BSPED2023 Oral Communications Oral Communications 10 (6 abstracts)
The real-world experience of long acting growth hormone in children with growth hormone deficiency
Pon Ramya Gokul 1 , Charlotte Jarvis 2 , Peter Laing 2 , Urmi Das 2 , Renuka Ramakrishnan 2 , Poonam Dharmaraj 2 , Jo Blair 2 , Mohammed Didi 2 & Senthil Senniappan 2
1Alderhey Children’s Hospital, Liverpool, United Kingdom. 2Alderhey Children’s Hospital, Liverpool, United Kingdom
Introduction: Long-acting growth hormone (LaGH) therapy has emerged as a newer treatment option for children, with the potential to improve adherence and compliance. Clinical trials have shown LaGH formulations to be effective and safe in children with GH deficiency (GHD). Somatrogon (Ngenla) is licensed for use in children with GHD over than 3 years of age.
Objective: We report the real-world experience of using once weekly Somatrogon (0.66mg/kg/week) injection in a group of children with GHD managed at a tertiary paediatric endocrine centre.
Methods: 19 patients diagnosed with GHD (M:F,12:7, mean age of 5.78 years) were started on LaGH therapy. 5 patients were GH naïve, and 14 patients were switched from daily GH to LaGH therapy. Auxology, and IGF-1 levels (measured on day 4 after the weekly injection) at baseline and 3 months after starting treatment was analysed.
Results: All patients demonstrated a good, annualised height velocity (9.9cm/year) on LaGH therapy (average dose 15mg weekly). The mean IGF1 level improved compared to baseline and was within the normal range [baseline mean IGF1 levels was 15.54nmol/L, and post LaGH therapy was 28.1nmol/L. The patients preferred to continue the once-weekly LaGH injections in the long run due to the reduced frequency of injections and reported better adherence and improved quality of life. The weekly injections were tolerated well, and no side effects were reported during this period.
Conclusion: We report the real-world experience of using LaGH for the first time in the UK children with GHD, outside the trial setting. Our study provides early evidence highlighting the acceptance, safety, and effectiveness of LaGH in a routine clinical setting. Further longitudinal data in a larger group of patients would help to establish the long-term benefits, efficacy, and safety of LaGH.
Volume 95
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Endocrine Abstracts
ISSN 1470-3947 (print) | ISSN 1479-6848 (online)
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