ESPE2023 Poster Category 1 Growth and Syndromes (75 abstracts)
A systematic review of core outcomes reported in clinical trials of growth hormone therapy in children with growth hormone deficiency
Xanthippi Tseretopoulou 1,2 , Angela Lucas-Herald 1,2 , Jiajia Chen 3 , Tania Bachega 4 , Evangelia Charmandari 5 , Jin-Ho Choi 6 , Xinyu Dou 3 , Chunxiu Gong 3 , Rasha Hamza 7 , Jamie Harvey 8 , Andrew R. Hoffman 9 , Reiko Horikawa 10 , Gudmundur Johannsson 11 , Alexander Jorge 12 , Bradley S. Miller 13 , Sebastian Roehrich 14 , Lars Sävendahl 15 , Diana Vitali 16 , Michael Wajnrajch 17 , Suet Ching Chen 1,2 & Syed Faisal Ahmed 1,2
1Developmental Endocrinology Research Group, University of Glasgow, Royal Hospital for Children, Glasgow, UK, Glasgow, United Kingdom. 2Office for Rare Conditions, University of Glasgow, Glasgow, UK, Glasgow, United Kingdom. 3Beijing Children’s Hospital, the Capital Medical University, National Center for Children’s Health, Beijing, China, Beijing, China. 4Unidade de Endocrinologia do Desenvolvimento, Laboratório de Hormônios e Genética Molecular/LIM42, Disciplina de Endocrinologia, Hospital Das Clinicas, Faculdade De Medicina, Universidade de Sao Paulo, São Paulo, Brazil, Sao Paulo, Brazil. 5Division of Endocrinology, Metabolism and Diabetes, First Department of Pediatrics, National and Kapodistrian University of Athens Medical School, ‘Aghia Sophia’ Children’s Hospital, Athens, 11527, Greece, Athens, Greece. 6University of Ulsan College of Medicine, Seoul, Korea, Seoul, Korea, Republic of. 7Department of Pediatrics, Pediatric Endocrinology Unit, Ain Shams University, Cairo, Egypt, Cairo, Egypt. 8MAGIC Foundation, Illinois, USA, Illinois, USA. 9Department of Medicine, Division of Endocrinology, Metabolism and Gerontology, Stanford University School of Medicine, Stanford, California, USA, California, USA. 10Division of Endocrinology and Metabolism, National Center for Child Health and Development, Tokyo, Japan, Tokyo, Japan. 11Institute of Medicine, Sahlgrenska Academy, University of Gothenburg, Dept. Endocrinology, Sahlgrenska University Hospital, Gothenburg, Sweden, Gothenburg, Sweden. 12Genetic-Endocrinology Unit, Endocrinology Division of Hospital das Clinicas of University of Sao Paulo School of Medicine, Sao Paulo, Brazil, Sao Paulo, Brazil. 13University of Minnesota Medical School, MHealth Fairview Masonic Children’s Hospital, Minneapolis, Minnesota, USA, Minneapolis, USA. 14Novo Nordisk Health Care AG, Global Medical Affairs Rare Endocrine Disorders, Zurich, Switzerland, Zurich, Switzerland. 15Department of Women´s and Children´s Health, Karolinska Institutet and Karolinska University Hospital, Stockholm, Sweden, Stockholm, Sweden. 16SOD Italia, Rome, Italy, Rome, Italy. 17Pfizer BioPharmaceuticals, Rare Disease, Department of Pediatrics, New York University Langone Medical Center, New York, NY, USA, New York, USA
Introduction: Although the safety and effectiveness of recombinant human growth hormone therapy (rhGH) has been reported for several years, the level of consensus on the outcomes that should be reported is unclear. The aim of this systematic review is to study the frequency of reporting of these outcomes in children with GH deficiency (GHD).
Methods: A systematic review was performed in Medline, Embase, Cochrane Central Register of Controlled Trials (CENTRAL), Cochrane Reviews, WHO International Clinical Trials Registry Platform (ICTRP) portal, ClinicalTrials.gov, Wan Fang and Chinese Medical Journal between January-February 2023. Eligibility criteria included all studies published between 2013 and 2023, with participants who started rhGH before the age of 16 years for GHD; cross-sectional and case reports were excluded. These criteria were met in 109 studies, of which 77 were in English, 29 in Chinese and 3 in Spanish.
Results: Of the 109 studies, 81(74%) were cohort studies, 25 (23%) were controlled trials and 3 (3%) case-control studies. Studies originated from six continents, and 27 (25%) were multinational. The median age of the participants at start of the study was 9 years (10th,90th: 6.8,10.8 years). Frequently reported outcomes in the 109 studies included: change in height velocity in 56 (51%), change in height SDS in 53 (49%), IGF-1 in 38 (35%), IGF-1 SDS in 30 (28%), height in 29 (27%), bone age and injection site adverse events in 21 (19%), weight in 19 (17%), IGFBP-3 in 16 (15%) and headache in 15 (14%). The most frequently reported frequency of measurement of the above outcomes was annually, varying from 34% (10/29 studies) for height to 56% (15/27 studies) for IGF-1 SDS, with the exception of IGF-1 and IGFBP-3 which was 6-monthly (13/36 studies, 36% and 7/14, 50% respectively). Of the 276 different outcomes reported in total, 144 (52%) were on efficacy, 122 (44%) on safety and 10 (4%) on quality of life. The 144 effectiveness outcomes consisted of 88 (61%) biochemical, 19 (13%) auxological, 13 (9%) radiological, 5 (4%) body composition and 19 (13%) other clinical outcomes.
Conclusions: The current systematic review identifies the range of outcomes that are used to assess the safety and effectiveness of rhGH therapy in childhood GHD. These results demonstrate a level of consensus regarding which outcomes should be tracked and can be used to promote the development of a core outcome set that can standardise the routine collection of outcomes in a clinical setting.
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