Background

The use of ustekinumab in pediatric patients with inflammatory bowel disease (IBD) is off-label and the data are limited. We conducted a systematic review evaluating the efficacy and safety of ustekinumab in pediatric IBD.

Methods

We systematically searched PubMed, EMBASE and Cochrane databases for studies of ustekinumab in children and adolescents with IBD investigating clinical remission, clinical response, corticosteroid-free (CS-free) remission, endoscopic remission/response, or safety up to March 17, 2023. A random-effects model was used for calculating summary estimates.

Results

Eleven studies, comprising 370 patients were included. For Crohn’s disease (CD), the pooled clinical remission rates were 34% (73/204) at 8–16 weeks and 46% (60/129) at 1 year. The pooled CS-free clinical remission rates were 23% (10/44) at 8–16 weeks and 45% (42/96) at 1 year. For ulcerative colitis (UC)/IBD unspecified (IBD-U), the pooled CS-free clinical remission rates were 24% (6/25) at 26 weeks and 46% (16/35) at 1 year. Endoscopic remission was found in 0–37.5% of CD and 63.6% of UC. Serious adverse events were reported in 3.5% of patients. About one half of patients required reduction in dose intervals and 62.75% patients could continue ustekinumab therapy at 1 year or final visit.

Conclusions

According to low-quality evidence mainly from cohort studies and case series, approximately one half of patients with CD and UC/IBD-U achieved remission at 1 year. Ustekinumab has a reasonable safety profile and dose optimization is frequently required. Data on the long-term benefit and high-quality evidence are still needed.

中文翻译：

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乌司奴单抗治疗小儿炎症性肠病的有效性和安全性：系统评价

背景

乌特克单抗在儿童炎症性肠病 (IBD) 患者中的使用属于超适应证且数据有限。我们进行了一项系统评价，评估乌特克单抗治疗儿童 IBD 的有效性和安全性。

方法

我们系统地检索了 PubMed、EMBASE 和 Cochrane 数据库，以查找截至 2023 年 3 月 17 日的乌特克单抗在 IBD 儿童和青少年中的研究，调查临床缓解、临床反应、无皮质类固醇（无 CS）缓解、内镜缓解/反应或安全性。使用随机效应模型来计算汇总估计。

结果

纳入了 11 项研究，涉及 370 名患者。对于克罗恩病 (CD)，8-16 周时的汇总临床缓解率为 34% (73/204)，1 年时的汇总临床缓解率为 46% (60/129)。8-16 周时汇总的无 CS 临床缓解率为 23% (10/44)，1 年时为 45% (42/96)。对于溃疡性结肠炎 (UC)/未明确的 IBD (IBD-U)，26 周时汇总的无 CS 临床缓解率为 24% (6/25)，1 年时为 46% (16/35)。0-37.5% 的 CD 和 63.6% 的 UC 内镜缓解。3.5% 的患者报告出现严重不良事件。大约一半的患者需要缩短剂量间隔，62.75% 的患者可以在 1 年或最后一次就诊时继续乌特克单抗治疗。

结论

根据主要来自队列研究和病例系列的低质量证据，大约一半的 CD 和 UC/IBD-U 患者在 1 年时获得缓解。乌司奴单抗具有合理的安全性，经常需要优化剂量。仍需要有关长期效益的数据和高质量的证据。