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