Department of Pediatrics.
Department of Internal Medicine, Endocrine Division (SEMPR), Federal University of Parana, Curitiba, Brazil.
Curr Opin Pediatr. 2024 Aug 1;36(4):437-441. doi: 10.1097/MOP.0000000000001362. Epub 2024 May 7.
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.
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.
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.
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，并成为解决重组人生长激素（rhGH）每日注射挑战的潜在解决方案。在这篇综述中，我们简要介绍了 LAGH 的发展历史，并对目前已批准用于儿童治疗的五种 LAGH 的现有文献进行了批判性分析。
在临床试验中，LAGH 治疗在促进生长激素缺乏症儿童的线性生长方面与每日 rhGH 治疗相比显示出非劣效性，不良事件发生率相似。
在现实世界中，仍有许多问题需要解答，例如是否特定患者群体将从每周注射中获益最大，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，以及成本效益是否证明其在不同环境下的使用是合理的。
