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靶向治疗改善多关节型幼年特发性关节炎的结局研究。

Treat-to-target study for improved outcome in polyarticular juvenile idiopathic arthritis.

机构信息

Department of Pediatrics, Asklepios Kinderklinik Sankt Augustin, Sankt Augustin, Germany

Department of Pediatric and Adolescents Medicine, Medical Faculty, Medical faculty, University Hospital of Cologne, Cologne, Germany.

出版信息

Ann Rheum Dis. 2020 Jul;79(7):969-974. doi: 10.1136/annrheumdis-2019-216843. Epub 2020 Apr 16.

DOI:10.1136/annrheumdis-2019-216843
PMID:32299797
Abstract

BACKGROUND

Juvenile idiopathic arthritis is one of the most prevalent chronic inflammatory diseases in children. Evidence suggests that early effective treatment minimises the burden of disease during childhood and in further life. We hypothesise that a guided treat-to-target (T2T) approach is superior to routine care in polyarticular juvenile idiopathic arthritis (pJIA) in terms of reaching a clinical remission after 12 months of treatment.

METHODS

Patients with early and active pJIA were enrolled. Targets for treatment were the following: Recognisable Juvenile Arthritis Disease Activity Score (JADAS) improvement after 3 months, acceptable disease at 6 months, minimal disease activity at 9 months and as primary endpoint remission after 12 months. Initially, patients received methotrexate. Failure to meet a defined target required treatment modification at the specified intervals. The choice of biologics was not influenced by the protocol. Finally, T2T patients were compared with a cohort of matched controls of patients with pJIA with unguided therapy documented by BIKER.

RESULTS

Sixty-three patients were enrolled. Treatment targets after 3/6/9 and 12 months were reached by 73%/75%/77% and 48% of patients. Fifty-four patients completed the protocol. Compared with matched controls, on T2T guidance significantly more patients reached JADAS remission (48% vs 32%; OR 1.96 (1.1-3.7); p=0.033) and JADAS minimal disease activity (JADAS-MDA) (76% vs 59%; OR 2.2 (1.1-4.4); p=0.028). Patients from the T2T cohort received a biologic significantly more frequent (50% vs 9% after 12 months; OR 9.8 (4.6-20.8); p<0.0001).

CONCLUSION

The T2T concept was feasible and superior to unguided treatment. High rates of patients reached JADAS-MDA and JADA remission after 12 months. Approximately half of the patients achieved their therapy goals without a biologic.

摘要

背景

幼年特发性关节炎是儿童中最常见的慢性炎症性疾病之一。有证据表明,早期有效的治疗可以减轻儿童期和以后生活中的疾病负担。我们假设,在多关节幼年特发性关节炎(pJIA)中,与常规治疗相比,以目标为导向的治疗(T2T)方法在治疗 12 个月后达到临床缓解的效果更好。

方法

招募了早期和活动期 pJIA 患者。治疗目标如下:治疗后 3 个月 JADAS 改善(可识别的幼年关节炎疾病活动评分),6 个月时可接受疾病,9 个月时最小疾病活动度,12 个月时主要终点缓解。最初,患者接受甲氨蝶呤治疗。未达到规定目标的患者需要在指定时间间隔内进行治疗调整。生物制剂的选择不受方案影响。最后,将 T2T 患者与接受 BIKER 记录的无指导治疗的 pJIA 患者的匹配对照组进行比较。

结果

共纳入 63 例患者。73%/75%/77%和 48%的患者在治疗后 3/6/9 和 12 个月时达到了治疗目标。54 例患者完成了方案。与匹配对照组相比,在 T2T 指导下,显著更多的患者达到 JADAS 缓解(48% vs 32%;OR 1.96(1.1-3.7);p=0.033)和 JADAS 最小疾病活动度(JADAS-MDA)(76% vs 59%;OR 2.2(1.1-4.4);p=0.028)。T2T 队列的患者接受生物制剂治疗的频率显著更高(治疗后 12 个月时为 50% vs 9%;OR 9.8(4.6-20.8);p<0.0001)。

结论

T2T 概念是可行的,优于无指导治疗。高比例的患者在 12 个月后达到 JADAS-MDA 和 JADA 缓解。大约一半的患者无需使用生物制剂即可达到治疗目标。

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