Guo Cheng, Zhou Jin, Wang Guoli, Wu Jie
Department of Gastroenterology, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, No. 56 Nanlishi Road, Xicheng District, Beijing 100045, China.
Children (Basel). 2025 Jan 9;12(1):75. doi: 10.3390/children12010075.
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.
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.
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.
Dual biologic or small molecule therapy may be effective and safe for children with refractory PIBD in China.
目前，中国尚无关于儿童起病的炎症性肠病（PIBD）患者使用双重生物制剂治疗的临床数据报道。本研究旨在评估双重生物制剂治疗或生物制剂联合小分子药物治疗中国难治性PIBD患者的疗效和安全性。
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、内镜及超声数据。本研究纳入接受双重生物制剂治疗或生物制剂与小分子药物联合治疗的PIBD患者。记录无类固醇临床缓解情况及不良事件。
在这项回顾性研究中，520例IBD患儿中，12例（2.3%）被诊断为难治性PIBD并符合双重生物治疗标准，其中4例为溃疡性结肠炎（UC，33%），8例为克罗恩病（CD，67%）。双重生物制剂治疗符合条件时患者的中位年龄为13.64（范围1.2 - 17.1）岁。8例（67%）患者接受英夫利昔单抗/优特克单抗（IFX + UST）治疗，3例（25%）患者接受乌帕替尼/优特克单抗（UPA + UST）治疗，1例（8%）患者接受英夫利昔单抗/维多珠单抗（IFX + VDZ）治疗。在双重生物制剂治疗的3个月、6个月和12个月时，分别有91.2%（11/12）、100%（12/12）和100%（12/12）的患者实现无类固醇临床缓解。粪便钙卫蛋白中位数从基线时的1852.5µg/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和12个月时的82.5（IQR，40.25 - 122.25）µg/g。仅1例接受IFX + UST治疗的CD患者报告转氨酶轻度升高，经对症治疗后恢复。
双重生物制剂或小分子药物治疗对中国难治性PIBD儿童可能有效且安全。
