Jairath Vipul, Narula Neeraj, Ungaro Ryan C, Romo Bautista Itzel, Adsul Shashi
Department of Medicine, Division of Gastroenterology, Western University, London, ON, Canada.
Alimentiv Inc., London, ON, Canada.
J Crohns Colitis. 2025 Apr 4;19(4). doi: 10.1093/ecco-jcc/jjaf040.
Inflammatory bowel diseases (IBD), Crohn's disease (CD), and ulcerative colitis (UC) are lifelong chronic, relapsing, and remitting conditions that culminate in disease progression in many patients. Effective management of CD and UC requires consideration of both short- and long-term treatment outcomes. Historically, short-term outcomes such as clinical and endoscopic remission and symptom relief have been evaluated in clinical trials. With the expansion of treatments targeting underlying disease pathophysiology, there is the opportunity to develop management strategies that improve disease control and patients' lives in both the short and the long term. Researchers have been examining novel outcomes for assessing the efficacy of CD and UC treatments that are important to patients, and also those that go beyond symptomatic improvements or clinical remission. These include new patient-reported outcomes for symptoms, as well as transmural/histological healing and disease clearance that can be more reflective of deeper remission states and disease modification. This review analyses published clinical studies involving patients with UC and CD treated with biologics or small molecule therapies. It highlights novel IBD endpoints employed in published clinical trials and discusses their likely value for assessing disease activity and disease modification, and as predictors of reduced risk of complications and morbidities.
炎症性肠病(IBD)、克罗恩病(CD)和溃疡性结肠炎(UC)是终身慢性、复发性和缓解性疾病,许多患者最终会出现疾病进展。有效管理CD和UC需要考虑短期和长期治疗结果。从历史上看,临床试验中评估了短期结果,如临床和内镜缓解以及症状缓解。随着针对潜在疾病病理生理学的治疗方法不断扩展,有机会制定能在短期和长期内改善疾病控制和患者生活的管理策略。研究人员一直在研究新的结果指标,以评估对患者重要的CD和UC治疗的疗效,以及那些超越症状改善或临床缓解的指标。这些指标包括新的患者报告的症状结果,以及透壁/组织学愈合和疾病清除,这些指标能更准确地反映更深层次的缓解状态和疾病改善情况。本综述分析了已发表的涉及使用生物制剂或小分子疗法治疗的UC和CD患者的临床研究。它强调了已发表临床试验中采用的新型IBD终点指标,并讨论了它们在评估疾病活动和疾病改善方面的潜在价值,以及作为降低并发症和发病率风险预测指标的价值。