THU166 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

Abstract

Abstract Disclosure: S.C. Chen: None. E. Charmandari: None. J. Choi: None. X. Dou: None. C. Gong: None. R. Hamza: None. J. Harvey: None. A.R. Hoffman: None. R. Horikawa: None. G. Johannsson: None. A.A. Jorge: None. B.S. Miller: None. S. Roehrich: None. L.S. Savendahl: None. X. Tserotopolou: None. M.P. Wajnrajch: None. S.F. Ahmed: Grant Recipient; Self; Novo Nordisk. Objective 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 and their frequency for routine clinical practice is unclear. The objective of this study was to identify a minimum dataset (MDS) to be captured in routine clinical settings for children receiving all forms of rhGH therapy for GH deficiency (GHD). Methods This exercise was undertaken through the collaborative efforts of a group of experts who form the GH Scientific Study Group (SSG) in GloBE-Reg, a new international registry platform developed in close collaboration with clinicians, patients and industry and which can support long-term safety and effectiveness studies for drugs that require such surveillance. Twelve clinical experts from 7 international endocrine organisations identified by the GloBE-Reg Steering Committee, two patient advocates and representatives from two industry partners with previous GH registry expertise developed a comprehensive list of data fields routinely collected for children with GHD. Members graded the: (1) Importance of the field and (2) Ease of data collection. Fields that achieved a minimum of 70% consensus for importance qualified for the MDS, provided <50% deemed the item difficult to collect. Results Of 225 items graded, 113 (50%) items achieved at least 70% consensus as important to collect. Of the 225 items, 69 (31%) were deemed easy to collect and combining the criteria of importance and ease of data collection, 64 (28%) met both criteria for the MDS. Some other items including patient involvement in clinical trials, HbA1c in long-acting rhGH therapy and adherence did not meet the MDS criteria but were considered to be important enough to be included and some of the items were merged into one item. The final MDS consisted of 43 items; 20 to be entered once, 14 every 6 months and 9 every 12 months. Conclusions In summary, this exercise formulated the minimum dataset for the GloBE-Reg GH module which was launched in October 2022 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. Additional modules are being developed for specific GH products and other therapies for growth disorders. More information about the study and instructions on how to participate are available on the project’s website: www.globe-reg.net. Presentation: Thursday, June 15, 2023