Efficacy, adherence, and cost-efficiency of three growth hormone treatment strategies in children with idiopathic short stature: a retrospective cohort study

Abstract Objectives The study compares the efficacy, adherence, and cost-effectiveness of three recombinant human growth hormone (rhGH) regimens in children with idiopathic short stature (ISS) to optimize treatments. Methods This retrospective cohort study involved 105 children with ISS who were divided into three groups: short-acting rhGH for 48 weeks (Group 1), PEG-rhGH for 48 weeks (Group 2), and sequential therapy (PEG-rhGH for 24 weeks + short-acting rhGH for 24 weeks) (Group 3). The primary endpoints were the annual changes in the height standard deviation score (ΔHtSDS) and height velocity (HV). Adherence was measured by the missed injection rate (%), and the cost-effectiveness analysis employed the cost-effectiveness ratio. Results After one year, all regimens improved HtSDS and HV (p<0.05), with Groups 2 and 3 showing better HV and ΔHtSDS than Group 1. Group 2 had the highest adherence (0 % missed rate), followed by Group 3 (0.14 %) and Group 1 (0.27 %). Missed injections were the only significant predictor of ΔHtSDS (β=−0.196, p=0.048). Financially, Group 3 had lower costs (¥64,730) and a better cost-effectiveness ratio than Group 2 (¥67,479). No serious adverse events occurred. Conclusions Sequential therapy offers efficacy and compliance benefits for long-acting rhGH while lowering costs, thus making it suitable for ISS treatment. Long-acting therapy is ideal for individuals with the financial means to support such treatment, whereas short-acting therapy is an option for individuals facing budgetary constraints. Future research should include longer follow-up and quality-of-life (QoL) assessments to improve the cost-benefit analysis.