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单个中心的特杜格鲁肽的长期结果。

Long-Term Outcomes With Teduglutide From a Single Center.

机构信息

Department of Internal Medicine, University of Chicago Medicine, Chicago, Illinois, USA.

Nutrition Support Team, University of Chicago Medicine, Chicago, Illinois, USA.

出版信息

JPEN J Parenter Enteral Nutr. 2021 Feb;45(2):318-322. doi: 10.1002/jpen.1838. Epub 2020 May 11.

Abstract

BACKGROUND

The aim of this study was to quantify the long-term clinical outcomes for individuals receiving teduglutide for short-bowel syndrome (SBS).

METHODS

A single-center, retrospective study was performed for individuals commencing use of teduglutide between March 2013 and May 2019.

RESULTS

Eighteen patients were included in the final analysis, among which the median duration of teduglutide administration was 3.2 (range, 0.6-6.2) years. Twelve of 16 (75%) patients at 12 months, 10 of 13 (76.9%) at 24 months, 7 of 10 (70%) at 36 months, and 3 of 3 (100%) at 60 months had a response to teduglutide therapy, defined as a >20% reduction in parenteral support (PS) requirement. Among responders at 12, 24, and 36 months, the presence of a colon-in-continuity, an ileocecal valve, a response at 3 months, the length of small bowel, nor the baseline volume affected response to therapy (P > .05 for all comparisons). Five (28%) patients were able to achieve freedom from PS, among which all had a history of Crohn's disease with loss of the ileocecal valve and among which 3 had a colon-in-continuity. Four of the 5 patients discontinued PS by 6 months of teduglutide therapy.

CONCLUSIONS

In a real-world experience, teduglutide therapy results in rapid and sustained reductions in PS. Larger postmarketing studies will be required to reliably predict response to treatment and the factors associated with enteral autonomy.

摘要

背景

本研究旨在量化接受特杜古肽治疗短肠综合征(SBS)个体的长期临床结局。

方法

对 2013 年 3 月至 2019 年 5 月期间开始使用特杜古肽的个体进行了单中心回顾性研究。

结果

最终分析纳入 18 例患者,特杜古肽中位给药时间为 3.2(范围 0.6-6.2)年。12 例(75%)患者在 12 个月、10 例(76.9%)在 24 个月、7 例(70%)在 36 个月、3 例(100%)在 60 个月时对特杜古肽治疗有反应,定义为肠外支持(PS)需求减少≥20%。在 12、24 和 36 个月时的应答者中,结肠连续性、回盲瓣、3 个月时的应答、小肠长度以及基线容量均不影响治疗反应(所有比较的 P >.05)。5 例(28%)患者能够实现无 PS,其中均有回盲瓣缺失的克罗恩病病史,其中 3 例有结肠连续性。5 例患者中有 4 例在特杜古肽治疗 6 个月时停用 PS。

结论

在真实世界的经验中,特杜古肽治疗可迅速和持续减少 PS。需要更大的上市后研究来可靠预测治疗反应和与肠内自主相关的因素。

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