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

ABSTRACT 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.