维多珠单抗治疗54周的真实世界结局及溃疡性结肠炎停药后的反应持久性：一项多中心前瞻性POLONEZ研究的结果
Real-world outcomes of 54-week vedolizumab therapy and response durability after treatment discontinuation in ulcerative colitis: results from a multicenter prospective POLONEZ study.
作者信息
Eder Piotr, Kłopocka Maria, Cichoż-Lach Halina, Talar-Wojnarowska Renata, Kopertowska-Majchrzak Maria, Michalak Agata, Filip Rafał, Waszak Katarzyna, Stawczyk-Eder Kamila, Janiak Maria, Skrobot Krzysztof, Liebert Ariel, Zatorski Hubert, Solarska-Półchłopek Anna, Krogulecki Michał, Pękała Anna, Poniewierka Elżbieta, Smoła Izabela, Kaczka Aleksandra, Wojciechowski Krzysztof, Drygała Szymon, Zagórowicz Edyta
机构信息
Department of Gastroenterology, Dietetics, and Internal Diseases, Poznan University of Medical Sciences, H. Święcicki University Hospital, Poznań, Poland.
Department of Gastroenterology and Nutritional Disorders, Collegium Medicum in Bydgoszcz, Nicolaus Copernicus University in Toruń, Bydgoszcz, Poland.
出版信息
Therap Adv Gastroenterol. 2023 Feb 14;16:17562848231151295. doi: 10.1177/17562848231151295. eCollection 2023.
BACKGROUND
Vedolizumab is a gut-selective anti-lymphocyte trafficking agent used to treat ulcerative colitis (UC) and Crohn's disease.
OBJECTIVES
We aimed to evaluate the effectiveness, safety, and durability of the therapeutic effect of vedolizumab after treatment discontinuation in a real-world cohort of patients with UC treated in Poland.
DESIGN
This was a multicenter, prospective study involving patients with moderate to severely active UC from 12 centers in Poland who qualified for reimbursed treatment with vedolizumab between February and November 2019.
METHODS
The primary endpoints were clinical response (⩾2-point improvement from baseline on partial Mayo score) and clinical remission (partial Mayo score 0-1), including steroid-free remission, at week 54. Other outcomes included response durability at 26 weeks after treatment discontinuation, identification of predictors of response and remission, and safety assessment.
RESULTS
In all, 100 patients with UC were enrolled (55 biologic naïve and 45 biologic exposed). At baseline, 68% of patients were on corticosteroids and 45% on immunomodulators. Clinical response was observed in 62% of patients, clinical remission in 50%, and steroid-free remission in 42.6% at week 54. Within 26 weeks after treatment discontinuation, 37% of patients who maintained response by week 54 relapsed. The decreased number of liquid stools and rectal bleeding and endoscopic response at week 14 were predictive factors for response at week 54. Time from diagnosis ranging 2-5 years, decreased stool frequency, and non-concomitant use of corticosteroids at baseline and at week 14 were predictive factors for remission at week 54. Partial Mayo score < 3 with no subscale score > 1 at week 54 was a predictive factor for durable response after treatment discontinuation. The rate of serious adverse events related to treatment was 3.63 per 100 patient-years.
CONCLUSION
Vedolizumab is effective and safe in UC treatment in Polish patients. However, the relapse rate after the treatment cessation was high.
REGISTRATION
ENCePP (EUPAS34119).
背景
维多珠单抗是一种肠道选择性抗淋巴细胞转运药物，用于治疗溃疡性结肠炎（UC）和克罗恩病。
目的
我们旨在评估在波兰接受治疗的UC患者真实队列中，停用维多珠单抗治疗后其治疗效果的有效性、安全性和持久性。
设计
这是一项多中心前瞻性研究，纳入了2019年2月至11月间来自波兰12个中心的中度至重度活动性UC患者，这些患者符合维多珠单抗报销治疗条件。
方法
主要终点为第54周时的临床缓解（部分梅奥评分较基线改善⩾2分）和临床缓解（部分梅奥评分0 - 1分），包括无类固醇缓解。其他结局包括停药后26周时缓解的持久性、缓解和缓解预测因素的识别以及安全性评估。
结果
总共纳入了100例UC患者（55例未使用过生物制剂，45例曾使用过生物制剂）。基线时，68%的患者使用皮质类固醇，45%的患者使用免疫调节剂。第54周时，62%的患者观察到临床缓解，50%达到临床缓解，42.6%达到无类固醇缓解。停药后26周内，第54周时维持缓解的患者中有37%复发。第14周时大便次数减少、直肠出血减少和内镜缓解是第54周时缓解的预测因素。诊断时间为2 - 5年、大便频率降低以及基线和第14周时未同时使用皮质类固醇是第54周时缓解的预测因素。第54周时部分梅奥评分<3且各子量表评分均不>1是停药后持久缓解的预测因素。与治疗相关的严重不良事件发生率为每100患者年3.63例。
结论
维多珠单抗在波兰患者的UC治疗中有效且安全。然而，停药后的复发率较高。
注册信息
ENCEPP（EUPAS34119）。
Zotero 插件