Abstract: Objective　To analyze the clinical features，therapeutic evolution，and fatal cases of Still’s disease in children，thereby enhancing pediatricians’ understanding and management of this disease. Methods　This single-center retrospective study analyzed clinical data of 161 patients with Still’s disease admitted to Peking Union Medical College Hospital between January 1，2003 and December 31，2022. Diagnoses were made according to the 2001 criteria proposed by the International League of Associations for Rheumatology（ILAR） Taskforce. Clinical manifestations，treatments，and outcomes were retrospectively analyzed. Results　The median age at onset was 7.6 years，with 84 being boys （52.2%）. Still’s disease accounted for 3.3% of all pediatric rheumatology inpatients under 16 years and 14.3% of children hospitalized for fever of unknown origin during the same period. Common manifestations included fever，rash，lymphadenopathy，and hepatomegaly and/or splenomegaly. Around 88.2% of patients had joint involvement，mainly affecting the knees，ankles，and wrists. The past treatment regimens included glucocorticoids（GC） alone，GC combined with methotrexate（MTX），GC combined with cyclosporine（CsA），and tumor necrosis factor（TNF） inhibitors. In the past decade，new regimens with tocilizumab，interleukin-1 inhibitors have been introduced. Using time to treatment（IL-1） inhibitors，and Janus kinase（JAK） failure as the outcome indicator，tocilizumab showed significantly better efficacy than GC+MTX（HR=0.43，95% CI=0.22-0.85），while GC alone was significantly inferior to GC+MTX（HR=1.89，95% CI=1.17-3.08）. Two female patients died during hospitalization due to systemic infections. Conclusion　The therapeutic strategies for Still’s disease have evolved substantially over the past 20 years. In the last decade，IL-6-targeted therapies have markedly improved patient outcomes，though clinicians should remain vigilant for infection risks associated with high-dose glucocorticoid pulse therapy.

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摘要： 目的　分析Still病的临床特点、治疗变迁及死亡病例情况，以提高临床对该病的认识和诊治水平。方法　单中心回顾性研究，总结2003年1月1日至2022年12月31日于北京协和医院收治的161例Still病患儿临床资料，以2001年国际风湿病学联盟（ILAR）儿科专家组制订的标准进行诊断，并对其临床表现、治疗、预后进行回顾性分析。结果　患儿中位发病年龄为7.6岁，其中男性84例（52.2%）；Still病患儿占同期16岁以下儿童风湿免疫住院患儿的3.3%，占同期发热原因待查患儿的14.3%。临床表现为发热、皮疹、淋巴结大、肝大和（或）脾大等非特异性表现，关节受累率为88.2%，主要累及膝、踝和腕等关节；既往治疗方案包括单纯糖皮质激素（GC）、GC 联合甲氨蝶呤（MTX）、GC 联合环孢素（CsA）、肿瘤坏死因子（TNF）抑制剂，近10年开始采用托珠单抗、白介素 -1（IL-1）抑制剂、Janus激酶（JAK）抑制剂的新方案。以发生治疗失败的时间为结局指标，托珠单抗治疗显著优于GC联合MTX组（HR=0.43，95% CI=0.22~0.85），单纯GC治疗显著劣于GC联合MTX组（HR=1.89，95% CI=1.17~3.08）。2例女性患儿发生在院死亡，死因均为全身性感染。结论　Still病治疗方案在20年内发生变迁，近10年以IL-6为主的靶向治疗方案显著改善了患儿预后，仍需警惕大剂量激素冲击相关的感染风险。

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