Long-Acting Growth Hormone Therapy in Children with Growth Hormone deficiency – A two centre real world experience from UK

Gokul Pon Ramya; Valerie Wang Choon Li; Charlotte Jarvis; Peter Laing; Sandra Greetham; Joanne Blair; Renuka Ramakrishnan; Mohammed Didi; Urmi Das; Sanjay Gupta; Senthil Senniappan

Introduction: Recombinant human growth hormone (rhGH) has been pivotal in growth hormone (GH) therapies for children since its approval for paediatric growth hormone deficiency (PGHD). The recent introduction of long-acting growth hormone (LAGH) represents a further advancement in GH therapy, demonstrating equal efficacy compared to daily GH with the added benefit of decreased injection frequency. Clinical trials have underscored the effectiveness and safety of LAGH formulations in children with growth hormone deficiency (GHD). We present the first real-world experience of Somatrogon usage in children with GHD.

Objective: This study aims to offer real-world insights into the use of once-weekly Somatrogon injection in children with GHD treated at two paediatric endocrine centres in the UK.

Methods: A total of 52 patients with GHD (34 males), average age 9.63 years (range 3 to 16.58 years) were commenced on LAGH therapy (starting dose 0.66 mg/kg/week). 20 patients were GH naïve, and 32 were switched from daily GH to LAGH. Auxology and IGF-1 levels (measured four days post-weekly injection) were evaluated at baseline, 6 weeks, and between 3 to 6 months post-LAGH treatment. A formal, structured feedback questionnaire was gathered from patients and caregivers about their experiences with LAGH usage. The primary reasons for preferring LAGH treatment included needle phobia, to maintain good compliance, and convenience.

Results: All patients demonstrated favourable annualised height velocity, 8.5 ± 2.3 cm/year and 6.7 ± 2.9cm/ year in GH naïve and those switched from daily GH respectively. IGF-1 levels gradually improved and remained below +2 SDS post-treatment for most patients. 5 patients had IGF1 levels above +2SDS necessitating a 15% dose reduction, following which IGF-1 levels normalised. The weekly injections were well-tolerated, with no significant side effects, such as hypoglycaemia or symptoms indicative of raised intracranial pressure. Most of the patients favoured continuing weekly LAGH injections due to reduced frequency, improved adherence, and enhanced quality of life. 3 patients switched back to daily GH, 1 patient completed growth, and 1 patient declined further rhGH therapy.

Conclusion: To our knowledge, this is the first study providing initial real-world insights into the application of LAGH in the UK children with GHD, beyond clinical trial settings. Our findings suggest favourable acceptance, safety, and efficacy of LAGH in routine clinical practice. Longitudinal data from a larger patient cohort would further elucidate the enduring benefits and safety profile of LAGH.

ESPE Abstracts

P1-260