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小儿溃疡性直肠炎的长期病程及预后因素:一项多中心队列研究

Long-Term Course and Prognostic Factors in Pediatric Ulcerative Proctitis: A Multicenter Cohort Study.

作者信息

Miyazawa Ayako, Nambu Ryusuke, Shimizu Hirotaka, Kudo Takahiro, Nishizawa Takuya, Kumagai Hideki, Hagiwara Shin-Ichiro, Kaji Emiri, Mizuochi Tatsuki, Kurasawa Shingo, Kakuta Fumihiko, Ishige Takashi, Shimizu Toshiaki, Iwama Itaru, Arai Katsuhiro

机构信息

Division of Gastroenterology and Hepatology, Saitama Children's Medical Center, 1-2, Shintoshin, Chuo-ku, Saitama City, Saitama 330-8777, Japan.

Center for Pediatric Inflammatory Bowel Disease, Division of Gastroenterology, National Center for Child Health and Development, 2-10-1, Okura, Setagaya-City, Tokyo 157-8535, Japan.

出版信息

Inflamm Bowel Dis. 2025 Jul 7;31(7):1902-1909. doi: 10.1093/ibd/izae266.

Abstract

BACKGROUND

Although ulcerative proctitis (UP) in children is considered relatively mild, some patients have proximal disease extension and require immunosuppressive treatment. We investigated clinical characteristics and course of refractory UP in a multicenter pediatric cohort.

METHODS

Analyzing data obtained between 2013 and 2022 at 10 institutions specializing in pediatric inflammatory bowel disease, we elucidated natural history and factors predicting a need for immunosuppressive UP treatment. We compared patients given immunosuppressants and/or biologic agents (immunosuppressive treatment group) with those given 5-aminosalicylic acid (5-ASA) alone (5-ASA group).

RESULTS

Fifty-five patients were followed for 3.5 years. The median Pediatric Ulcerative Colitis Activity Index at diagnosis was 20. The commonest treatment, 5-ASA suppository monotherapy in 40% of patients, showed the worst compliance. Clinical remission was achieved at least once in 95% of all patients. Disease extension beyond the splenic flexure occurred in 51%. Immunosuppressive treatment was given to 37%; biologic agents were used for 18%. Rates of endoscopically demonstrated inflammation, including Ra/Rs at diagnosis and extension beyond the left-sided colon, were higher in the immunosuppressive treatment group (70% vs 38%, P < 0.05; 95% vs 27%, P < 0.0001). The log-rank test and multivariate Cox proportional hazards regression showed that time to first clinical remission exceeding 3 months predicted the need for biologics.

CONCLUSION

The typical initial treatment of pediatric UP was 5-ASA suppositories, despite poor compliance. Biologics or other immunosuppressive treatments were needed in 37% of patients. Close follow-up with adjustment of treatment should be considered in children with UP as its clinical course varies.

摘要

背景

尽管儿童溃疡性直肠炎(UP)被认为相对较轻,但一些患者会出现近端疾病扩展,需要免疫抑制治疗。我们在一个多中心儿科队列中研究了难治性UP的临床特征和病程。

方法

分析2013年至2022年期间在10家儿科炎症性肠病专科医院获得的数据,我们阐明了自然病程以及预测UP免疫抑制治疗需求的因素。我们将接受免疫抑制剂和/或生物制剂治疗的患者(免疫抑制治疗组)与仅接受5-氨基水杨酸(5-ASA)治疗的患者(5-ASA组)进行了比较。

结果

55例患者随访了3.5年。诊断时的儿童溃疡性结肠炎活动指数中位数为20。最常见的治疗方法是40%的患者采用5-ASA栓剂单药治疗,其依从性最差。95%的患者至少实现了一次临床缓解。脾曲以外的疾病扩展发生率为51%。37%的患者接受了免疫抑制治疗;18%的患者使用了生物制剂。免疫抑制治疗组内镜显示的炎症发生率更高,包括诊断时的Ra/Rs以及左侧结肠以外的扩展(70%对38%,P<0.05;95%对27%,P<0.0001)。对数秩检验和多变量Cox比例风险回归显示,首次临床缓解时间超过3个月可预测需要使用生物制剂。

结论

尽管依从性差,儿童UP的典型初始治疗方法仍是5-ASA栓剂。37%的患者需要生物制剂或其他免疫抑制治疗。由于UP的临床病程各异,对于患有UP的儿童应考虑密切随访并调整治疗。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/d1aa/12235130/753f9a05c4b2/izae266_fig12.jpg

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