Development of a Minimum Dataset for the Monitoring of Recombinant Human Growth Hormone Therapy in Children with Growth Hormone Deficiency: A GloBE-Reg Initiative.

Autor:	Chen SC; Developmental Endocrinology Research Group, University of Glasgow, Royal Hospital for Children, Glasgow, UK.; Office for Rare Conditions, University of Glasgow, Glasgow, UK., Bryce J; Office for Rare Conditions, University of Glasgow, Glasgow, UK., Chen M; Office for Rare Conditions, University of Glasgow, Glasgow, UK., Charmandari E; Division of Endocrinology, Metabolism and Diabetes, First Department of Pediatrics, National and Kapodistrian University of Athens Medical School, 'Aghia Sophia' Children's Hospital, Athens, Greece., Choi JH; Department of Pediatrics, Asan Medical Center Children's Hospital, University of Ulsan College of Medicine, Seoul, Republic of Korea., Dou X; Beijing Children's Hospital, The Capital Medical University, National Center for Children's Health, Beijing, China., Gong C; Beijing Children's Hospital, The Capital Medical University, National Center for Children's Health, Beijing, China., Hamza R; Department of Pediatrics, Pediatric Endocrinology Unit, Ain Shams University, Cairo, Egypt., Harvey J; MAGIC Foundation, Warrenville, Illinois, USA., Hoffman AR; Department of Medicine, Division of Endocrinology, Metabolism and Gerontology, Stanford University School of Medicine, Palo Alto, California, USA., Horikawa R; Division of Endocrinology and Metabolism, National Center for Child Health and Development, Tokyo, Japan., Johannson G; Institute of Medicine, Sahlgrenska Academy, University of Gothenburg, Department of Endocrinology, Sahlgrenska University Hospital, Gothenburg, Sweden., Jorge AAL; Genetic-Endocrinology Unit, Endocrinology Division of Hospital das Clinicas of University of Sao Paulo School of Medicine, Sao Paulo, Brazil., Miller BS; University of Minnesota Medical School, MHealth Fairview Masonic Children's Hospital, Minneapolis, Minnesota, USA., Roehrich S; Novo Nordisk Health Care AG, Global Medical Affairs Rare Endocrine Disorders, Zurich, Switzerland., Sävendahl L; Department of Women's and Children's Health, Karolinska Institutet and Karolinska University Hospital, Stockholm, Sweden., Tseretopoulou X; Developmental Endocrinology Research Group, University of Glasgow, Royal Hospital for Children, Glasgow, UK.; Office for Rare Conditions, University of Glasgow, Glasgow, UK., Vitali D; SOD Italia, Rome, Italy., Wajnrajch M; Pfizer Biopharmaceuticals, Global Medical Affairs, Rare Disease, New York, New York, USA.; Division of Paediatric Endocrinology, New York University Langone Medical Center, New York, New York, USA., Ahmed SF; Developmental Endocrinology Research Group, University of Glasgow, Royal Hospital for Children, Glasgow, UK.; Office for Rare Conditions, University of Glasgow, Glasgow, UK.
Jazyk:	angličtina
Zdroj:	Hormone research in paediatrics [Horm Res Paediatr] 2024; Vol. 97 (4), pp. 365-373. Date of Electronic Publication: 2023 Sep 13.
DOI:	10.1159/000533763
Abstrakt:	Introduction: Although there are some recommendations in the literature on the assessments that should be performed in children on recombinant human growth hormone (rhGH) therapy, the level of consensus on these measurements is not clear. The objective of the current study was to identify the minimum dataset (MDS) that could be measured in a routine clinical setting across the world, aiming to minimise burden on clinicians and improve quality of data collection. Methods: This study was undertaken by the growth hormone (GH) scientific study group in GloBE-Reg, a new project that has developed a common registry platform that can support long-term safety and effectiveness studies of drugs. Twelve 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 growth hormone deficiency (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 MDS, provided <50% deemed the item difficult to collect. Results: A total of 246 items were compiled and 27 were removed due to redundancies, with 219 items subjected to the grading system. Of the 219 items, 111 achieved at least 70% consensus as important data to collect when monitoring children with GHD on rhGH treatment. Sixty-nine of the 219 items were deemed easy to collect. Combining the criteria of importance and ease of data collection, 63 met the criteria for the MDS. Several anomalies to the MDS rule were identified and highlighted for discussion, including whether the patients were involved in current or previous clinical trials, need for HbA1c monitoring, other past medical history, and adherence, enabling formulation of the final MDS recommendation of 43 items; 20 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 MDS 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 the newer long-acting GH. (© 2023 The Author(s). Published by S. Karger AG, Basel.)
Databáze:	MEDLINE
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