Metabolic and bone effects of long-acting polyethylene glycol-recombinant human growth hormone in growth hormone deficiency during the transition period: a prospective study

Purpose: Polyethylene glycol-recombinant human growth hormone (PEG-rhGH) is the first commercial long-acting rhGH preparation approved in China. This study was designed to assess the efficacy and safety of PEG-rhGH in treating growth hormone deficiency (GHD) during the transition period.Methods: A prospective, multicenter and single-arm study was performed. The entire study duration spanned 66 weeks, comprising a 2-week trial screening phase, a 12-week dose-adjustment period, and a 52-week PEG-rhGH treatment phase. A total of 10 interviews were conducted over the course of the study. The primary endpoint was change in body composition; secondary endpoints were changes in lumbar bone mineral density (BMD, L1–4) and lean body mass (LBM). Sex- and age-specific equations were developed to adjust body composition z-scores.Results: : A total of 31 subjects were included in this study. Throughout the 64-week therapy with PEG-rhGH, the insulin-like growth factor-1 (IGF-1) standard deviation score (SDS) demonstrated progressive elevation from baseline (-3.07 to -1.38; P<0.001). Significant alterations in body composition parameters were observed throughout the study. Body weight increased 3.96 kg (P<0.001), and the body weight SDS exhibited significant elevation from baseline (0.67) to 64 weeks (1.14; P=0.005). LBM significantly increased (4.54 kg; P<0.001), but there was a statistically significant reduction in fat percentage (Fat%) from 37.40% to 33.94% (P=0.008) and Fat% SDS from 1.91 to 1.47 (P= 0.009). The lumbar BMD z-score improved from -1.54 at baseline to -1.12 postintervention, but did not reach statistical significance (P>0.05). The triglyceride (TG) levels of the subjects decreased significantly at week 64 (P=0.018). There was no statistically significant difference in the changes of other lipid profile indicators. In this study, no adverse events attributable to direct drug-related causes were identified.Conclusion: Based on our study, PEG-rhGH can improve IGF-1 levels and alter body composition parameters by increasing LBM and decreasing body fat and TG levels in patients with GHD during the transition period, and it has a comparable safety profile and helps improve therapeutic adherence.