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儿科短肠综合征中特杜古肽的应用经验:首个真实世界数据。

Experience With Teduglutide in Pediatric Short Bowel Syndrome: First Real-life Data.

机构信息

Intestinal Rehabilitation Unit, Pediatric Gastroenterology and Nutrition Unit, University Hospital La Paz, Madrid.

University Hospital Vall d'Hebron, Barcelona.

出版信息

J Pediatr Gastroenterol Nutr. 2020 Dec;71(6):734-739. doi: 10.1097/MPG.0000000000002899.

Abstract

OBJECTIVES

The aim of the study was to describe the experience with teduglutide of several Spanish hospitals in pediatric patients with SBS (SBS).

METHODS

Seventeen pediatric patients with intestinal failure associated with SBS were treated with teduglutide. Patients received 0.05 mg · kg · day of subcutaneous teduglutide. Patients' demographics and changes in parenteral nutrition (PN) needs, fecal losses, and citrulline level initially and at 3, 6, and 12 months were collected, as well as any adverse events.

RESULTS

Patients were receiving 55 ml · kg · day and 33 kcal · kg · day of parenteral supplementation on average at baseline (2 patients received only hydroelectrolytic solution). A total of 12/17 patients achieved parenteral independence: 3 patients after 3 months of treatment, 4 patients at 6 months, and 5 after 12 months. One patient discontinued treatment 1 year after the beginning as no changes in parenteral support or fecal losses were obtained. All others decreased their intravenous requirements by 50%. One patient suffered an episode of cholecystitis, and another one with a pre-existing cardiac disease, developed a cardiac decompensation.

CONCLUSIONS

Teduglutide seems to be a safe and effective treatment in the pediatric SBS population with better results than in the pivotal study as well as in the adult population.

摘要

目的

本研究旨在描述几家西班牙医院在患有短肠综合征(SBS)的儿科患者中使用特杜古肽的经验。

方法

17 名患有与 SBS 相关的肠道衰竭的儿科患者接受了特杜古肽治疗。患者接受 0.05mg/kg/天的皮下特杜古肽。收集了患者的人口统计学数据以及肠外营养(PN)需求、粪便丢失量和瓜氨酸水平的变化,最初为 3、6 和 12 个月,以及任何不良反应。

结果

患者在基线时平均接受 55ml/kg/天和 33kcal/kg/天的肠外补充(2 名患者仅接受水电解质溶液)。总共 12/17 名患者实现了肠外独立:3 名患者在治疗 3 个月后,4 名患者在 6 个月后,5 名患者在 12 个月后。1 名患者在开始治疗 1 年后停止治疗,因为没有获得肠外支持或粪便丢失量的变化。其他所有人的静脉需求减少了 50%。1 名患者患有胆囊炎,另 1 名患有先前存在的心脏病的患者出现了心脏失代偿。

结论

特杜古肽似乎是患有短肠综合征的儿科患者的一种安全有效的治疗方法,其结果优于关键性研究以及成人患者。

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