IMPE2023 Poster Presentations GH and IGFs (14 abstracts)
Development of A Minimum Data Set for the Monitoring of Recombinant Human Growth Hormone (rhGH) Therapy in Children with Growth Hormone Deficiency (GHD)– A GloBE-Reg Initiative
Suet Ching Chen 1,2 , Evangelia Charmandari 3 , Jin-Ho Choi 4 , Xinyu Dou 5 , Chunxiu Gong 5 , Rasha Hamza 6 , Jamie Harvey 7 , Andrew R. Hoffman 8 , Reiko Horikawa 9 , Gudmundur Johannson 10 , Alexander Jorje 11 , Bradley Miller 12 , Lars Sävendahl 13 , Xanthippi Tseretopoulou 1,2 , Diana Vitali 14 & S. Faisal Ahmed 1,2
1Royal Hospital for Children, Glasgow, United Kingdom. 2Office for Rare Conditions, University of Glasgow, Glasgow, United Kingdom. 3Aghia Sophia Children’s Hospital, Athens, Greece. 4University of Ulsan College of Medicine, Seoul, Korea, Democratic People's Republic of. 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. 7MAGIC Foundation, Illinois, USA. 8Department of Medicine, Division of Endocrinology, Metabolism and Gerontology, Stanford University School of Medicine, California, USA. 9Division of Endocrinology and Metabolism, National Center for Child Health and Development, Tokyo, Japan. 10Institute of Medicine, Sahlgrenska Academy, University of Gothenburg, Sahlgrenska University Hospital, Gothenburg, Sweden. 11Genetic-Endocrinology Unit, Endocrinology Division of Hospital das Clinicas of University of Sao Paulo School of Medicine, Sao Paolo, Brazil. 12University of Minnesota Masonic Children’s Hospital, Minneapolis, USA. 13Karolinska Institute, Stockholm, Sweden. 14SOD Italia, Rome, Italy
Objectives: Although there are some recommendations in the literature on the assessments that should be performed in children on rhGH therapy, the level of consensus on these measurements is unclear. The objective of the current study was to identify the minimum dataset that could be measured in a routine clinical setting across the world.
Methods: This exercise was undertaken through the GH Scientific Study Group (SSG) in GloBE-Reg, a new project that has developed a common registry platform that can support long-term safety and effectiveness studies of drugs. 12 clinical experts from 7 international endocrine organisations identified by the GloBE-Reg Steering Committee, 2 patient representatives and representatives from 2 pharmaceutical companies with previous GH registry expertise collaborated to develop this recommendation. A comprehensive list of data fields routinely collected by each of the clinical and industry experts for children with GHD was compiled. Each member was asked to determine the: (1) Importance of the data field and (2) Ease of data collection. Data fields that achieved 70% consensus in terms of importance qualified for the minimum dataset, provided <50% deemed the item difficult to collect.
Results: 246 items were compiled and 21 removed due to redundancies, with 225 items subjected to the grading system. 113 of the 225 items achieved at least 70% consensus as important data to collect when monitoring children with growth hormone deficiency on rhGH treatment. 69 of the 225 items were deemed easy to collect. Combining the criteria of importance and ease of data collection, 64 met the criteria for minimum dataset. Of the 64, only 62 required active data entry as 2 were calculated parameters, namely mid-parental height and BMI. Several anomalies to the minimum dataset rule were identified and highlighted for discussion, including whether the patients were involved in current or previous clinical trials, need for HbA1c monitoring, date of first start of GH therapy, other past medical history, and adherence, enabling formulation of the final minimum dataset recommendation of 43 items; 20 of which will need to be completed once, 14 every 6 months and 9 every 12 months.
Conclusion: In summary, this exercise performed through the GloBE-Reg initiative provides a recommendation of the minimum dataset requirement, collected through real-world data, for the monitoring of safety and effectiveness of rhGH in children with GHD, both for the current daily preparations and also the newer long-acting growth hormone.
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