ESPE2024 Poster Category 1 Pituitary, Neuroendocrinology and Puberty 4 (9 abstracts)
The Use Of GloBE-Reg As A Global Platform For Performing Safety & Efficacy Studies Of rhGH Therapy
Malika Alimussina 1 , Jillian Bryce 1 , Minglu Chen 1 , Sanhita Koley 1 , Abdulmoein E Al-Agha 2 , Nadia Amin 3 , Navoda Atapattu 4 , Kate C Chan 5 , Jiajia Chen 6 , Ying Ki Chung 7 , Yana Deyanova 8 , Antony Fu 9 , Chunxiu Gong 6 , Violeta Iotova 10 , Dominika Janus 11 , Alexander AL Jorge 12 , Renata Markosyan 13 , Aristides Maniatis 14 , Sumudu Seneviratne 15 , Guftar M Shaikh 16,17 , Sheila Shepherd 17 , Lloyd JW Tack 18 , Malgorzata Wasniewska 19 , Sammy WC Wong 20 , Wai Yu Wai 21 , Suet Ching Chen 1,17 & S Faisal Ahmed 1,17
1Office for Rare Conditions, University of Glasgow, Glasgow, United Kingdom. 2Department of Paediatric Endocrinology, King Abdulaziz University Hospital, Jeddah, Saudi Arabia. 3Department of Paediatric Endocrinology, Leeds Children's Hospital, Leeds Teaching Hospitals NHS Trust, Leeds, United Kingdom. 4Endocrine and Diabetic Unit, Lady Ridgeway Hospital for Children, Colombo, Sri Lanka. 5Department of Paediatrics, Prince of Wales Hospital, The Chinese University of Hong Kong, Hong Kong, Hong Kong. 6Department of Endocrinology, Genetics and Metabolism, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing, China. 7Department of Paediatrics and Adolescent Medicine, Caritas Medical Centre, Hong Kong, Hong Kong. 8Medical University – Varna, First Paediatric Clinic with PICU, UMHAT Sveta Marina, Varna, Bulgaria. 9Department of Paediatrics and Adolescent Medicine, Princess Margaret Hospital and Yan Chai Hospital, Hong Kong, Hong Kong. 10Medical university – Varna and PC with PICU, UMHAT Sveta Marina, Varna, Bulgaria. 11Department of Pediatric and Adolescent Endocrinology, Jagiellonian University Medical College, Krakow, Poland. 12Genetic Endocrinology Unit, Cellular and Molecular Endocrinology Laboratory (LIM-25), University of Sao Paulo School of Medicine, Sao Paulo, Brazil. 13Wigmore clinic, Muracan University Hospital, Yerevan, Armenia. 14Rocky Mountain Pediatric Endocrinology, Centennial, USA. 15Department of Paediatrics, Faculty of Medicine, University of Colombo, Colombo, Sri Lanka. 16Department of Paediatric Endocrinology, Royal Hospital for Children, Glasgow, United Kingdom. 17Developmental Endocrinology Research Group, University of Glasgow, Royal Hospital for Children, Glasgow, United Kingdom. 18Department of Paediatric Endocrinology, Department of Paediatrics and Internal Medicine, Ghent University Hospital, Ghent University, Ghent, Belgium. 19Department of Human Pathology in Adulthood and Childhood, University of Messina, Messina, Italy. 20Department of Paediatrics & Adolescent Medicine, Alice Ho Miu Ling Nethersole Hospital, Hong Kong, Hong Kong. 21Paediatrics and Adolescent Medicine, Tuen Mun Hospital, Hong Kong, Hong Kong
Introduction: The Global Registry For Novel Therapies In Rare Bone & Endocrine Conditions (GloBE-Reg, https://globe-reg.net/) project was launched in 2022 with the aim of supporting studies that focus on effectiveness and long-term safety of specific therapies. The project’s initial focus has been on recombinant human growth hormone therapy (rhGH) given that there are existing gaps in knowledge with the introduction of new indications and novel forms of rhGH.
Methods: The GloBE-Reg registry has three layers of datasets, with the first dataset consisting of internationally agreed core data elements that can apply to any rare condition, another set of data elements that allows the selection of a specific therapy and diagnosis, a third set of data elements that include a therapy and diagnosis-specific minimum dataset (MDS) which collects information on diagnosis, therapy, clinician reported outcomes, patient reported outcomes and adverse events. The fields within the MDS are developed following guidance from expert working groups.
Results: Since its launch, 62 centres from 31 countries have registered their interest to use the platform and of these, 19 centres from 12 countries in 4 continents have already enrolled 738 (M:F, 443:295) patients with a median age of 12.7yr (range 1.8, 29.6). Of these, 580 (76%) were on daily rhGH, 148 (20%) on long-acting rhGH, 5 on daily rhGH as adults and in 5, rhGH therapy had been discussed but not started. Ten different brands of rhGH were being used across these centres for 8 indications, whereas long-acting rhGH was being used for 4 indications. In addition, there were 23 (3%) cases receiving rhGH for other conditions associated with short stature or growth retardation. The largest indication group was Growth Hormone Deficiency (GHD) (59%) followed by Turner Syndrome (10%), Small for Gestational Age (9%), Prader-Willi Syndrome (7%), Idiopathic Short Stature (8%). Of the 738 cases, 111 (15%) had also been entered in other disease registries and 18 (16%) of these had been entered by one centre into GloBE-Reg through its bulk upload facility. Childhood GHD, Adulthood GHD, and Noonan syndrome MDS are now fully operational and MDS for other conditions are under development.
Conclusion: GloBE-Reg has shown that a relatively low-cost, brand-agnostic platform can have sufficient stakeholder acceptability and versatility for collecting information that can support the development of long-term safety and effectiveness studies. The preliminary data that have been collected on rhGH show the utility of the platform for safety and effectiveness for a wide group of drugs.
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