Long-acting growth hormone—A new treatment option for children with growth hormone deficiency: status 2024 Lang wirksames Wachstumshormon – eine neue Therapieoption für Kinder mit Wachstumshormonmangel: Status 2024

Dörr HG, Bettendorf M, Wölfle J, Saenger P, Ranke MB (2024)

Publication Type: Journal article

Publication year: 2024

Journal

Monatsschrift Kinderheilkunde Springer Verlag (Germany)

DOI: 10.1007/s00112-024-02022-7

Abstract

Until recently, daily singular subcutaneous injections of recombinant human growth hormone (rhGH) was the treatment of choice in short-statured children with growth hormone deficiency (GHD). This concept has been effective and safe for almost four decades; however, with this practice of replacement therapy approximately 10–20% of the children did not reach an adult height within the target range of the parents. Among the various factors involved, insufficient adherence, as the consequence of the necessary but inconvenient daily injections has been identified as a particularly important factor. This prompted the development of long-acting growth hormone (LAGH). At the end of 2023 there were three approved LAGH preparations available in Germany for the treatment of children older than 3 years with GHD due to an insufficient secretion of GH. All approved LAGH formulations use different technologies to prolong the half-life of rhGH. The LAGH as well as rhGH bind to the GH receptor and initiate the signal cascade that leads to the activation of growth and metabolism. In phase 3 studies LAGH showed non-inferiority to daily rhGH injections in terms of increasing height as well as comparability in the safety profiles. Thus, the treatment with LAGH offers a new option for children and adolescents with GHD; however, the long-term efficacy and safety need further systematic evaluation. Parents and patients need to be informed about all relevant aspects of the new treatment. This review article discusses the currently approved LAGH preparations in Germany together with the published data from a pediatric perspective.

Authors with CRIS profile

Helmuth-Günther Dörr Medizinische Fakultät Joachim Wölfle Lehrstuhl für Kinder- und Jugendmedizin

Involved external institutions

Universitätsklinikum Heidelberg DE Germany (DE) NYU Langone Medical Center US United States (USA) (US) Universitätsklinikum Tübingen DE Germany (DE)

How to cite

APA:

Dörr, H.-G., Bettendorf, M., Wölfle, J., Saenger, P., & Ranke, M.B. (2024). Long-acting growth hormone—A new treatment option for children with growth hormone deficiency: status 2024 Lang wirksames Wachstumshormon – eine neue Therapieoption für Kinder mit Wachstumshormonmangel: Status 2024. Monatsschrift Kinderheilkunde. https://doi.org/10.1007/s00112-024-02022-7

MLA:

Dörr, Helmuth-Günther, et al. "Long-acting growth hormone—A new treatment option for children with growth hormone deficiency: status 2024 Lang wirksames Wachstumshormon – eine neue Therapieoption für Kinder mit Wachstumshormonmangel: Status 2024." Monatsschrift Kinderheilkunde (2024).

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