Division of Immunology, Boston Children's Hospital and Harvard Medical School, 300 Longwood Ave, Fegan 6 Boston, Boston, MA, 02115, USA.
Amgen Inc, Thousand Oaks, CA, USA.
Pediatr Rheumatol Online J. 2024 Oct 7;22(1):88. doi: 10.1186/s12969-024-01022-x.
Chronic anterior uveitis (CAU) carries a significant risk for eye complications and vision loss. The Childhood Arthritis and Rheumatology Research Alliance (CARRA) introduced consensus treatment plans (CTPs) to standardize treatment for CAU and facilitate future comparative effectiveness studies. Two CTPs were developed to address: 1) initiation of methotrexate (MTX) in patients with CAU naïve to steroid-sparing therapy, and 2) initiation of a TNF inhibitor (TNFi) in patients with severe uveitis or uveitis refractory to MTX. We evaluated implementation of the uveitis CTPs using existing CARRA Registry infrastructure and assessed feasibility of the CTPs for comparative effectiveness research.
This prospective observational cohort study was conducted at nine pilot sites between February 2020 and August 2022. Patients with JIA-associated CAU (JIA-U) were treated according to either the MTX or TNFi CTP. Uveitis activity and medication use were recorded at 0, 3, and 6 months. We assessed patient enrollment rates, CTP arm selection, uveitis control, and quality of data collection. We also evaluated CTP arm selection in a retrospective cohort of similar JIA-U patients enrolled in the CARRA Registry during the same study period.
Seventeen patients were included in the pilot cohort. Eight were treated with the MTX CTP (4 oral MTX, 4 subcutaneous MTX), and 9 with the TNFi CTP (9 received standard-dose adalimumab, none selected high-dose adalimumab or infliximab). Uveitis was controlled in 13 of 17 patients by 6 months. Query of the CARRA-wide Registry identified 42 patients with JIA-U who were treated according to the MTX or TNFi CTPs. Among these, 26 were treated with MTX (8 oral, 18 subcutaneous) and 16 with TNFi (12 standard dose adalimumab, 2 high dose adalimumab, and 2 infliximab).
Both the MTX and TNFi uveitis CTPs can practically be implemented in clinical settings and are currently being utilized across Registry sites. However, in patients starting TNFi therapy, all pilot study participants and most patients across the CARRA Registry were treated with a standard dose of adalimumab. This consensus on the treatment approach underscores its broad acceptance but also limits the applicability of the uveitis TNFi CTP for comparative effectiveness research.
慢性前葡萄膜炎（CAU）存在发生眼部并发症和视力丧失的重大风险。儿童关节炎和风湿病研究联盟（CARRA）引入了共识治疗方案（CTP），以规范 CAU 的治疗，并促进未来的比较效果研究。制定了两种 CTP 来解决：1）对接受类固醇保存治疗的 CAU 患者开始使用甲氨蝶呤（MTX），以及 2）对严重葡萄膜炎或 MTX 难治性葡萄膜炎患者开始使用 TNF 抑制剂（TNFi）。我们使用现有的 CARRA 注册中心基础设施评估了葡萄膜炎 CTP 的实施情况，并评估了 CTP 用于比较效果研究的可行性。
这是一项在 2020 年 2 月至 2022 年 8 月期间在 9 个试点站点进行的前瞻性观察队列研究。患有 JIA 相关 CAU（JIA-U）的患者根据 MTX 或 TNFi CTP 进行治疗。在 0、3 和 6 个月时记录葡萄膜炎活动和药物使用情况。我们评估了患者入组率、CTP 臂选择、葡萄膜炎控制和数据收集质量。我们还在同一研究期间在 CARRA 注册中心招募的类似 JIA-U 患者的回顾性队列中评估了 CTP 臂选择。
17 名患者纳入试点队列。8 名患者接受 MTX CTP（4 名口服 MTX，4 名皮下 MTX）治疗，9 名患者接受 TNFi CTP（9 名接受标准剂量阿达木单抗，无患者选择高剂量阿达木单抗或英夫利昔单抗）治疗。到 6 个月时，17 名患者中有 13 名葡萄膜炎得到控制。在 CARRA 范围内的注册表中查询到 42 名患有 JIA-U 的患者，他们根据 MTX 或 TNFi CTP 进行治疗。其中，26 名患者接受 MTX 治疗（8 名口服，18 名皮下），16 名患者接受 TNFi 治疗（12 名标准剂量阿达木单抗，2 名高剂量阿达木单抗，2 名英夫利昔单抗）。
MTX 和 TNFi 葡萄膜炎 CTP 均可在临床环境中实际实施，并且目前正在注册中心站点使用。然而，在开始 TNFi 治疗的患者中，所有试点研究参与者和 CARRA 注册表中的大多数患者均接受标准剂量的阿达木单抗治疗。对治疗方法的共识强调了其广泛接受，但也限制了葡萄膜炎 TNFi CTP 用于比较效果研究的适用性。
