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

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.

This work aims to understand the frequency and trends in reported outcomes of safety and effectiveness for rhGH therapy for growth hormone deficiency (GHD) in childhood.

A systematic review was performed in 7 English- and Chinese-language databases. Eligibility criteria included all studies published between 2003 and 2022, with participants who started rhGH before age 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 9.5 years (10th-90th: 7.1-11.9). The most commonly reported outcomes included change in height SDS in 117 (53%), height velocity in 105 (48%), insulin-like growth factor-1 (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%), insulin-like growth factor-binding protein 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.