儿童使用长效生长激素的最新情况。

IF 2.2 3区 医学 Q2 PEDIATRICS Current opinion in pediatrics Pub Date : 2024-08-01 Epub Date: 2024-05-07 DOI:10.1097/MOP.0000000000001362
Margaret Cristina da Silva Boguszewski, Cesar Luiz Boguszewski
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引用次数: 0

摘要

综述目的:经过广泛的研究和多年的等待,长效生长激素(LAGH)制剂终于在临床实践中成为现实,并成为解决每日注射重组人生长激素(rhGH)难题的潜在解决方案。在这篇综述中,我们简要介绍了LAGH的发展历史,并对现有文献进行了批判性分析,介绍了迄今为止可用于儿童治疗并获得批准的五种LAGH:在临床试验中,LAGH疗法在促进GH缺乏症儿童的线性生长方面与每日rhGH疗法相比并无劣势,且不良反应发生率相似。总结:在现实世界中,许多问题仍有待解答,如特定患者群体是否会从每周注射中获益最多,与每日rhGH相比,依从性是否会更好,不同LAGH化合物的长期疗效、监测和安全性是否相同,以及成本效益是否能证明它们在不同环境中的使用是合理的。
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Update on the use of long-acting growth hormone in children.

Purpose of review: After extensive research and many years of waiting, long-acting growth hormone (LAGH) formulations have finally become a reality in clinical practice and emerge as a potential solution to address the challenges of daily injections of recombinant human GH (rhGH). In this review, we present a brief history of the development of LAGH and provide a critical analysis of the existing literature on the five LAGH available and approved to date for treatment in children.

Recent findings: In clinical trials, LAGH therapy has shown noninferiority compared with daily rhGH therapy in promoting linear growth in children with GH deficiency, with similar rates of adverse events.

Summary: In the real world, many questions still need to be answered, such as whether a specific group of patients will benefit most from the weekly injection, whether compliance will be better compared with daily rhGH, whether long-term efficacy, monitoring and safety profile will be the same for the different LAGH compounds, and whether the cost-effectiveness will justify their use in different settings.

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来源期刊
CiteScore
6.20
自引率
0.00%
发文量
184
审稿时长
6-12 weeks
期刊介绍: ​​​​​Current Opinion in Pediatrics is a reader-friendly resource which allows the reader to keep up-to-date with the most important advances in the pediatric field. Each issue of Current Opinion in Pediatrics contains three main sections delivering a diverse and comprehensive cover of all key issues related to pediatrics; including genetics, therapeutics and toxicology, adolescent medicine, neonatology and perinatology, and orthopedics. Unique to Current Opinion in Pediatrics is the office pediatrics section which appears in every issue and covers popular topics such as fever, immunization and ADHD. Current Opinion in Pediatrics is an indispensable journal for the busy clinician, researcher or student.
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