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儿童炎症性肠病非标签疗法现状综述

A Review of the Current State of Off-Label Therapies for Pediatric Inflammatory Bowel Disease.

作者信息

El-Najjar Yassin, Touma Mary-Joe, Tan Shuai, Zhou Xiaoqin, Liu Qi

机构信息

Department of Internal Medicine University of Texas Southwestern Medical Center Dallas Texas USA.

Department of Gastroenterology Children's Hospital of Chongqing Medical University National Clinical Research Center for Child Health and Disorders Ministry of Education Key Laboratory of Child Development and Disorders China International Science and Technology Cooperation Base of Child Development and Critical Disorders Chongqing Key Laboratory of Child Rare Diseases in Infection and Immunity Chongqing China.

出版信息

Pediatr Discov. 2025 Jun 17;3(2):e70011. doi: 10.1002/pdi3.70011. eCollection 2025 Jun.

Abstract

Off-label use of biologic therapies in patients with pediatric inflammatory bowel disease (IBD) has seen an increase in utilization. In this paper, we review the current state of off-label therapies in the pediatric IBD population. Real-world use of ustekinumab (UST), vedolizumab (VDZ), upadacitinib (UPA), tofacitinib, and ozanimod in the adult population could prove positive outcomes in the pediatric population. Established off-label therapies inch closer to comparable safety, efficacy, and outcomes in pediatric IBD use. Outcomes and use of newer biologic therapies in patients with pediatric IBD have improved with increased rates of steroid-free clinical remission (SFCR). Novel therapies, including Janus kinase (JAK) inhibitors and sphingosine-1-phosphate receptor (S1Pr) modulators, require further studies but could also prove effective.

摘要

生物疗法在小儿炎症性肠病(IBD)患者中的非标签使用有所增加。在本文中,我们回顾了小儿IBD人群中未按标签规定用药疗法的现状。乌司奴单抗(UST)、维多珠单抗(VDZ)、乌帕替尼(UPA)、托法替布和奥扎莫德在成人中的实际应用可能在小儿人群中产生积极结果。已确立的未按标签规定用药疗法在小儿IBD使用中更接近可比的安全性、有效性和结果。随着无类固醇临床缓解(SFCR)率的提高,小儿IBD患者使用新型生物疗法的结果和情况有所改善。包括 Janus 激酶(JAK)抑制剂和 1-磷酸鞘氨醇受体(S1Pr)调节剂在内的新型疗法需要进一步研究,但也可能证明是有效的。

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