Long-acting recombinant human growth hormone in the treatment of pediatric growth hormone deficiency, how far have we got?

ABSTRACT

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.