Hormone Therapy

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

TL;DR

This interim analysis of the first 100 patients receiving long-acting growth hormone therapy in the INSIGHTS-GHT registry provides early real-world evidence on patient selection and physician preferences, finding that a majority of pediatric patients received starting doses below manufacturer recommendations and about half were switch patients from daily therapy.

Key Findings

The INSIGHTS-GHT registry enrolled 70 pediatric patients from 15 German centers and 31 adult patients from 6 German centers receiving long-acting growth hormone (LAGH) therapy.

  • INSIGHTS-GHT is described as the first product-independent registry to document real-world use of recombinant human growth hormone replacement therapy within the labelling.
  • Three LAGH products were available following market launch in Germany: lonapegsomatropin, somapacitan, and somatrogon.
  • This report represents an interim analysis of the registry.

The majority of pediatric patients receiving LAGH therapy were male, with a mean age of 9.2 years at LAGH initiation.

  • 76% of pediatric patients were male.
  • Mean age at LAGH initiation was 9.2 years.
  • About half of the pediatric patients (54%) were switch patients transitioning from daily GH therapy.
  • Before start of GH therapy, mean IGF-I (SDS) was -2.1 ± 1.1 SDS and mean IGFBP-3 (SDS) was -2.0 ± 1.5 SDS.

The majority of pediatric patients received a LAGH starting dose below the manufacturer's recommendation.

  • 82% of pediatric patients received a LAGH starting dose below the manufacturer's recommendation.
  • The median dose administered was 92% of the recommended level.

All adult patients switched from daily GH therapy to LAGH, with over half receiving the manufacturer's recommended starting dose.

  • 100% of adult patients were switch patients transitioning from daily GH therapy.
  • 65% of adult patients were male, with a mean age of 38.2 years at LAGH initiation.
  • More than half (55%) received the LAGH starting dose according to the manufacturer's recommendation.
  • 41% of adult patients began with a lower-than-recommended dose.

The registry findings highlight the need for continued follow-up to evaluate long-term efficacy, adherence, and safety of LAGH therapy in real-world settings.

  • The development of LAGH formulations is described as offering a promising approach to reduce injection frequency and improve adherence in growth hormone deficiency treatment.
  • The authors note that this interim analysis provides only an 'initial picture on patient selection and physician preferences outside of clinical trials.'
  • Continued follow-up is identified as necessary to evaluate long-term outcomes.

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Citation

Woelfle J, Kreitschmann-Andermahr I, Strasburger C, Pittrow D, 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. https://doi.org/10.1186/s13023-025-03898-8