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阿达木单抗治疗儿童肠道白塞病的疗效

Therapeutic effect of adalimumab in the treatment of intestinal Behçet's disease in children.

作者信息

Gao Tianjiao, Ren Xiaoxia, Han Yanan, Wang Fengfan, Liu Bianhua, Fang Ying

机构信息

Department of Gastroenterology, Xi'an Children's Hospital, Xi'an, China.

出版信息

Front Pediatr. 2025 Jun 30;13:1619065. doi: 10.3389/fped.2025.1619065. eCollection 2025.

Abstract

OBJECTIVE

Intestinal Behçet's disease is a rare but severe manifestation of Behçet's disease, particularly in pediatric populations. Currently, there is no cure for intestinal Behçet's disease, and the goal of treatment is to control acute episodes and reduce inflammation. Adalimumab, a tumor necrosis factor-alpha (TNF-α) inhibitor, has shown promise in adult patients, but data on its efficacy and safety in children are limited. The aim of this study was to evaluate the efficacy and safety of adalimumab in the treatment of intestinal Behçet's disease in children.

METHODS

A retrospective analysis was conducted on 8 children with intestinal Behçet's disease treated with adalimumab at Xi'an Children's Hospital from January 2021 to December 2024. Clinical symptoms, endoscopic findings, and inflammatory markers were assessed before and after treatment. Efficacy was evaluated on the basis of symptom resolution, ulcer healing, and changes in inflammatory markers.

RESULTS

The cohort included 4 males and 4 females, with an average age of 11.9 ± 1.4 years and a disease course of 10.5 (6.75, 22.5) months. All patients presented with gastrointestinal symptoms, including abdominal pain ( = 7), vomiting ( = 1), and complications such as intestinal obstruction ( = 3) and perforation ( = 1). Colonoscopy revealed ileocecal ulcers in 7 patients and only terminal ileal ulcers in 1 patient. After treatment with adalimumab, complete resolution of clinical symptoms was observed in all patients. Gastrointestinal endoscopy revealed that 6 patients had completely healed ulcers, while the remaining 2 patients had ulcers that were reduced to 50% of their original size. The levels of inflammatory markers significantly decreased, with the erythrocyte sedimentation rate (ESR) decreasing from 44.63 ± 43.48 mm/h to 10.50 ± 7.65 mm/h ( = 0.046) and the high sensitivity C-reactive protein (hs-CRP) level decreasing from 43.87 ± 39.10 mg/L to 0.96 ± 0.67 mg/L ( = 0.017). On the basis of clinical symptoms and endoscopic findings, 6 patients (75%) achieved complete remission, and 2 patients (25%) showed improvement. Adalimumab was well tolerated, with only one case of mild eczema reported.

CONCLUSION

Adalimumab is safe and effective in the treatment of intestinal Behçet's disease in children.

摘要

目的

肠道白塞病是白塞病一种罕见但严重的表现形式,在儿童群体中尤为如此。目前,肠道白塞病无法治愈,治疗目标是控制急性发作并减轻炎症。阿达木单抗是一种肿瘤坏死因子-α(TNF-α)抑制剂,在成年患者中已显示出前景,但关于其在儿童中的疗效和安全性的数据有限。本研究的目的是评估阿达木单抗治疗儿童肠道白塞病的疗效和安全性。

方法

对2021年1月至2024年12月在西安市儿童医院接受阿达木单抗治疗的8例肠道白塞病患儿进行回顾性分析。在治疗前后评估临床症状、内镜检查结果和炎症标志物。根据症状缓解、溃疡愈合和炎症标志物变化评估疗效。

结果

该队列包括4名男性和4名女性,平均年龄为11.9±1.4岁,病程为10.5(6.75,22.5)个月。所有患者均出现胃肠道症状,包括腹痛(n = 7)、呕吐(n = 1)以及肠梗阻(n = 3)和穿孔(n = 1)等并发症。结肠镜检查显示,7例患者存在回盲部溃疡,1例患者仅存在末端回肠溃疡。使用阿达木单抗治疗后,所有患者的临床症状均完全缓解。胃肠道内镜检查显示,6例患者的溃疡完全愈合,其余2例患者的溃疡缩小至原来大小的50%。炎症标志物水平显著下降,红细胞沉降率(ESR)从44.63±43.48 mm/h降至10.50±7.65 mm/h(P = 0.046),高敏C反应蛋白(hs-CRP)水平从43.87±39.10 mg/L降至0.96±0.67 mg/L(P = 0.017)。根据临床症状和内镜检查结果,6例患者(75%)实现完全缓解,2例患者(25%)有所改善。阿达木单抗耐受性良好,仅报告1例轻度湿疹病例。

结论

阿达木单抗治疗儿童肠道白塞病安全有效。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/6da2/12256472/1b7c0ba196aa/fped-13-1619065-g001.jpg

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