Trends In Reported Outcomes For Growth Hormone Therapy In Children With Growth Hormone Deficiency

To understand the frequency and trends in reported outcomes of safety and effectiveness for recombinant human growth hormone (rhGH) therapy for growth hormone deficiency (GHD) in childhood. A systematic review was performed in seven English and Chinese language databases. Eligibility criteria included all studies published between 2003 and 2022, with participants who started rhGH before the age of 16 years for GHD. The 219 eligible studies that were identified included 171 cohort studies, 39 controlled trials and 9 case-control studies. The median age of the participants at start of rhGH was 9.5yrs (10th, 90th: 7.1, 11.9). The most commonly reported outcomes included change in height SDS in 117 (53%), height velocity in 105 (48%), IGF-1 in 66 (30%), height in 63 (29%), bone age in 58 (26%), IGF-1 SDS in 45 (21%), injection site adverse events in 44 (20%), glucose concentration in 42 (19%), IGFBP-3 in 34 (16%) and thyroid function in 30 (14%). Of the 342 different outcomes, 178 (52%) were considered as outcomes that were mainly focused on safety, 94 (28%) on efficacy, and 70 (20%) on both. Over the two decades, height SDS and height velocity remained the most frequently reported outcomes. Of the 342 outcomes, 9 (3%) were on quality of life and these were reported in 29 of 219 (13%) studies. The results of this systematic review allow the development of a core outcome set that is recommended for routine use in all children receiving rhGH for childhood GHD.