利桑珠单抗诱导和维持治疗难治性克罗恩病的疗效:批准前准入计划和早期获得药物计划的实际经验

IF 2.4 Q3 GASTROENTEROLOGY & HEPATOLOGY
Benjamin Zare, Beatriz Gros, Natasha Lal, Patrick Dawson, Esha Sharma, Robin J Dart, Samuel Lim, Shuvra Ray, Simon H C Anderson, Joel Mawdsley, Peter M Irving, Charlie W Lees, Mark A Samaan
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Clinical response was observed in 33% (n=14/42), 45% (n=17/38) and 52% (n=13/25), and clinical remission in 31% (n=13/42), 40% (n=15/38) and 44% (n=11/25) at weeks 12, 28 and 52, respectively. Median C reactive protein decreased from 12 mg/L (IQR: 4–30; n=50) at baseline to 6 mg/L (IQR: 2–16; p=0.03 vs baseline; n=49) at week 12, 3 mg/L (IQR: 2–8, p=0.003; n=44) at week 28 and 3 mg/L (IQR 1–4, p=0.007; n=31) at week 52. Median faecal calprotectin concentration was 668 µg/g (IQR: 246–1098; n=32) at baseline, 298 µg/g (IQR: 176–546, p=NS; n=21) at week 12, 358 µg/g (IQR: 133–622, p=0.03; n=14) at week 28 and 63 µg/g (IQR: 38–120, p=0.007; n=12) at week 52. 12 out of 18 patients discontinued corticosteroids at week 12, 16 by week 28 and 18 by week 52. Four major adverse events—three elective and one emergency surgery—were recorded. Conclusion Risankizumab is effective in a refractory real-world population with CD. 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引用次数: 0

摘要

目的 自从利桑珠单抗被批准用于克罗恩病(CD)以来,该药已得到广泛应用。然而,真实世界的数据却很有限,我们的目的就是要填补这一空白。设计/方法 我们在英国的两个中心对 2021 年 1 月至 2023 年 1 月期间开始接受治疗的 CD 患者使用利坦珠单抗的情况进行了回顾性观察研究。在基线、第 4 周、第 12 周、第 28 周和第 52 周测量了临床活动、生化和粪便生物标志物。主要结果是第12、28和52周时的临床反应。结果 53 名患者(51% 为女性);年龄中位数(范围)为 40 岁(20-70 岁);病程中位数为 15 年(6-52 年)。在第12周、第28周和第52周,分别有33%(n=14/42)、45%(n=17/38)和52%(n=13/25)的患者出现临床应答,31%(n=13/42)、40%(n=15/38)和44%(n=11/25)的患者出现临床缓解。C反应蛋白中位数从基线时的12毫克/升(IQR:4-30;n=50)下降到第12周时的6毫克/升(IQR:2-16;与基线相比,p=0.03;n=49)、第28周时的3毫克/升(IQR:2-8,p=0.003;n=44)和第52周时的3毫克/升(IQR 1-4,p=0.007;n=31)。基线时粪便钙蛋白浓度中位数为 668 微克/克(IQR:246-1098;n=32),第 12 周时为 298 微克/克(IQR:176-546,p=NS;n=21),第 28 周时为 358 微克/克(IQR:133-622,p=0.03;n=14),第 52 周时为 63 微克/克(IQR:38-120,p=0.007;n=12)。18 名患者中有 12 人在第 12 周停用皮质类固醇,16 人在第 28 周停用,18 人在第 52 周停用。共记录到四次重大不良事件--三次择期手术和一次急诊手术。结论 利桑珠单抗对现实世界中的CD难治性患者有效。如有合理要求,可提供相关数据。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Effectiveness of risankizumab induction and maintenance therapy for refractory Crohn’s disease: a real-world experience from a preapproval access programme and early access to medicines scheme
Objective Since approval in Crohn’s disease (CD) of risankizumab, there has been widespread use. Real-world data are, however, limited and our aim is to address that gap. Design/method We performed a retrospective, observational study of risankizumab use in patients with CD starting treatment between January 2021 and January 2023 at two UK centres. Clinical activity, biochemical and faecal biomarkers were measured at baseline, weeks 4, 12, 28 and 52. The primary outcome was clinical response at weeks 12, 28 and 52. Results 53 patients (51% women); median (range) age 40 years (20–70); median disease duration 15 years (6–52). Clinical response was observed in 33% (n=14/42), 45% (n=17/38) and 52% (n=13/25), and clinical remission in 31% (n=13/42), 40% (n=15/38) and 44% (n=11/25) at weeks 12, 28 and 52, respectively. Median C reactive protein decreased from 12 mg/L (IQR: 4–30; n=50) at baseline to 6 mg/L (IQR: 2–16; p=0.03 vs baseline; n=49) at week 12, 3 mg/L (IQR: 2–8, p=0.003; n=44) at week 28 and 3 mg/L (IQR 1–4, p=0.007; n=31) at week 52. Median faecal calprotectin concentration was 668 µg/g (IQR: 246–1098; n=32) at baseline, 298 µg/g (IQR: 176–546, p=NS; n=21) at week 12, 358 µg/g (IQR: 133–622, p=0.03; n=14) at week 28 and 63 µg/g (IQR: 38–120, p=0.007; n=12) at week 52. 12 out of 18 patients discontinued corticosteroids at week 12, 16 by week 28 and 18 by week 52. Four major adverse events—three elective and one emergency surgery—were recorded. Conclusion Risankizumab is effective in a refractory real-world population with CD. Data are available on reasonable request.
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来源期刊
Frontline Gastroenterology
Frontline Gastroenterology GASTROENTEROLOGY & HEPATOLOGY-
CiteScore
4.70
自引率
11.50%
发文量
93
期刊介绍: Frontline Gastroenterology publishes articles that accelerate adoption of innovative and best practice in the fields of gastroenterology and hepatology. Frontline Gastroenterology is especially interested in articles on multidisciplinary research and care, focusing on both retrospective assessments of novel models of care as well as putative future directions of best practice. Specifically Frontline Gastroenterology publishes articles in the domains of clinical quality, patient experience, service provision and medical education.
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