Gastroenterology, Guy's and St Thomas' Hospital, Guy's and St Thomas' NHS Foundation Trust, London, United Kingdom.
Pfizer Inc, New York City, NY.
J Manag Care Spec Pharm. 2024 Sep;30(9):1026-1040. doi: 10.18553/jmcp.2024.30.9.1026.
Effectiveness and safety of advanced therapies for ulcerative colitis (UC) warrant assessment in the real world.
To perform a systematic review and summarize real-world evidence of advanced therapies approved for moderate-to-severe UC.
A systematic literature review was conducted using real-world studies of biologics or small molecules in UC using Embase, MEDLINE, and MEDLINE-In Process databases. Only products approved in any jurisdiction during the search were included. English-language full-papers (January 2005 to February 2022) and congress abstracts (January 2019 to February 2022) were included. Studies with less than 30 patients or only biologic-naive patients were excluded.
A total of 139 studies were included out of 3,930 identified articles (75%, published between 2019 and 2022; 64%, retrospective observational; 53%, from 5 countries [Italy, United States, Spain, United Kingdom, and Belgium]). Most studies were single agent (highest: vedolizumab = 50, tofacitinib = 24, and adalimumab = 18), and rates of clinical remission (CR) and adverse events varied widely. From the published comparative effectiveness studies (16), the rates of CR were numerically higher with vedolizumab vs anti-tumor necrosis factor (TNF)-α agents. Compared with vedolizumab, the effectiveness of tofacitinib was numerically greater in CR (occasionally significant). Rates of steroid-free CR were comparable between ustekinumab and tofacitinib. Infliximab was the most effective anti-TNFα agent, as reported by 2 studies. Remarkably, adverse events were similar across therapies in comparative studies.
Vedolizumab and tofacitinib were the most assessed therapies. In comparative studies, remission rates were numerically higher with tofacitinib vs vedolizumab and for vedolizumab vs anti-TNFα. Tofacitinib was comparable with ustekinumab for steroid-free CR. Safety was comparable across therapies. Future studies should explore the literature gaps identified, including limited comparative studies with small sample sizes, variations in study designs and patient characteristics, varied definitions of CR, and limited use of patient-reported outcome measures in real-world settings.
溃疡性结肠炎（UC）的先进疗法的有效性和安全性需要在真实世界中进行评估。
进行系统综述，总结已批准用于中重度 UC 的先进疗法的真实世界证据。
使用 Embase、MEDLINE 和 MEDLINE-In Process 数据库对生物制剂或小分子治疗 UC 的真实世界研究进行系统文献检索。仅纳入在检索期间获得任何司法管辖区批准的产品。纳入英语全文论文（2005 年 1 月至 2022 年 2 月）和大会摘要（2019 年 1 月至 2022 年 2 月）。排除患者少于 30 例或仅为生物制剂初治患者的研究。
在 3930 篇文章中，共纳入 139 项研究（75%发表于 2019 年至 2022 年期间；64%为回顾性观察研究；53%来自 5 个国家[意大利、美国、西班牙、英国和比利时]）。大多数研究为单药治疗（最高：vedolizumab = 50，tofacitinib = 24，adalimumab = 18），临床缓解（CR）率和不良反应发生率差异很大。在已发表的比较疗效研究（16 项）中，vedolizumab 对比抗肿瘤坏死因子（TNF）-α 药物的 CR 率略高。与 vedolizumab 相比，tofacitinib 在 CR 方面的疗效略高（偶尔有显著差异）。ustekinumab 和 tofacitinib 的无激素 CR 率相当。2 项研究报告称，infliximab 是最有效的抗 TNFα 药物。值得注意的是，在比较研究中，各治疗药物的不良反应相似。
vedolizumab 和 tofacitinib 是评估最多的药物。在比较研究中，tofacitinib 对比 vedolizumab 和 vedolizumab 对比抗 TNFα 药物的 CR 率略高。tofacitinib 与 ustekinumab 相比在无激素 CR 方面相当。各治疗药物的安全性相当。未来研究应探讨已确定的文献空白，包括样本量较小的有限比较研究、研究设计和患者特征的差异、CR 的定义差异以及真实世界环境中患者报告结局测量的使用有限。
