Bouhours-Nouet N, Teinturier C
Pediatric Endocrinology and Diabetology Unit, Angers University Hospital, Angers, France; Reference center for rare diseases of thyroid origin, Angers University Hospital, Angers, France; Reference center for rare diseases of pituitary origin (constitutive center), Angers University Hospital, Angers, France.
Pediatric Endocrinology and Diabetology Unit, Le Kremlin-Bicêtre University Hospital, Le Kremlin Bicêtre, France; Reference center for rare diseases of pituitary origin (constitutive center), Le Kremlin-Bicêtre University Hospital, Le Kremlin Bicêtre, France.
Arch Pediatr. 2022 Feb;28(8S1):8S14-8S20. doi: 10.1016/S0929-693X(22)00038-0.
The widespread availability of recombinant human growth hormone (GH) since 1985 has made it possible to extend growth hormone therapy indications for children with different etiologies of short stature. It is now accepted that GH is effective in increasing height velocity in children with GH deficiency (GHD) and well tolerated in both the short and long terms. Nevertheless, one major factor in the inter-individual variability of the growth response to GH in GHD children is adherence to daily injections. In an attempt to improve patient adherence, there has been a strong effort from industry to create a long-acting form of growth hormone to ease the burden of its use. It is theorized that by decreasing injection frequency, Long-Acting GH (LAGH) would improve adherence and therefore outcomes. These agents can be divided broadly into depot formulations, PEGylated formulations, prodrug formulations, noncovalent albumin-binding GH and GH fusion proteins. In 2021, two LAGH formulations are on the market in China and South Korea, and several promising agents are under clinical investigation at various stages of development throughout the world. A number of safety issues related to LAGH have been identified and need to be further investigated. © 2022 French Society of Pediatrics. Published by Elsevier Masson SAS. All rights reserved.
自1985年重组人生长激素（GH）广泛可得以来，已能够扩大生长激素治疗对不同病因身材矮小儿童的适应症。目前公认，GH对生长激素缺乏症（GHD）儿童增加身高增长速度有效，且短期和长期耐受性良好。然而，GHD儿童对GH生长反应个体间差异的一个主要因素是对每日注射的依从性。为了提高患者依从性，业界一直在大力研发长效生长激素以减轻其使用负担。理论上，通过减少注射频率，长效GH（LAGH）将提高依从性，从而改善治疗效果。这些药物大致可分为长效注射剂、聚乙二醇化制剂、前药制剂、非共价白蛋白结合GH和GH融合蛋白。2021年，两种LAGH制剂在中国和韩国上市，全球有几种有前景的药物正处于不同开发阶段的临床研究中。已发现一些与LAGH相关的安全问题，需要进一步研究。© 2022法国儿科学会。由爱思唯尔马松SAS出版。保留所有权利。
