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First 100 patients receiving long-acting growth hormone therapy: real-world evaluation from INSIGHTS-GHT registry

Wölfle J, Kreitschmann-Andermahr I, Strasburger CJ, Pittrow DB, Pausch C, Schnabel D (2025)

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Publication Type: Journal article

Publication year: 2025

Journal

Book Volume: 20

Article Number: 372

Journal Issue: 1

DOI: 10.1186/s13023-025-03898-8

Abstract

The development of long-acting growth hormone (LAGH) formulations offers a promising approach to reduce injection frequency and to improve adherence in growth hormone deficiency (GHD) treatment. INSIGHTS-GHT is the first product-independent registry to document the real-world use of recombinant human (rh) growth hormone (GH) replacement therapy within the labelling. Following the market launch of three LAGH products in Germany (lonapegsomatropin, somapacitan, and somatrogon) we aimed to provide early real-world evidence on their use in order to obtain an initial picture on patient selection and physician preferences outside of clinical trials. We report in this interim analysis on 70 pediatric patients from 15 centers across Germany as well as 31 adult patients from 6 German centers under LAGH treatment. The majority of the pediatric patients (76%) were male, with a mean age at LAGH initiation of 9.2 years. About half of the pediatric patients (54%) were switch patients transitioning from daily GH therapy. Notably, 82% of patients received a LAGH starting dose below the manufacturer’s recommendation, with a median dose of 92% of the recommended level. In the group of adult patients, 65% were male, with a mean age of 38.2 years at LAGH initiation. In pediatric patients, before start of GH therapy, mean IGF-I (SDS) was − 2.1 ± 1.1 SDS, mean IGFBP-3 (SDS) was − 2.0 ± 1.5 SDS. All adult patients switched from daily GH therapy. More than half (55%) received the LAGH starting dose according to the manufacturer’s recommendation, while 41% began with a lower-than-recommended dose. Our findings provide early insights into LAGH therapy adoption and highlight the need for continued follow-up to evaluate long-term efficacy, adherence, and safety in real-world settings.

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How to cite

APA:

Wölfle, J., Kreitschmann-Andermahr, I., Strasburger, C.J., Pittrow, D.B., Pausch, C., & Schnabel, D. (2025). First 100 patients receiving long-acting growth hormone therapy: real-world evaluation from INSIGHTS-GHT registry. Orphanet Journal of Rare Diseases, 20(1). https://doi.org/10.1186/s13023-025-03898-8

MLA:

Wölfle, Joachim, et al. "First 100 patients receiving long-acting growth hormone therapy: real-world evaluation from INSIGHTS-GHT registry." Orphanet Journal of Rare Diseases 20.1 (2025).

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