背景:溃疡性结肠炎(UC)是一种慢性疾病,对健康相关生活质量(HRQoL)有相当大的负面影响,最近被认为是一个重要的治疗目标。这项研究的目的是比较不同生物制剂和小分子疗法在UC患者中获得更好的患者报告结果和HRQoL的疗效。
方法:我们对EMBASE进行了系统评价和网络荟萃分析,MEDLINE,和CochraneCentral数据库从成立到2024年2月1日。主要终点是在诱导和维持阶段接受不同生物制剂或小分子治疗的UC患者的患者报告结果(PRO-2)评分的临床缓解。PRO-2评分是大便次数和直肠出血子评分的总和。次要结果是HRQoL改善,定义为炎症性肠病问卷评分从基线增加≥16分或总分从基线的任何变化。使用了随机效应模型,结果报告为比值比,95%置信区间.根据SUCRA(累积排名曲线下的表面)得分对干预进行排名。
结果:共有54项研究纳入主要结果分析,15项研究纳入次要结果分析。主要分析表明,在诱导阶段,所有纳入的药物在改善PRO-2评分方面均优于安慰剂。有趣的是,研究发现,upadacitinib在提高PRO-2评分方面优于大多数药物.二次分析显示,guselkumab在炎症性肠病问卷评分的改善中排名第一,然后是诱导期的upadacitinib.
结论:Upadacitinib在诱导期和维持期的PRO-2临床缓解中排名第一。Guselkumab,mirikizumab,托法替尼,和upadacitinib是唯一一种在改善UC患者HRQoL方面优于安慰剂的新型药物,guselkumab排名最高,其次是托法替尼和upadacitinib.在维持缓解期间,托法替尼在改善HRQoL方面排名最高。
患者报告的结果(PRO-2)和疾病对健康相关生活质量(HRQoL)的影响已被认为是溃疡性结肠炎的重要治疗目标。在这篇系统综述和网络荟萃分析中,我们比较了不同的生物制剂和小分子在实现这些结果方面的作用.我们发现,在诱导和维持阶段,upadacitinib在PRO-2临床缓解中排名第一。Guselkumab,托法替尼,和upadacitinib是唯一一种在溃疡性结肠炎诱导期间改善HRQoL优于安慰剂的新型药物,guselkumab排名最高,其次是托法替尼和upadacitinib.在维持缓解期间,托法替尼在改善HRQoL方面排名最高。
BACKGROUND: Ulcerative colitis (UC) is a chronic disorder with a considerable negative impact on health-related quality of life (HRQoL), which has been recently recognized as an important treatment target. The purpose of this study is to compare the efficacy of different biologics and small molecule therapies in achieving better patient-reported outcomes and HRQoL in patients with UC.
METHODS: We performed a systematic review and network meta-analysis of the EMBASE, MEDLINE, and Cochrane Central databases from inception until February 1, 2024. The primary endpoint was clinical remission in the patient-reported outcome (PRO-2) score in UC patients who were treated with different biologics or small molecules during induction and maintenance phases. PRO-2 score is the sum of both stool frequency and rectal bleeding subscores. The secondary outcome was improvement of HRQoL defined as an increase in Inflammatory Bowel Disease Questionnaire score of ≥16 points from baseline or any change in total score from baseline. A random effects model was used, and outcomes were reported as odds ratio with 95% confidence interval. Interventions were ranked per the SUCRA (surface under the cumulative ranking curve) score.
RESULTS: A total of 54 studies were included in the primary outcome analysis and 15 studies were included in the secondary outcome analysis. The primary analysis showed that during the induction phase all of included drugs were better than placebo in improving the PRO-2 score. Interestingly, upadacitinib was found to be superior to most medications in improving PRO-2 scores. The secondary analysis showed that guselkumab ranked first in the improvement of the Inflammatory Bowel Disease Questionnaire score, followed by upadacitinib during the induction phase.
CONCLUSIONS: Upadacitinib ranked first in PRO-2 clinical remission during the induction and maintenance phases. Guselkumab, mirikizumab, tofacitinib, and upadacitinib were the only novel medications that were superior to placebo in improving HRQoL in UC, with guselkumab ranking the highest, followed by tofacitinib and upadacitinib. During maintenance of remission, tofacitinib ranked highest in improving HRQoL.
Patient-reported outcome (PRO-2) and disease impact on health-related quality of life (HRQoL) have been recognized as important treatment targets in ulcerative colitis. In this systematic review and network meta-analysis, we compared different biologics and small molecules in achieving these outcomes. We found that upadacitinib ranked first in PRO-2 clinical remission during induction and maintenance phases. Guselkumab, tofacitinib, and upadacitinib were the only novel medications that were superior to placebo in improving HRQoL during induction in ulcerative colitis, with guselkumab ranking the highest, followed by tofacitinib and upadacitinib. During maintenance of remission, tofacitinib ranked highest in improving HRQoL.