Evolving Treatment Patterns and Clinical Outcomes in Children with Newly Diagnosed Crohn's Disease and Ulcerative Colitis in the United States, 2007–2023

Michelle M. Gonzalez,Dhruv Ahuja,Yuchen Qi,Ronghui Xu,Vipul Jairath,Shane W. Goodwin,Ola Olen,William A. Faubion,Michael D. Kappelman,Siddharth Singh
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Abstract

Background & Aims: We examined treatment patterns and clinical outcomes in a contemporary cohort of children with newly diagnosed inflammatory bowel disease (IBD) in a large, commercially insured U.S. population. Methods: Using the OptumLabs® Data Warehouse, we created an inception cohort of physician-diagnosed IBD in children aged 6–17 years between 2007–23. We examined cumulative incidence of hospitalization, surgery, safety events, and medication use in patients with Crohn's disease (CD) and ulcerative colitis (UC), overall and stratified by diagnostic era, race and ethnicity, sex, and age at diagnosis. Results: Among 787 children with newly diagnosed CD (age at diagnosis, 13.4±2.9y; 57% male; 82% White, 5% Black, 7% Hispanic) followed over mean 4.6±3.0y, 5-year risk of IBD-related hospitalization and major surgery was 30% and 10%, respectively. In children diagnosed in 2019–23, 1-year cumulative use of corticosteroids and advanced therapy was 46% and 75%, respectively; advanced therapy use increased significantly over time (aHR, 3.43 [95% CI, 2.71–4.32] for 2019–23 vs. 2007–12), while corticosteroid use declined (0.67 [0.53–0.84]). Risk of IBD-related hospitalization was lower in children diagnosed between 2019–23 (0.65 [0.43–0.97]) compared with those diagnosed between 2007-12; surgical risk did not decline significantly, with confidence intervals compatible with a clinically meaningful reduction. Among 399 children with newly diagnosed UC (age at diagnosis, 14.0±3.0y; 53% male; 78% White, 5% Black, 7% Hispanic) followed over 4.5±3.0y, 5-year risk of IBD-related hospitalization and colectomy was 36% and 8%, respectively. Advanced therapy use increased significantly over time (4.46 [2.85–6.98] for 2019–23 vs. 2007–12), with no significant change in hospitalization or surgical risk across eras. No consistent racial and ethnic, age, or sex differences in advanced therapy use or clinical outcomes were identified in either CD or UC. Conclusions: In a contemporary cohort of commercially insured children with newly diagnosed IBD, early advanced therapy use has increased markedly over time. Surgical risk did not decline significantly in either disease; however, the study was not powered to exclude a clinically meaningful reduction, and these findings are hypothesis-generating rather than conclusive.
2007-2023年美国新诊断的克罗恩病和溃疡性结肠炎儿童的治疗模式和临床结果
背景和目的:我们研究了在美国大量商业保险人群中新诊断为炎症性肠病(IBD)的儿童当代队列的治疗模式和临床结果。方法:使用OptumLabs®数据仓库,我们创建了2007-23年间6-17岁医生诊断的IBD儿童的初始队列。我们检查了克罗恩病(CD)和溃疡性结肠炎(UC)患者的住院、手术、安全事件和药物使用的累积发生率,并按诊断时代、种族和民族、性别和诊断年龄进行了总体和分层。结果:在787例新诊断为CD的儿童中(诊断时年龄13.4±2.9岁,57%为男性,82%为白人,5%为黑人,7%为西班牙裔),随访时间平均为4.6±3.0岁,ibd相关住院和大手术的5年风险分别为30%和10%。在2019-23年诊断的儿童中,1年累计使用皮质类固醇和高级治疗的比例分别为46%和75%;随着时间的推移,高级治疗的使用显著增加(2019-23年与2007-12年的aHR为3.43 [95% CI, 2.71-4.32]),而皮质类固醇的使用下降(0.67[0.53-0.84])。与2007-12年诊断的儿童相比,2019-23年诊断的儿童ibd相关住院的风险较低(0.65 [0.43-0.97]);手术风险没有显著下降,可信区间与临床意义的降低相符。399名新诊断为UC的儿童(诊断时年龄14.0±3.0岁,53%为男性,78%为白人,5%为黑人,7%为西班牙裔)随访超过4.5±3.0岁,ibd相关住院和结肠切除术的5年风险分别为36%和8%。随着时间的推移,高级治疗的使用显著增加(2019-23年与2007-12年相比为4.46[2.85-6.98]),住院或手术风险在不同时期没有显著变化。在CD或UC的高级治疗使用或临床结果中,没有发现一致的种族、民族、年龄或性别差异。结论:在当代新诊断IBD的商业保险儿童队列中,早期晚期治疗的使用随着时间的推移显着增加。两种疾病的手术风险均未显著下降;然而,这项研究并不能排除有临床意义的减少,这些发现是假设产生的,而不是结论性的。
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