抗肿瘤坏死因子生物制剂在极早发性炎症性肠病中的应用:来自北美的一项多中心回顾性队列研究。

Utilization of Antitumor Necrosis Factor Biologics in Very Early Onset Inflammatory Bowel Disease: A Multicenter Retrospective Cohort Study From North America.

机构信息

From the University of Massachusetts Medical School (UMMS), Worcester, MA.

the The Hospital for Sick Children, University of Toronto, Toronto, Ontario, Canada.

出版信息

J Pediatr Gastroenterol Nutr. 2022 Jul 1;75(1):64-69. doi: 10.1097/MPG.0000000000003464. Epub 2022 May 27.

Abstract

BACKGROUND

Research on the utilization and effectiveness of antitumor necrosis factor (TNF) biologics in children with very early onset inflammatory bowel disease (VEOIBD) is urgently needed. Here we describe anti-TNF use and durability in a multicenter cohort.

METHODS

We performed a retrospective cohort study of patients diagnosed with VEOIBD (<6 years) between 2008 and 2013 at 25 North American centers. We performed chart abstraction at diagnosis and 1, 3, and 5 years after diagnosis. We examined the rate of initiation and durability of infliximab and adalimumab and evaluated associations between treatment durability and the following covariates with multivariate Cox proportional hazard regression: age at diagnosis, sex, disease duration, disease classification, and presence of combined immunomodulatory treatment versus monotherapy.

RESULTS

Of 294 children with VEOIBD, 120 initiated treatment with anti-TNF therapy and 101 had follow-up data recorded [50% Crohn disease (CD), 31% ulcerative colitis (UC), and 19% IBD unclassified (IBD-U)]. The cumulative probability of anti-TNF treatment was 15% at 1 year, 30% at 3 years, and 45% at 5 years from diagnosis; 56 (55%) were treated between 0 and 6 years old. Anti-TNF durability was 90% at 1 year, 75% at 3 years, and 55% at 5 years. The most common reason for discontinuation of anti-TNF were loss of response in 24 (57%) children. Children with UC/IBD-U had lower durability than those with CD (hazard ratio [HR] 0.17; 95% confidence interval [CI], 0.06-0.51; P = 0.001).

CONCLUSIONS

Utilization and durability of anti-TNF in VEOIBD is relatively high and comparable with older children. Having Crohn disease (compared with UC/IBD-U) is associated with greater durability.

摘要

背景

迫切需要研究抗肿瘤坏死因子(TNF)生物制剂在非常早发性炎症性肠病(VEOIBD)患儿中的应用和疗效。在此,我们描述了多中心队列中抗 TNF 的使用和持久性。

方法

我们对 2008 年至 2013 年间在北美 25 个中心诊断为 VEOIBD(<6 岁)的患者进行了回顾性队列研究。我们在诊断时和诊断后 1、3 和 5 年进行了图表提取。我们检查了英夫利昔单抗和阿达木单抗的起始率和持久性,并使用多变量 Cox 比例风险回归评估了治疗持久性与以下协变量之间的关系:诊断时的年龄、性别、疾病持续时间、疾病分类以及联合免疫调节治疗与单药治疗的存在。

结果

在 294 例 VEOIBD 患儿中,120 例患儿接受了抗 TNF 治疗,101 例患儿记录了随访数据[50%克罗恩病(CD)、31%溃疡性结肠炎(UC)和 19%IBD 未分类(IBD-U)]。从诊断开始,抗 TNF 治疗的累积概率为 1 年时为 15%,3 年时为 30%,5 年时为 45%;56 例(55%)患儿在 0 至 6 岁之间接受治疗。抗 TNF 的持久性在 1 年时为 90%,3 年时为 75%,5 年时为 55%。抗 TNF 停药的最常见原因是 24 例(57%)患儿失去反应。UC/IBD-U 患儿的持久性低于 CD 患儿(风险比 [HR]0.17;95%置信区间 [CI],0.06-0.51;P=0.001)。

结论

VEOIBD 中抗 TNF 的应用和持久性相对较高,与年龄较大的儿童相当。患有克罗恩病(与 UC/IBD-U 相比)与更高的持久性相关。

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