Treatment endpoints in ulcerative colitis: Does one size fit all?

Nikola Mitrev, V. Kariyawasam
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Abstract

A treat-to-target strategy in inflammatory bowel disease (IBD) involves treatment intensification in order to achieve a pre-determined endpoint. Such uniform and tight disease control has been demonstrated to improve clinical outcomes compared to treatment driven by a clinician’s subjective assessment of symptoms. However, choice of treatment endpoints remains a challenge in management of IBD via a treat-to-target strategy. The treatment endpoints for ulcerative colitis (UC), recommended by the Selecting Therapeutic Targets in Inflammatory Bowel Disease (STRIDE) consensus have changed somewhat over time. The latest STRIDE-II consensus advises immediate (clinical response), intermediate (clinical remission and biochemical normalisation) and long-term treatment (endoscopic healing, absence of disability and normalisation of health-related quality of life, as well as normal growth in children) endpoints in UC. However, achieving deeper levels of remission, such as histologic normalisation or healing of the gut barrier function, may further improve outcomes among UC patients. Generally, all medical therapy should seek to improve short- and long-term mortality and morbidity. Hence treatment endpoints should be chosen based on their ability to predict for improvement in short- and long-term mortality and morbidity. Potential benefits of treatment intensification need to be weighed against the potential harms within an individual patient. In addition, changing therapy that has achieved partial response may lead to worse outcomes, with failure to recapture response on treatment reversion. Patients may also place different emphasis on certain potential benefits and harms of various treatments than clinicians, or may have strong opinions re certain therapies. Potential benefits and harms of therapies, incremental benefits of achieving deeper levels of remission, as well as uncertainties of the same, need to be discussed with individual patients, and a treatment endpoint agreed upon with the clinician. Future research should focus on quantifying the incremental benefits and risks of achieving deeper levels of remission, such that clinicians and patients can make an informed decision about appropriate treatment end-point on an individual basis.
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溃疡性结肠炎的治疗终点:是否 "一刀切"?
炎症性肠病(IBD)的 "靶向治疗 "策略包括加强治疗,以达到预先确定的终点。事实证明,与根据临床医生对症状的主观评估进行治疗相比,这种统一而严格的疾病控制能改善临床疗效。然而,治疗终点的选择仍然是通过 "靶向治疗 "策略管理 IBD 的一项挑战。随着时间的推移,炎症性肠病治疗目标选择(STRIDE)共识推荐的溃疡性结肠炎(UC)治疗终点也发生了一些变化。最新的 STRIDE-II 共识建议 UC 的近期(临床反应)、中期(临床缓解和生化指标正常化)和长期治疗(内镜下痊愈、无残疾、健康相关生活质量正常化以及儿童发育正常)终点。然而,实现更深层次的缓解,如组织学正常化或肠道屏障功能愈合,可能会进一步改善 UC 患者的治疗效果。一般来说,所有药物治疗都应力求改善短期和长期死亡率和发病率。因此,应根据预测短期和长期死亡率和发病率改善情况的能力来选择治疗终点。需要权衡加强治疗的潜在益处和对个体患者的潜在危害。此外,改变已取得部分应答的治疗方法可能会导致更糟糕的结果,因为治疗恢复后无法重新获得应答。患者对各种疗法的某些潜在益处和危害的重视程度可能与临床医生不同,或对某些疗法有强烈的意见。治疗的潜在益处和危害、实现更深层次缓解的增量益处以及不确定性都需要与患者进行讨论,并与临床医生商定治疗终点。未来的研究应侧重于量化实现更深层次缓解的增量效益和风险,以便临床医生和患者能够根据个体情况就适当的治疗终点做出明智的决定。
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Treatment endpoints in ulcerative colitis: Does one size fit all? New paradigm of oral rehydration in patients affected by irritable bowel syndrome with chronic diarrhea
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