this study. Four of these patients achieved remission at week 12 (remission rate 31%). Four patients withdrew from the study due to adverse events (n = 2) and inefficacy (n = 2). At week 52, all 9 patients who had completed the first year achieved remission. Eight patients completed the drug-free second year, with 7 maintaining remission. Conclusions TCZ monotherapy is well tolerated and can lead to remission in most patients with PMR in the absence of glucocorticoids.