ESPE2022 Poster Category 1 GH and IGFs (27 abstracts)
A Long-Acting Growth Hormone Module For Paediatric Growth Hormone Deficiency In The Global Registry For Novel Therapies In Rare Bone & Endocrine Conditions – The GloBE-Reg LAGH Study
Bradley Miller 1 , Lars Savendahl 2 , Krystina Hickman 3 , Christopher Smythe 3 , Ching Chen 3 , Jin-Ho Choi 4 , Xinyu Dou 5 , Chunxiu Gong 5 , Rasha Hamza 6 , Reiko Horikawa 7 , Alexander Jorge 8 & S. Faisal Ahmed 3
1University of Minnesota Masonic Children's Hospital, Minneapolis, USA; 2Karolinska Institute, Stockholm, Sweden; 3Office for Rare Conditions, University of Glasgow, Glasgow, United Kingdom; 4Department of Pediatrics, Asan Medical Center, University of Ulsan College of Medicine, Seoul, Republic of South Korea; 5Beijing Children's Hospital, the Capital Medical University, National Center for Children's Health, Beijing, China; 6Department of Pediatrics, Pediatric Endocrinology Unit, Ain Shams University, Cairo, Egypt; 7Division of Endocrinology and Metabolism, National Center for Child Health and Development, Tokyo, Japan; 8Genetic-Endocrinology Unit, Endocrinology Division of Hospital das Clinicas of University of Sao Paulo School of Medicine, Sao Paulo, Brazil
Introduction: Post-marketing surveillance registries provided extensive information about the safety and efficacy of daily growth hormone (GH) therapy during treatment. With the availability of novel long-acting GH (LAGH) therapies, it is important to determine whether the novel molecules or the different pattern of GH exposure lead to changes in the efficacy and safety profile. Therefore, new surveillance registries of LAGH are warranted.
Objective: Develop an international electronic platform to capture real world safety and efficacy data for children receiving LAGH for GH deficiency (GHD).
Methods: To capture key safety and efficacy data points for multiple different LAGH products, the project will rely on the GloBE-Reg platform, a new international registry project that has been designed to collect relevant real world data through a joint collaborative approach between scientific centres, industry partners and medical societies. The steering committee of GloBE-Reg consists of representatives from a wide range of paediatric endocrine organisations across the world as well as members of industry. The fields within the LAGH module will be guided by the GloBe-Reg LAGH Scientific Study Group and will have diagnosis and drug specificity. Electronic data entry by local institutions will focus on a group of core condition- and treatment-specific items, a group of optional broader data and data targeting specific research questions submitted by investigator groups.
Results: The core fields within GloBE-Reg have been developed to align with international accepted standards and the GloBe-Reg LAGH Scientific Study Group has started to meet and advise on the specific module for novel LAGH therapy in paediatric GHD. It is anticipated that the module specific fields shall be agreed by the third quarter of 2022 and the module will be live by the end of 2022.
Conclusion: GloBE-Reg LAGH is being developed to capture real world evidence for the safety and efficacy of LAGH in an international cohort of children. GloBE-Reg will also use this approach to serve the needs of the endocrine community for assessing longer term clinical outcomes, which are particularly important to continuously expand our scientific knowledge and improve care in children. Additional modules may be developed for specific LAGH products and other therapies for growth disorders in partnership with industry. More information is available on the project’s website: www.GloBE-Reg.net and by following @globe_reg on Twitter.
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