Background Few approved treatment options exist for paediatric patients (pts) with Crohn’s disease (CD). Paediatric inflammatory bowel disease (IBD) medication approvals are delayed nearly 7 years after adult approvals,1 resulting in off-label use and potential for incorrect dosing. The REALITI Study evaluated the effectiveness and safety of ustekinumab (UST) in routine clinical care in paediatric pts (age 2-<18) and young adults (age 18-25) with moderately to severely active CD (Short Paediatric CD activity [sPCDAI] score ≥30) using data from the ImproveCareNow (ICN) Registry. Methods Effectiveness data from pts with CD treated with UST were extracted from the ICN Registry between 10 January 2010 and 29 February 2020. A supplemental, retrospective chart review was conducted to collect data not in the ICN Registry. The primary endpoint was clinical remission (sPCDAI≤10) at Week (Wk) 52. All data were summarized descriptively, and the proportion of pts achieving clinical remission and associated 2-sided 95% confidence interval were calculated across multiple populations. Rates of discontinuations, IBD-related hospitalizations, surgeries, and serious infections were also calculated. Results A total of 479 CD pts in ICN were treated with UST, including 348 paediatric pts and 131 young adults. We report an analysis of 114 paediatric pts who weighed >40 kg and had a sPCDAI ≥30. All pts were treatment refractory; 99.1% had not responded to prior biologic therapies, with less than 1% biologic naïve. Results were compared to 51 ICN young adults with moderately to severely active CD, a population for which UST is approved. Clinical remission at Wk 52 was achieved in 22.8% (26/114; 95% CI: 16.1%, 31.3%) of paediatric pts vs 21.6% (11/51; 95% CI:12.5%, 34.6%) of young adults (Figure 1A). Discontinuation rates through Wk 52 were similar between paediatric pts (25.4%) and young adults (25.5%; Figure 1B). Overall, 36.0% of paediatric pts and 21.6% of young adults had IBD-related hospitalizations. IBD-related surgery was reported in 14.0% of paediatric pts and 11.8% of young adults. Serious infections occurred in 9.6% of paediatric pts and 3.9% of young adults. Opportunistic infections occurred in 1.8% of paediatric pts and 0% of young adults. No events of tuberculosis, malignancy, or anaphylaxis requiring UST discontinuation occurred in either group. No deaths were reported. Conclusion This study of real-world data from the ICN Registry found similar remissions rates in paediatric pts and young adults with CD treated with UST, suggesting comparable effectiveness of UST in both age groups. No new safety signals were identified. (Crowley E, et al. J Crohns Colitis. 2022 Feb 23;16(2):331-335.)

中文翻译：

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P958 REALITI 真实世界证据有效性研究中使用乌司奴单抗治疗中度至重度活动性克罗恩病的儿科患者和年轻人的比较

背景 对于患有克罗恩病 (CD) 的儿科患者 (pts)，批准的治疗方案很少。儿童炎症性肠病 (IBD) 药物的批准比成人批准延迟了近 7 年1，导致超说明书使用和可能的剂量错误。REALITI 研究评估了优特克单抗 (UST) 在患有中度至重度活动性 CD（短儿科 CD 活动 [sPCDAI]）的儿科患者（2-<18 岁）和年轻人（18-25 岁）的常规临床护理中的有效性和安全性] 分数 ≥30），使用 ImproveCareNow (ICN) 注册中心的数据。方法 从 2010 年 1 月 10 日至 2020 年 2 月 29 日期间从 ICN 登记处提取 CD 患者接受 UST 治疗的有效性数据。进行补充性回顾性图表审查以收集 ICN 登记处未包含的数据。主要终点是第 52 周 (Wk) 时的临床缓解 (sPCDAI≤10)。所有数据均进行描述性总结，并计算多个人群中实现临床缓解的患者比例以及相关的 2 侧 95% 置信区间。还计算了停药率、IBD 相关住院率、手术率和严重感染率。结果 ICN 共有 479 名 CD 患者接受了 UST 治疗，其中儿童患者 348 名，青壮年患者 131 名。我们报告了对 114 名体重 > 40 kg 且 sPCDAI ≥ 30 的儿科患者的分析。所有患者均难以治疗；99.1% 的患者对之前的生物疗法没有反应，只有不到 1% 的患者未接受过生物疗法。结果与 51 名患有中度至重度活动性 CD 的 ICN 年轻人进行了比较，UST 已批准用于该人群。第 52 周时，儿科患者的临床缓解率为 22.8% (26/114; 95% CI: 16.1%, 31.3%)，而年轻人的临床缓解率为 21.6% (11/51; 95% CI:12.5%, 34.6%)。图 1A）。直至第 52 周，儿科患者 (25.4%) 和年轻人 (25.5%；图 1B) 的停药率相似。总体而言，36.0% 的儿科患者和 21.6% 的年轻人因 IBD 相关住院治疗。据报道，14.0% 的儿童患者和 11.8% 的年轻人接受了 IBD 相关手术。9.6% 的儿童患者和 3.9% 的年轻人发生严重感染。1.8% 的儿科患者和 0% 的年轻人发生机会性感染。两组均未发生需要停止 UST 的结核病、恶性肿瘤或过敏反应事件。没有死亡报告。结论 这项针对 ICN 注册中心真实世界数据的研究发现，接受 UST 治疗的儿科患者和年轻 CD 患者的缓解率相似，表明 UST 在两个年龄组中的疗效相当。没有发现新的安全信号。（Crowley E 等人 J Crohns Colitis。2022 年 2 月 23 日；16(2):331-335。）