Kimura Yukiko, Pinho Paulo, Walco Gary, Higgins Gloria, Hummell Donna, Szer Ilona, Henrickson Michael, Watcher Sandra, Reiff Andreas
Joseph M. Sanzari Children's Hospital at Hackensack University Medical Center, Hackensack, New Jersey 07601, USA.
J Rheumatol. 2005 May;32(5):935-42.
. To assess the efficacy and safety of etanercept in a large cohort of children with refractory systemic onset juvenile rheumatoid arthritis (SOJRA).
Standardized questionnaires were sent to US pediatric rheumatologists about patients with SOJRA treated with etanercept. Data were collected at baseline and at the last visit on etanercept. Response to treatment was assessed and compared to baseline as the mean percentage reduction in the following: acute phase reactants, prednisone dose, active joint count, and physician global assessment of disease activity. Response was defined as poor if the mean reduction was < 30%, fair if 30% to < 50%, good if 50% to < 70%, and excellent if > 70%.
We analyzed data obtained by survey of 82 SOJRA patients treated with etanercept for a mean of 25 months. Poor response to treatment was observed in 45% of the children, fair response in 9%, good in 13%, and excellent in 33%. Baseline steroid therapy could be discontinued in 27/59 (46%) patients. One or more disease flares occurred in 45% of all patients. Twenty-nine patients (35%) discontinued therapy, mostly due to lack of response or flare. There were 32 adverse event reports, most not considered serious, except for 2 cases of macrophage activation syndrome.
In this cohort of children with SOJRA, 46% had a good or excellent response, and most were able to reduce concomitant corticosteroid doses. The response to etanercept was fair or poor in more than half our study population, and disease flares were common. Due to the unique cytokine profile of SOJRA, tumor necrosis factor blockade may not be the optimal therapeutic approach for children with treatment-resistant SOJRA.
评估依那西普在一大群难治性全身型幼年类风湿关节炎（SOJRA）患儿中的疗效和安全性。
向美国儿科风湿病学家发送标准化问卷，询问接受依那西普治疗的SOJRA患者情况。在基线期和依那西普治疗的最后一次随访时收集数据。评估治疗反应，并与基线期比较，以下列指标的平均降低百分比表示：急性期反应物、泼尼松剂量、活动关节数以及医生对疾病活动的整体评估。若平均降低幅度<30%，则反应为差；若为30%至<50%，则为中等；若为50%至<70%，则为良好；若>70%，则为优秀。
我们分析了对82例接受依那西普治疗平均25个月的SOJRA患者进行调查获得的数据。45%的患儿治疗反应差，9%为中等反应，13%为良好反应，33%为优秀反应。59例患者中有27例（46%）可停用基线期的类固醇治疗。45%的患者发生了一次或多次疾病复发。29例患者（35%）停止治疗，主要原因是无反应或复发。有32例不良事件报告，大多数不被认为严重，除了2例巨噬细胞活化综合征。
在这组SOJRA患儿中，46%有良好或优秀反应，且大多数能够减少同时使用的皮质类固醇剂量。在我们的研究人群中，超过一半对依那西普的反应为中等或差，且疾病复发很常见。由于SOJRA独特的细胞因子谱，肿瘤坏死因子阻断可能不是治疗抵抗性SOJRA患儿的最佳治疗方法。
