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. 2025 Jul 23;20(1):372.
doi: 10.1186/s13023-025-03898-8.

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

Joachim Woelfle  1 , I Kreitschmann-Andermahr  2 , C J Strasburger  3 , D B Pittrow  4   5 , C Pausch  5 , D Schnabel  6
Affiliations

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

Joachim Woelfle et al. Orphanet J Rare Dis. .

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.

Keywords: Growth hormone; Lonapegsomatropin; Non-interventional study; Pediatrics; Somapacitan; Somatrogon.

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Conflict of interest statement

Declarations. Ethics approval and consent to participate: The registry materials (protocol, patient information and informed consent form (ICF), and other required documents) were approved by the primary ethics committee (Ethics Committee of the Physician Chamber Saxony EK-BR-8/22 − 1 dated 26 January 2022). Any site that joins the registry has to provide additional ethics approval, if required by their local institutions. Protocol amendments, if any, will be subject to the same requirements as the original protocol. Written informed consent is a prerequisite for patients to be eligible for documentation in the study. Consent for publication: All patients or their caregivers have given consent for publication of their pseudonymised data in a pooled format. The manuscript does not contain any individual person’s data in any form. Competing interests: DS: received consultant and / or speaker fees from Ascendis, Biomarin, Kyowa Kirin, Merck/Serono, Novo Nordisk, Pfizer, Sandoz/Novartis. CS: received consultancy fees from NovoNordisk, Sandoz-Hexal/Novartis, Ascendis, Pfizer, Consilient Health, Crinetics and Ipsen. DP: received consultancy fees from Amgen, Aspen, Biogen, Daiichi Sankyo, Sanofi-Genzyme, Sandoz/Novartis and MSD. CP: has no competing interests. JW: received consultant and / or speaker fees from Ascendis, Biomarin, Merck/Serono, Novo Nordisk, Pfizer, Sandoz/Novartis. IKA: received consultant and/or speaker fees from Sandoz/Novartis, Novo Nordisk and Pfizer. Study registration: BfArM Nr. NIS7492, DRKS registry DRKS00027394.

References

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