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在短肠综合征和慢性肠衰竭患者中使用胰高血糖素样肽 2 类似物的真实世界经验:来自专家肠衰竭中心的国际调查结果。

Real-world experience with glucagon-like peptide 2 analogues in patients with short bowel syndrome and chronic intestinal failure: Results from an international survey in expert intestinal failure centers.

机构信息

Leuven Intestinal Failure and Transplantation (LIFT), Division of Gastroenterology and Hepatology, Leuven University Hospitals, Leuven, Belgium; Translational Research Center for Gastrointestinal Disorders (TARGID), Department of Chronic Diseases and Metabolism (ChroMetA), KULeuven, Leuven, Belgium.

Division of Clinical Nutrition, Department of Medicine, Faculty of Medicine, Chulalongkorn University, King Chulalongkorn Memorial Hospital, Bangkok 10330, Thailand.

出版信息

Clin Nutr ESPEN. 2024 Dec;64:496-502. doi: 10.1016/j.clnesp.2024.10.161. Epub 2024 Nov 1.

Abstract

BACKGROUND AND AIMS

Glucagon-like peptide 2 (GLP-2) analogues are the first available disease-modifying treatments for patients with intestinal failure (IF) due to short bowel syndrome (SBS). Efficacy in terms of reduction of parenteral support (PS) has been demonstrated in multiple studies and real-world reports. However, it remains unclear how many patients are eligible to receive the treatment, when treatment is started after intestinal resection, how treatment efficacy is assessed outside of clinical trials, and how the treatment is modified in case of non-response or adverse events. The aim of this study was to investigate the real-world management of patients treated with GLP-2 analogues in expert centers around the world.

METHODS

A survey questionnaire was developed by a multidisciplinary working group consisting of 52 questions related to various aspects of multidisciplinary care of SBS-IF patients. The 17 questions related to the use of GLP-2 analogues in clinical practice were analyzed for this study. The online survey was sent to 33 participating centers in a phase 3 study of a long-acting GLP-2 analogue. Only responses from countries with access to commercially available GLP-2 analogues were included in the study. A descriptive analysis was performed for each question. Results are presented as median (interquartile range).

RESULTS

The responses from the 19 expert IF centers with access to GLP-2 analogues indicated that 10 (10-20) % of patients with SBS-IF were treated with a GLP-2 analogue, which was less than the number of eligible patients (30 (25-40) %). In most centers (10 centers, 53 %), GLP-2 therapy was started 6-12 months after the last intestinal resection, with 5 centers (26 %) starting later (12-24 months). Multiple parameters were used in combination to determine the response to GLP-2 analogues of which the three most common were >20 % decrease in PS (95 %), at least 1 day of PS reduction per week (84 %) and increased urinary output (68 %). In non-responders GLP-2 therapy was stopped within the first year by 67 % of the centers. Finally, strategies in case of significant adverse events include stopping the GLP-2 analogue (used by 79 % of experts), dose reduction (67 %) and temporary treatment interruption (62 %).

CONCLUSION

The results of this survey completed by expert IF centers show the real-life use of GLP-2 analogues in clinical practice. Key learning points identified include the accounting for a period of intestinal adaptation before starting GLP-2 analogues and not stopping the treatment too early in case of non-response. The best strategy in case of adverse effects should be studied further.

摘要

背景与目的

胰高血糖素样肽 2(GLP-2)类似物是首个可用于治疗短肠综合征(SBS)相关肠衰竭(IF)患者的疾病修正治疗药物。多项研究和真实世界报告均证实了其在减少肠外支持(PS)方面的疗效。然而,目前尚不清楚有多少患者符合治疗条件,以及在肠切除术后何时开始治疗,如何在临床试验之外评估治疗效果,以及在出现无应答或不良反应时如何调整治疗。本研究旨在调查全球专家中心中接受 GLP-2 类似物治疗的患者的真实世界管理情况。

方法

由一个由 52 名来自多学科团队的成员组成的多学科工作组制定了一份调查问卷,其中包含了 52 个与 SBS-IF 患者多学科护理相关的问题。本研究分析了与临床实践中 GLP-2 类似物使用相关的 17 个问题。该在线调查发送给了参与长效 GLP-2 类似物 3 期研究的 33 个参与中心。本研究仅纳入了有商业销售 GLP-2 类似物的国家的回复。对每个问题进行了描述性分析。结果以中位数(四分位距)表示。

结果

19 家具有 GLP-2 类似物使用经验的 IF 专家中心的回复表明,仅有 10(10-20)%的 SBS-IF 患者接受了 GLP-2 类似物治疗,这低于符合治疗条件的患者人数(30(25-40)%)。在大多数中心(10 家中心,53%)中,GLP-2 治疗在最后一次肠切除术后 6-12 个月开始,而 5 家中心(26%)在更晚(12-24 个月)开始。有多种参数联合用于确定 GLP-2 类似物的应答情况,其中最常见的三个参数为 PS 减少>20%(95%)、每周至少减少 1 天 PS(84%)和尿排量增加(68%)。在无应答者中,有 67%的中心在第一年停用了 GLP-2 治疗。最后,在出现严重不良反应的情况下,策略包括停用 GLP-2 类似物(79%的专家使用)、减少剂量(67%)和暂时中断治疗(62%)。

结论

本项由 IF 专家中心完成的调查结果展示了 GLP-2 类似物在临床实践中的真实应用。确定的主要学习要点包括在开始 GLP-2 类似物治疗之前考虑一段肠道适应期,以及在无应答时不要过早停止治疗。在出现不良反应时的最佳策略应进一步研究。

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