Romero Roberta Krause, Magro Daniela Oliveira, Queiroz Natalia Sousa Freitas, Damião Aderson Omar Mourão Cintra, Teixeira Fabio Vieira, Nones Rodrigo Bremer, Sassaki Ligia Yukie, Saad-Hossne Rogerio, Kotze Paulo Gustavo
Pontifícia Universidade Católica do Paraná (PUCPR), Curitiba, Brazil.
Universidade Estadual de Campinas (UNICAMP), Campinas, Brazil.
Gastroenterol Hepatol. 2022 Aug-Sep;45(7):499-506. doi: 10.1016/j.gastrohep.2021.09.005. Epub 2021 Oct 8.
In the last decade, new therapies with different mechanisms of action have been approved for the treatment of moderate to severe Crohn's disease (CD) and ulcerative colitis (UC). Due to the lack of comparative head-to-head trials, the ideal positioning of agents as the most appropriate first- or second-line therapies remains to be defined.
This survey aimed to evaluate the perception and decisions of Brazilian Inflammatory Bowel Diseases (IBD) specialists in positioning of new therapies (vedolizumab [VEDO], ustekinumab [UST] and tofacitinib [TOFA]) in the management of IBD in different clinical scenarios.
An anonymous national web-based questionnaire was used to determine the positioning of treatment options in different clinical scenarios (using Google Forms platform), which involved different age ranges, phenotypes, clinical situations and previous exposure to anti-TNF agents (14 scenarios for CD and 10 scenarios for UC). In CD, physicians could choose between UST or VEDO, whilst in UC, between UST, VEDO or TOFA. Six reasons for the specific choice were proposed, such as mechanism of action, safety, method of administration or onset of action. Statistical analysis was carried out with chi-square and t-tests.
A total of 150 out of 672 GEDIIB IBD specialists (22.32%) responded to the survey. In CD scenarios, UST was the most dominant choice (11/14 scenarios), with VEDO dominating only 3 clinical situations. In UC scenarios, VEDO was the dominant choice (8/10), with UST being chosen for scenarios that included extraintestinal manifestations. Among the reasons for specific choices, the most commonly chosen were the higher efficacy due to the intrinsic mechanism of action and safety profile.
UST was the dominant choice as compared to VEDO in CD in most scenarios, especially due to its mechanism of action and safety. VEDO was the dominant choice as compared to UST and TOFA in UC scenarios, mainly for reasons also related to its mechanism of action and safety profile. Comparative studies including patient outcomes are needed to better define the positioning of new IBD therapeutic options in our country.
在过去十年中，已批准了具有不同作用机制的新疗法用于治疗中度至重度克罗恩病（CD）和溃疡性结肠炎（UC）。由于缺乏对比的直接头对头试验，作为最合适的一线或二线疗法的药物的理想定位仍有待确定。
本调查旨在评估巴西炎症性肠病（IBD）专家在不同临床场景中IBD管理中对新疗法（维多珠单抗[VEDO]、乌司奴单抗[UST]和托法替布[TOFA]）定位的看法和决策。
使用基于网络的匿名全国性问卷来确定不同临床场景（使用谷歌表单平台）中治疗方案的定位，这些场景涉及不同年龄范围、表型、临床情况以及既往抗TNF药物暴露情况（CD有14种场景，UC有10种场景）。在CD中，医生可在UST或VEDO之间选择，而在UC中，可在UST、VEDO或TOFA之间选择。提出了特定选择的六个原因，如作用机制、安全性、给药方法或起效时间。采用卡方检验和t检验进行统计分析。
672名GEDIIB IBD专家中共有150名（22.32%）回复了调查。在CD场景中，UST是最主要的选择（14种场景中的11种），VEDO仅在3种临床情况中占主导。在UC场景中，VEDO是主导选择（10种中的8种），对于包括肠外表现的场景则选择UST。在特定选择的原因中，最常选择的是由于内在作用机制和安全性概况而具有更高疗效。
在大多数CD场景中，与VEDO相比，UST是主要选择，特别是由于其作用机制和安全性。在UC场景中，与UST和TOFA相比，VEDO是主要选择，主要原因也与其作用机制和安全性概况有关。需要开展包括患者预后的对比研究，以更好地确定我国IBD新治疗方案的定位。
