The assessment of the effectiveness and safety of teduglutide in the treatment of children with chronic intestinal insufficiency. A systematic review

F. Ampar, V. Rozinov, Maria M. Chernobabova
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Abstract

Chronic intestinal failure is a pathogenetic component in children with short bowel syndrome. Short bowel syndrome limits the patient's capacity to socialize fully, the bad prognosis of the child's physical and spiritual development, and the patient's family's organizational and material issues. The severity of the problem is aggravated by the limited prospects for surgical treatment of patients, including transplantology, the imperfection of the legal framework for health care, particularly in terms of parenteral nutrition at home, and the supply of expensive drugs, consumables, and equipment to children with short bowel syndrome under state guarantees. Developing an equivalent of glucagon-like peptide-2 (GLP-2), a drug teduglutide representing the endogenous intestinal growth factor and intestinal adaptation, created an additional therapeutic possibility for individuals with short bowel syndrome. There are no data in Russian academic literature on the efficacy and safety of teduglutide in pediatric practice. This study aims to conduct a systematic search and critical analysis of publications on the effectiveness and safety of using teduglutide in children with short bowel syndrome. Publications from the PubMed, Scopus, and eLibrary databases, as well as scientific journals, were examined from 2012 to 2022. The articles were reviewed in English and Russian. Seven hundred seventy publications on using teduglutide in experimental and clinical practice were found; 94 sources matched the keywords; and eight articles were selected according to the PRISMA criteria. The risks of confounding bias were assessed using the ROBINS-I tool. Most patients received the therapeutic impact of teduglutide, and 8.4%64.7% of patients achieved enteral autonomy. The drug's dose was 0.05 mg/kg/day. Without a therapeutic effect, the time limits for the inappropriate use of teduglutide ranged from 6 months to 1 yr. There is no causal relationship between taking teduglutide and undesirable reactions to treatment. The review of publications revealed an insufficient evidence foundation for the efficacy of teduglutide due to a lack of control groups in most studies, a small sample size, and errors in data methodology. Despite the encouraging preliminary results of individual studies, the problem of a shortage of randomized controlled tests of teduglutide remains.
特杜鲁肽治疗儿童慢性肠功能不全的有效性和安全性评价。系统回顾
慢性肠衰竭是儿童短肠综合征的一个致病因素。短肠综合征限制了患者充分社交的能力,儿童身心发展预后不良,患者家庭的组织和物质问题。由于手术治疗病人的前景有限,包括移植,保健法律框架不完善,特别是在家庭外营养方面,以及在国家保障下向患有短肠综合症的儿童提供昂贵的药物、消耗品和设备,使问题的严重性更加严重。开发胰高血糖素样肽-2 (GLP-2)等效物,一种代表内源性肠道生长因子和肠道适应性的药物teduglutide,为短肠综合征患者创造了额外的治疗可能性。在俄罗斯的学术文献中没有关于特杜鲁肽在儿科实践中的有效性和安全性的数据。本研究的目的是进行系统的搜索和批判性分析的出版物使用的有效性和安全性的特杜卢肽短肠综合征的儿童。从2012年到2022年,研究人员检查了PubMed、Scopus和图书馆数据库以及科学期刊的出版物。这些文章用英文和俄文进行了评论。在实验和临床实践中发现了770篇关于特杜葡肽使用的出版物;94个来源匹配关键字;根据PRISMA标准选出8篇文章。使用ROBINS-I工具评估混杂偏倚的风险。大多数患者获得了特杜鲁肽的治疗效果,8.4%的患者达到了64.7%的肠内自主性。给药剂量为0.05 mg/kg/d。在没有治疗效果的情况下,不适当使用teduglutide的时限从6个月到1年不等。服用teduglutide与治疗不良反应之间没有因果关系。对出版物的回顾显示,由于大多数研究缺乏对照组,样本量小以及数据方法错误,teduglutide有效性的证据基础不足。尽管个别研究的初步结果令人鼓舞,但缺乏teduglutide随机对照试验的问题仍然存在。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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