Dual Biologic or Small Molecule Therapy in Pediatric Inflammatory Bowel Disease: A Single Center Experience.

IF 2.1 4区医学 Q2 PEDIATRICS Children-Basel Pub Date : 2025-01-09 DOI:10.3390/children12010075

Cheng Guo, Jin Zhou, Guoli Wang, Jie Wu

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Abstract

Purpose: Currently, there is no clinical data reported on the therapy of dual biological agents in pediatric-onset inflammatory bowel disease (PIBD) patients in China. The purpose of this study was to evaluate the efficacy and safety of dual biologic therapy or biologics combined with small molecule drugs in refractory PIBD patients in China.

Methods: Clinical, laboratory, endoscopic, and ultrasound data of PIBD patients from the Department of Gastroenterology of Beijing Children's Hospital between January 2021 and October 2024 were retrospectively analyzed. PIBD patients who received dual biologic treatment or a combination of biologic and small molecule therapy were included in this study. Steroid-free clinical remission and adverse events were recorded.

Results: In this retrospective study, out of 520 children with IBD, twelve children (2.3%) were diagnosed with refractory PIBD and met the criteria for dual biotherapy, including four with UC (33%) and eight with CD (67%). The median age of patients was 13.64 (range, 1.2-17.1) years at eligibility for dual biologic therapy. There are eight (67%) patients treated with infliximab/ustekinumab (IFX + UST), three (25%) patients with upadacitinib/ustekinumab (UPA + UST), one (8%) patient with infliximab/vedolizumab (IFX + VDZ). At 3, 6, and 12 months of dual biological treatment, 91.2% (11/12), 100% (12/12), and 100% (12/12) patients showed steroid-free clinical remission, respectively. The median fecal calprotectin decreased significantly from 1852.5 µg/g (IQR, 762.5-1988.25) at baseline to 359.0 (IQR, 217.5-730.25) μg/g at 3 months, 113 (IQR, 73.7-256) μg/g at 6 months, and 82.5 (IQR, 40.25-122.25) μg/g at 12 months. Only one CD patient with IFX + UST reported mild elevation of aminotransferase, who recovered after symptomatic treatment.

Conclusions: Dual biologic or small molecule therapy may be effective and safe for children with refractory PIBD in China.

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儿童炎症性肠病的双生物或小分子治疗：单中心经验。

目的：目前，国内尚无双生物制剂治疗小儿炎症性肠病（PIBD）的临床资料报道。本研究的目的是评价双重生物疗法或生物制剂联合小分子药物治疗难治性PIBD患者的疗效和安全性。方法：回顾性分析2021年1月至2024年10月北京儿童医院消化内科PIBD患者的临床、实验室、内镜和超声资料。本研究纳入了接受双重生物治疗或生物与小分子联合治疗的PIBD患者。记录无类固醇临床缓解和不良事件。结果：在这项回顾性研究中，520名患有IBD的儿童中，12名儿童（2.3%）被诊断为难治性PIBD并符合双重生物治疗标准，其中4名患有UC(33%)， 8名患有CD（67%）。符合双生物治疗条件的患者中位年龄为13.64岁（范围1.2-17.1岁）。有8例（67%）患者使用英夫利昔单抗/ustekinumab (IFX + UST)， 3例（25%）患者使用upadacitinib/ustekinumab (UPA + UST)， 1例（8%）患者使用英夫利昔单抗/vedolizumab （IFX + VDZ）。在双重生物治疗3、6和12个月时，91.2%（11/12）、100%（12/12）和100%（12/12）的患者分别显示无类固醇临床缓解。粪钙保护蛋白中位数从基线时的1852.5 μg/g （IQR, 762.5-1988.25）显著下降至3个月时的359.0 (IQR, 217.5-730.25) μg/g， 6个月时的113 (IQR, 73.7-256) μg/g， 12个月时的82.5 (IQR, 40.25-122.25) μg/g。仅有1例合并IFX + UST的CD患者报告转氨酶轻度升高，经对症治疗后恢复。结论：在中国，双生物或小分子治疗难治性PIBD患儿是安全有效的。

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来源期刊

Children-Basel PEDIATRICS-

CiteScore

2.70

自引率

16.70%

发文量

1735

审稿时长

6 weeks

期刊介绍： Children is an international, open access journal dedicated to a streamlined, yet scientifically rigorous, dissemination of peer-reviewed science related to childhood health and disease in developed and developing countries. The publication focuses on sharing clinical, epidemiological and translational science relevant to children’s health. Moreover, the primary goals of the publication are to highlight under‑represented pediatric disciplines, to emphasize interdisciplinary research and to disseminate advances in knowledge in global child health. In addition to original research, the journal publishes expert editorials and commentaries, clinical case reports, and insightful communications reflecting the latest developments in pediatric medicine. By publishing meritorious articles as soon as the editorial review process is completed, rather than at predefined intervals, Children also permits rapid open access sharing of new information, allowing us to reach the broadest audience in the most expedient fashion.