人源化白细胞介素-6受体抗体治疗全身型幼年特发性关节炎的疗效及安全性

Efficacy and safety of humanized interleukin-6 receptor antibody in treatment of systemic juvenile idiopathic arthritis

目的:探讨人源化IL-6受体抗体(托珠单抗注射液)治疗全身型幼年特发性关节炎(sJIA)的临床疗效及安全性。方法:回顾性分析2015年12月—2016年11月在浙江大学医学院附属儿童医院应用托珠单抗治疗的13例sJIA患儿的临床资料,包括血常规、C反应蛋白(CRP)、红细胞沉降率(ESR)、IL-6、血清铁蛋白、美国风湿病学儿科(ACR Pedi)30/50/70/90评分、激素使用情况以及治疗期间的不良反应。结果:与治疗前比较,治疗后第3天患儿的CRP和ESR明显下降(均P<0.05);治疗后第2周血红蛋白增加和血小板减少(均P<0.05);第4周血清铁蛋白水平下降(P<0.05);白细胞在治疗后第8周时减少(P<0.05)。IL-6水平在治疗后先上升,第4周时下降,但与治疗前比较差异均无统计学意义(均P>0.05)。治疗第4周ACR Pedi 30或以上达100%;第20周时,61.5%的患儿达到ACR Pedi 90和停用糖皮质激素。随访至20周,所有患儿共发生不良反应22例次,其中感染发生率占54.5%(12/22),无严重不良反应发生。结论:托珠单抗能快速控制sJIA炎症,改善疾病活动度,有助于糖皮质激素的顺利减量及停药,且安全有效。

Objective： To evaluate the efficacy and safety of humanized anti-IL-6 receptor monoclonal antibody （tocilizumab） in treatment of systemic juvenile idiopathic arthritis （sJIA）. Methods： Thirteen sJIA patients admitted between December 2015 and November 2016 and received tocilizumab treatment were enrolled in the study. The complete blood count （CBC） , C-reactive protein （ CRP）, erythrocyte sedimentation rate （ESR）, interleukin-6 （IL-6） and ferritin levels were measured; American College of Rheumatology Pediatric （ACR Pedi）30/50/70/90 scores were assessed; and the use of glucocorticosteroid and adverse events were documented. Results： Compared with the baseline levels, the CRP and ESR at d3 were decreased （all P 〈 0. 05） ; hemoglobin was increased and platelet was decreased at week 2 （ all P 〈 0.05 ）, ferritin decreased at week 4, white blood cell （WBC） decreased at week 8 after treatment with tocilizumab （ all P 〈 0. 05 ）. The level of IL-6 was rising at d3 and week 2 and descending at week 4, but no significant difference was observed compared with the baseline level （ all P 〉 0.05 ）. All 13 patients achieved ACR Pedi 30 remission at week 4, 61.5% achieved ACR Pedi 90 remission and glucocorticosteroids were withdrawn at week 20. Twenty two adverse events occurred, and infection accounted for 54.5% （12/22）; no severe adverse reactions were observed during 20-week follow-up. Conclusion： Tocilizumab is safe and effective in treatment of sJIA, with decreasing inflammation, improving disease activity and reducing glucocorticosteroid use.