Objective:

To compare the efficacy and safety of long-acting human growth hormone (LAGH) preparations with conventional short-acting daily growth hormone (SA-GH) in children with isolated growth hormone deficiency (GHD), while exploring potential differences across four distinct LAGH platforms through subgroup analyses.

Key Findings:

• LAGH resulted in a statistically significant improvement in first-year Ht-SDS (MD: 0.08; 95% CrI: 0.04–0.11) across 16 RCTs involving 2,435 children.
• LAGH showed superior HV-SDS compared with SA-GH, driven by the PEG-LAGH subgroup (MD: 4.35; 95% CrI: 3.78–4.92).
• IGF-1 SDS was consistently higher in the LAGH group (MD: 0.51; 95% CrI: 0.21–0.80).
• AE rates did not differ significantly between groups; PEG-LAGH subgroup had the lowest AE incidence (31.1%).

Interpretation:

Among children with isolated GHD, LAGH offers superior first-year height velocity and acceptable safety compared to SA-GH, suggesting a preferred treatment option in clinical practice.

Limitations:

• The study may have limitations related to the variability in molecular structure and pharmacokinetics among different LAGH platforms, as well as potential biases such as selection bias and reporting bias in included studies.

Conclusion:

LAGH is preferred for treating children with isolated GHD due to its efficacy in height velocity and safety profile.