Roszkiewicz Justyna, Orczyk Krzysztof, Smolewska Elżbieta
Department of Paediatric Cardiology and Rheumatology, Medical University of Łódź, Poland.
Reumatologia. 2018;56(5):279-284. doi: 10.5114/reum.2018.79497. Epub 2018 Oct 31.
The aim of the study was to evaluate the efficacy and long-term safety of tocilizumab treatment in children with systemic-onset juvenile idiopathic arthritis in a single centre.
The study was based on a retrospective analysis of a cohort of 10 patients with systemic-onset juvenile idiopathic arthritis who were treated with tocilizumab in the period September 2011-July 2017. Their medical records were analysed taking into consideration the effectiveness of tocilizumab treatment and frequency of side effects.
Before the initiation of treatment, 9/10 patients from the study group complained of fever and had significantly increased values of inflammatory markers, with the median CRP concentration 41.1 mg/l (norm < 5 mg/l) and ESR 37 mm/h (norm < 12 mg/l). The period of the initial 12 weeks of treatment was a quantum leap in the course of the disease: all children were afebrile, and inflammatory markers values decreased by 99.4% in the case of CRP and 91.9% in ESR. All patients fulfilled ACR Pedi 50 criteria, and 3 of them achieved ACR Pedi 70. In the next stages of treatment the response to tocilizumab was sustained, reaching 10 children achieving ACR Pedi 70 and 5 ACR Pedi 90 after one year of therapy. Tocilizumab appeared to be relatively safe in the study group. Although elevation of transaminases and neutropenia were observed in 5/10 patients, they were usually mild and transitional in their course.
Tocilizumab is both effective and has a relatively good safety profile in children with severe systemic-onset juvenile idiopathic arthritis. It should be considered in the recommendations as a first-line treatment of this disease.
本研究旨在评估在单中心使用托珠单抗治疗全身型幼年特发性关节炎患儿的疗效及长期安全性。
本研究基于对2011年9月至2017年7月期间接受托珠单抗治疗的10例全身型幼年特发性关节炎患儿队列的回顾性分析。分析他们的病历,考量托珠单抗治疗的有效性及副作用发生频率。
治疗开始前,研究组10例患者中有9例主诉发热,炎症标志物值显著升高,CRP浓度中位数为41.1mg/l(正常<5mg/l),ESR为37mm/h(正常<12mg/l)。治疗最初12周是疾病进程中的一个巨大飞跃:所有患儿均无发热,CRP炎症标志物值下降99.4%,ESR下降91.9%。所有患者均达到美国风湿病学会儿科50标准,其中3例达到美国风湿病学会儿科70标准。在治疗的后续阶段,对托珠单抗的反应持续存在,治疗1年后10例患儿达到美国风湿病学会儿科70标准,5例达到美国风湿病学会儿科90标准。在研究组中托珠单抗似乎相对安全。尽管10例患者中有5例观察到转氨酶升高和中性粒细胞减少,但通常程度较轻且病程呈一过性。
托珠单抗治疗重度全身型幼年特发性关节炎患儿既有效且安全性相对良好。在治疗推荐中应考虑将其作为该病的一线治疗药物。