儿童炎症性肠病的联合生物治疗：至少 12 个月随访期的安全性和疗效。

Combination biologic therapy in pediatric inflammatory bowel disease: Safety and efficacy over a minimum 12-month follow-up period.

机构信息

Department of Gastroenterology, Hepatology, Feeding Disorders and Pediatrics, The Children's Memorial Health Institute, Warsaw, Poland.

出版信息

J Pediatr Gastroenterol Nutr. 2024 Jul;79(1):54-61. doi: 10.1002/jpn3.12179. Epub 2024 Mar 13.

DOI:10.1002/jpn3.12179

PMID:38477410

Abstract

OBJECTIVES

The severe course of inflammatory bowel diseases (IBDs) refractory to advanced therapies in children results in the search for new therapeutic methods. The aim of this study was to evaluate the efficacy and safety of dual therapy with biologics in a cohort of children with IBD.

METHODS

Retrospective analysis of data from 29 children with a diagnosis of IBD, 19 with ulcerative colitis (66%), 10 with Crohn's disease (CD) (34%) qualified for dual biological therapy (DBT). The median age of patients was five (interquartile range [IQR], 1-15) years at diagnosis of IBD and 14 (IQR, 3-17) years at eligibility for dual therapy. Thirteen (45%) patients were treated with vedolizumab/adalimumab (VDZ + ADA), 13 (45%) with ustekinumab/adalimumab (UST + ADA), three (10%) with infliximab/vedolizumab (IFX + VDZ).

RESULTS

Clinical remission was achieved in 13 (45%; seven UC and six CD) and 12 (41%; seven UC and five CD) Pediatric Weighted Crohn's Disease Activity Index (wPCDAI)/Pediatric Ulcerative Colitis Activity Index (PUCAI) patients after 4 and 12 months at the initiation of dual therapy. Clinical response based on wPCDAI/PUCAI was reported in 16 (55%; nine UC and seven CD) and 12 (41% seven UC and five CD) children after 4 and 12 months of follow-up, respectively. The median fecal calprotectin decreased significantly from 1240 µg/g (53-10,100) to 160 µg/g (5-2500; p = 0.004) between baseline and Month 4 and from 749 at baseline (57-10,100) to 17 (5-3110; p = 0.12) over 12 months. Moreover, 34% (six UC and four CD) of patients achieved endoscopic remission.

CONCLUSIONS

DBT seems to be an effective alternative therapeutic option for patients with moderate and severe IBD.

摘要

目的

儿童中对高级治疗无效的炎症性肠病（IBD）的严重病程导致人们寻求新的治疗方法。本研究的目的是评估生物制剂双重治疗在一组 IBD 儿童中的疗效和安全性。

方法

对 29 名诊断为 IBD 的儿童的数据进行回顾性分析，其中 19 名溃疡性结肠炎（UC）（66%），10 名克罗恩病（CD）（34%）符合双重生物治疗（DBT）的条件。患者的中位年龄为 5 岁（四分位距 [IQR]，1-15 岁），诊断为 IBD，14 岁（IQR，3-17 岁）适合接受双重治疗。13 名（45%）患者接受维得利珠单抗/阿达木单抗（VDZ+ADA）治疗，13 名（45%）患者接受乌司奴单抗/阿达木单抗（UST+ADA）治疗，3 名（10%）患者接受英夫利昔单抗/维得利珠单抗（IFX+VDZ）治疗。

结果

在开始双重治疗后的 4 个月和 12 个月时，13 名（45%；7 名 UC 和 6 名 CD）和 12 名（41%；7 名 UC 和 5 名 CD）患儿达到了临床缓解，根据 wPCDAI/PUCAI 评估。在 4 个月和 12 个月的随访中，分别有 16 名（55%；9 名 UC 和 7 名 CD）和 12 名（41%；7 名 UC 和 5 名 CD）患儿报告了临床反应。粪便钙卫蛋白中位数从基线时的 1240μg/g（53-10100）显著降至 4 个月时的 160μg/g（5-2500；p=0.004），从 749μg/g（57-10100）降至 12 个月时的 17μg/g（5-3110；p=0.12）。此外，34%（6 名 UC 和 4 名 CD）的患者达到了内镜缓解。