Abstract
This systematic review aims to summarize the therapeutic benefits and safety profile of long-acting PEGylated recombinant human growth hormone (PEG‑rhGH) compared with daily recombinant human growth hormone (DGH) in pediatric growth hormone deficiency (PGHD) participants. We conducted an extensive literature review utilizing multiple databases, and evaluated change in height standard deviation score (∆Ht-SDS) as the primary outcome. Secondary outcomes included change in height velocity (∆HV) and incidence of total adverse events (AEs). The meta-analysis focused on comparing the standard dose of PEG‑rhGH (0.20 mg/kg/w) and DGH. The systematic review encompassed ten studies involving a total of 1,393 participants. In RCTs and cohort studies, ∆Ht-SDS at 6 months showed no significant difference between PEG-rhGH and DGH (RCTs: MD = 0.02, 95%CI: -0.02 to 0.07, p = 0.32; Cohort studies: MD = -0.02, 95%CI: -0.24 to 0.19, p = 0.82). However, PEG-rhGH had superior ∆Ht-SDS (MD = 0.19, 95%CI: 0.03 to 0.35, p = 0.02) at 12 months. Results from RCTs also showed that the incidence of total AEs was comparable for PEG-rhGH and DGH (OR = 1.12, 95%CI: 0.84 to 1.49, p = 0.45). PEG-rhGH showed superior efficacy to DGH at 12 months and comparable efficacy at other time points. The safety profiles were similar for the two treatments.