ECE2024 Poster Presentations Pituitary and Neuroendocrinology (120 abstracts)
Minimum data set for global monitoring of the safety and efficacy of growth hormone replacement in adults - an expert group recommendation
Suet Ching Chen 1 , Faisal Ahmed 2 , Deno Andrews 3 , Beverly M.K. Biller 4 , Cesar Luiz Boguszewski 5 , Jillian Bryce 6 , Minglu Chen 7 , Peter Clayton 8 , Maria Fleseriu 9 , Judith Gebauer 10 , Ken Ho 11 , Jens Otto Jørgensen 12 , Xiaoping Luo 13 , Bradley Miller 14 , Sebastian Neggers 15 , Lars Savendahl 16 , Katharina Schilbach 17 , Christian J. Strasburger 18 , Yutaka Takahashi 19 , Diana Vitali 20 , Kevin Yuen 21 , Andrew Hoffman 22 & Gudmundur Johannsson 23
1Office for Rare Conditions, University of Glasgow, Developmental Endocrinology Research Group, Royal Hospital for Children, Paediatric Endocrinology, Glasgow, United Kingdom; 2Office for Rare Conditions, University of Glasgow, Developmental Endocrinology Research Group, Royal Hospital for Children, Paediatric Endocrinology, Glasgow, United Kingdom; 3ICOSEP, Chicago, United States; 4Neuroendocrine & Pituitary Tumor Clinical Center, Massachusetts General Hospital, United States; 5SEMPR, Endocrine Division, Federal University of Parana, Brazil; 6Office for Rare Conditions, University of Glasgow, United Kingdom; 7, Office for Rare Conditions, University of Glasgow, Glasgow, United Kingdom; 8University of Manchester, Manchester, United Kingdom; 9Pituitary Center, Departments of Medicine and Neurological Surgery, Oregon Health & Science University, Portland, United States; 10Department of Internal Medicine I, University Hospital of Schleswig-Holstein, Lubeck, Germany; 11The Garvan Institute of Medical Research and St. Vincent’s Hospital, Sydney, Australia; 12Aarhus University Hospital, Aarhus, Denmark; 13Department of Pediatrics, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, China; 14Division of Endocrinology, Department of Pediatrics, University of Minnesota Medical School, M Health Fairview Masonic Children’s Hospital, Minneapolis, United States; 15Dept. of Internal Medicine section Endocrinology and Pituitary Center Rotterdam, Rotterdam, Netherlands; 16Dept. of Women´s and Children´s Health, Karolinska Institutet, Stockholm, Sweden; 17Medizinische Klinik und Poliklinik IV, Faculty of Medicine, LMU Munich, Munich, Germany; 18Dept. of Endocrinology and Metabolic Disorders, Charite universitaetsmedizin, Berlin, Germany; 19Department of Diabetes and Endocrinology, Nara Medical University, Nara, Japan; 20SOD Italia, Rome, Italy; 21Departments of Neuronedocrinology and Neurosurgery, Barrow Pituitary Center, Barrow Neurological Institute, University of Arizona College of Medicine, Phoenix, United States; 22Dept. of Medicine, Stanford University School of Medicine, Stanford, United States; 23Department of Endocrinology, Sahlgrenska University Hospital and Institute of Medicine, Sahlgrenska, Sahlgrenska, Sweden
Introduction: Growth hormone (GH) replacement therapy is safe and confers significant benefits in body composition, bone health, cardiometabolic risk factors, and quality of life in adults with GH deficiency. However, longer term safety and efficacy data are limited by the lack of consistency in data collection between studies. The study aim was to develop a simple global register by standardising and developing a minimum data set (MDS) to facilitate international collection of safety and efficacy data.
Methods: This study was undertaken by the Adulthood GHD Expert Working Group (EWG) in GloBE-Reg, the Global Registry for Novel Therapies in Rare Bone and Endocrine Conditions (https://globe-reg.net/). Seventeen international clinical experts from 10 countries and two representatives from patient organisations participated in this initiative using published methodology for the grading system of MDS in childhood-onset GHD (cGHD) (doi: 10.1159/000533763). Inclusion criteria required >70% consensus for importance as defined for the MDS, provided <50% of the group deemed the item difficult to collect. The development of the finalised MDS took place over four months, which included two virtual meetings among the EWG members.
Results: A total of 190 data fields compiled from routine clinical practice of the EWG and fields in the cGHD MDS were graded. Of these, 111 items fulfilled the criteria for importance whilst 117 items were deemed easy to collect. Combining the criteria for importance and ease of data collection, 86 items fulfilled the criteria for MDS. Six items were excluded as one was redundant, another was a calculated field and four were designated as core data. Several fields such as adherence and patient reported outcomes while important, were considered difficult to assess objectively in routine clinical settings. Following detailed discussions, twenty items unrelated to safety and efficacy of GH therapy were removed, with the remaining 60 items merged into the finalised MDS recommendation of 43 items.
Conclusion: In summary, GloBE-Reg has developed a MDS suitable for monitoring the long-term safety and effectiveness of GH replacement therapy in adults during routine clinical practice world-wide. Such data harmonisation that bridges the monitoring of safety and efficacy of growth hormone therapy across the age span in patients of different healthcare settings will be pivotal in facilitating longer term surveillance and improving patient outcomes.
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Endocrine Abstracts
ISSN 1470-3947 (print) | ISSN 1479-6848 (online)
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