背景:由于许多生物药物专利已过期,已经开发了生物类似剂(BIOs);然而,它们的使用仍然有一些保留,尤其是在童年。本研究的目的是评估肿瘤坏死因子(TNF)抑制剂BIOs治疗小儿非感染性葡萄膜炎(NIU)的疗效和安全性。
方法:来自接受TNF抑制剂BIOs治疗的NIU儿科患者的数据来自国际自身炎症性疾病联盟(AIDA)注册中心,专门研究葡萄膜炎和Behçet病。根据复发频率评估BIOs的有效性和安全性,发生眼部耀斑的风险,最佳矫正视力(BCVA),糖皮质激素(GC)-保留作用,药物生存,眼部并发症的频率,和不良药物事件(AE)。
结果:纳入47例患者(77只受影响的眼)。采用的生物标志物是阿达木单抗(ADA)(89.4%),依那西普(ETA)(5.3%),英夫利昔单抗(IFX)(5.3%)。在BIOs之前12个月和最后一次随访时复发的数量为每100名患者/年282.14和52.43。与BIOs开始后相比,BIOs引入前发生眼部耀斑的相对风险为4.49(95%置信区间[CI]3.38-5.98,p=0.004)。眼部耀斑需要治疗的数量(NNT)为3.53。在整个BIOs治疗期间维持了平均BCVA(p=0.92)。在整个治疗期间观察到显著的GCs节约效应(p=0.002)。估计的药物保留率(DRR)为12-,24-,36个月的随访率分别为92.7、83.3和70.8%,分别。在开始BIOs治疗之前,发生结构性眼部并发症的风险率为89.9/100名患者/年,在BIOs治疗期间为12.7/100名患者/年。没有BIOs的新眼部并发症的风险比为7.1(CI3.4-14.9,p=0.0003)。报告了3次轻微的AE。
结论:TNF抑制剂BIOs可有效减少眼葡萄膜炎复发的次数,保持视力,允许显著的节省GC的效果,预防结构性眼部并发症。
背景:ClinicalTrials.govIDNCT05200715。
BACKGROUND: Since many biological drug patents have expired, biosimilar agents (BIOs) have been developed; however, there are still some reservations in their use, especially in childhood. The aim of the current study is to evaluate the efficacy and safety of tumor necrosis factor (TNF) inhibitors BIOs as treatment for pediatric non-infectious uveitis (NIU).
METHODS: Data from pediatric patients with NIU treated with TNF inhibitors BIOs were drawn from the international AutoInflammatory Disease Alliance (AIDA) registries dedicated to uveitis and Behçet\'s disease. The effectiveness and safety of BIOs were assessed in terms of frequency of relapses, risk for developing ocular flares, best-corrected visual acuity (BCVA), glucocorticoids (GCs)-sparing effect, drug survival, frequency of ocular complications, and adverse drug event (AE).
RESULTS: Forty-seven patients (77 affected eyes) were enrolled. The BIOs employed were adalimumab (ADA) (89.4%), etanercept (ETA) (5.3%), and infliximab (IFX) (5.3%). The number of relapses 12 months prior to BIOs and at last follow-up was 282.14 and 52.43 per 100 patients/year. The relative risk of developing ocular flares before BIOs introduction compared to the period following the start of BIOs was 4.49 (95% confidence interval [CI] 3.38-5.98, p = 0.004). The number needed to treat (NNT) for ocular flares was 3.53. Median BCVA was maintained during the whole BIOs treatment (p = 0.92). A significant GCs-sparing effect was observed throughout the treatment period (p = 0.002). The estimated drug retention rate (DRR) at 12-, 24-, and 36-month follow-up were 92.7, 83.3, and 70.8%, respectively. The risk rate for developing structural ocular complications was 89.9/100 patients/year before starting BIOs and 12.7/100 patients/year during BIOs treatment, with a risk ratio of new ocular complications without BIOs of 7.1 (CI 3.4-14.9, p = 0.0003). Three minor AEs were reported.
CONCLUSIONS: TNF inhibitors BIOs are effective in reducing the number of ocular uveitis relapses, preserving visual acuity, allowing a significant GCs-sparing effect, and preventing structural ocular complications.
BACKGROUND: ClinicalTrials.gov ID NCT05200715.