Clinical outcome of ulcerative colitis with severe onset in children: a multicenter prospective cohort study.
Clinical outcome of ulcerative colitis with severe onset in children: a multicenter prospective cohort study.
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儿童严重发作的溃疡性结肠炎的临床结果：一项多中心前瞻性队列研究。
DOI：
10.1007/s00535-023-01972-1
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发表时间：
2023
期刊：
影响因子：
--
通讯作者：
Shimiz
中科院分区：
文献类型：
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作者：
Nambu R;Arai K;Kudo T;Murakoshi T;Kunisaki R;Mizuochi T;Kato S;Kumagai H;Inoue M;Ishige T;Saito T;Noguchi A;Yodoshi T;Hagiwara SI;Iwata N;Nishimata S;Kakuta F;Tajiri H;Hiejima E;Toita N;Mochizuki T;Shimizu H;Iwama I;Hirano Y;Shimiz
背景由于在生物制剂时代治疗儿童严重溃疡性结肠炎的最佳实践仍存在争议，我们前瞻性地研究了多中心队列的治疗和结局。我们比较了S1组（诊断时儿童溃疡性结肠炎活动指数≥ 65分）和S 0组（诊断时儿童溃疡性结肠炎活动指数≥ 65分）的管理和治疗结果，结果共纳入21个单位的301例溃疡性结肠炎患儿，随访3.6 ± 1.9年。其中，75例（25.0%）为S1;诊断时年龄为12.3 ± 2.9岁，93%患有全结肠炎。S1组1年后的结肠切除率为89%，2年后为79%，5年后为74%，显著低于S 0组（P= 0.0003）。钙调磷酸酶抑制剂和生物制剂分别用于53%和56%的S1患者，显著高于S 0患者（P< 0.0001）。在S1患者治疗钙调神经磷酸酶抑制剂时，类固醇失败，23%既不需要生物制剂，也不结肠切除术，类似于S 0组（P= 0.46）。ConclusionsChildren with severe ulcerative colitis are likely to require powerful agents such as calcineurin inhibitors and biological agents;有时结肠切除术最终证明是必要的。通过介入CI的治疗试验，而不是立即转向生物制剂或结肠切除术，可能会在一定程度上减少类固醇耐药患者对生物制剂的需求。
BackgroundAs best practices for treating children with severe-onset ulcerative colitis remain controversial in the era of biologic agents, we prospectively investigated treatments and outcomes in a multicenter cohort.MethodsUsing a Web-based data registry maintained in Japan between October 2012 and March 2020, we compared management and treatment outcomes in an S1 group defined by a Pediatric Ulcerative Colitis Activity Index of 65 or more points at diagnosis with those in an S0 group defined by an index value below 65.ResultsThree hundred one children with ulcerative colitis treated at 21 institutions were included, with follow-up for 3.6 ± 1.9 years. Among them, 75 (25.0%) were in S1; their age at diagnosis was 12.3 ± 2.9 years, and 93% had pancolitis. Colectomy free rates in S1 were 89% after 1 year, 79% after 2, and 74% after 5, significantly lower than for S0 (P= 0.0003). Calcineurin inhibitors and biologic agents, respectively, were given to 53% and 56% of S1 patients, significantly more than for S0 patients (P< 0.0001). Among S1 patients treated with calcineurin inhibitors when steroids failed, 23% required neither biologic agents nor colectomy, similarly to the S0 group (P= 0.46).ConclusionsChildren with severe ulcerative colitis are likely to require powerful agents such as calcineurin inhibitors and biologic agents; sometimes colectomy ultimately proves necessary. Need for biologic agents in steroid-resistant patients might be reduced to an extent by interposing a therapeutic trial of CI rather than turning to biologic agents or colectomy immediately.