Precision Dosing for Pediatric Growth Hormone Deficiency With Daily and Weekly Growth Hormone.

Pediatric growth hormone deficiency (GHD) typically requires daily injections of recombinant human growth hormone (rhGH). Long-acting PEGylated recombinant human growth hormone (PEG-rhGH) formulations have been developed to reduce injection frequency and improve adherence and quality of life. However, guidance on switching between short-acting and long-acting regimens during treatment and biomarker-driven precision dose-conversion strategies remains lacking in real-world clinical practice. Therefore, this study analyzed data from two populations comprising healthy adult male subjects (n = 39) and pediatric patients with GHD (n = 408). The analysis used population pharmacokinetic (PopPK) and population pharmacokinetic/pharmacodynamic (PopPK/PD) models. The models showed adequate fit and external predictability. Biomarker-clinical growth endpoint relationship analysis linked changes in insulin-like growth factor-1 standard deviation score (IGF-1 SDS) to growth outcomes, and simulations assessed subgroup responses, missed-dose scenarios, and IGF-1 SDS-guided switching. Simulations enabled subgroup-specific efficacy projections. Missed doses caused a greater loss of benefit with rhGH than with PEG-rhGH. Switching simulations supported IGF-1 SDS-guided conversion within accepted safety limits. The integrated framework provides concise, clinically interpretable rules for individualized titration, adherence-aware care, and safer switching in pediatric GHD. This study supports precision dosing, missed-dose management, and switching between daily rhGH and weekly PEG-rhGH in pediatric GHD.