Evaluating the effectiveness and safety of recombinant human growth hormone therapy in children with idiopathic short stature across Gulf Cooperation Council countries: a systematic review

Idiopathic short stature (ISS) is a heterogeneous growth disorder characterized by height below − 2 standard deviations from the population mean for age and sex in the absence of identifiable pathological causes. Recombinant human growth hormone (rhGH) has been approved for ISS treatment in several countries; however, region-specific evidence from Gulf Cooperation Council (GCC) countries remains sparse. This systematic review was conducted in accordance with PRISMA 2020 guidelines to evaluate the effectiveness and safety of rhGH therapy in children with ISS across GCC countries. Six information sources were searched for peer-reviewed studies published between January 2015 and July 2025. Study selection, data extraction, and quality appraisal were performed independently by two reviewers. Due to substantial clinical and methodological heterogeneity across the included studies, data were synthesized narratively. Seven observational studies enrolling a combined ISS cohort of 301 children from the UAE, Saudi Arabia, Qatar, and Kuwait were included. All studies reported positive growth responses to rhGH therapy, measured predominantly by change in height standard deviation score (ΔHtSDS). First-year ΔHtSDS ranged from + 0.17 (median, IQR 0.16–0.41) to + 0.58 ± 0.31, with most studies reporting gains of + 0.3 to + 0.5 SDS. Longer treatment duration was associated with greater cumulative gains, with some cohorts exceeding + 1.0 SDS at final adult height (FAH). Reported adverse events were predominantly mild; no serious adverse events were reported, although adverse-event reporting was incomplete. rhGH therapy is associated with consistent, though modest and variable, improvements in linear growth in children with ISS across GCC countries. Growth benefit accumulates with continued therapy. Standardized prospective multicenter studies within the GCC region are required to strengthen the evidence base and improve cross-study comparability.