Efficacy and Safety of PEGylated Recombinant Human Growth Hormone in Children With Growth Hormone Deficiency and Idiopathic Short Stature: A Real‐World Cohort Study

Background Growth hormone deficiency (GHD) and idiopathic short stature (ISS) are common causes of short stature in children. In China, PEGylated recombinant human GH (PEG‐rhGH, Jintrolong) has been approved for the treatment of both conditions. This study aimed to evaluate the efficacy and safety of PEG‐rhGH in children diagnosed with GHD or ISS and to compare clinical outcomes between the two groups. Methods This real‐world study included 91 treatment‐naïve children with short stature at Kunming Children’s Hospital between 2020 and 2021. Participants were categorized into the GHD group (n = 39) and the ISS group (n = 52) based on etiological diagnosis. All subjects received weekly subcutaneous PEG‐rhGH injections at an initial dose of 0.20 mg/kg/wk and were followed for 18 months. Growth‐related parameters were assessed throughout the study. Results PEG‐rhGH treatment significantly improved height standard deviation score (Ht SDS) in both groups. In the GHD group, Ht SDS increased from −3.14 (−4.06, −2.02) at baseline to −1.53 (−1.98, −1.08) at Month 18 (p < 0.001), with a mean ΔHt SDS of 1.69 ± 0.98. The ISS group demonstrated an improvement from −3.33 ± 1.23 at baseline to −1.33 (−2.03, −0.92) at 18 months (p < 0.001), with a mean ΔHt SDS of 1.77 ± 1.06. No significant differences were identified between the groups regarding Ht SDS, ΔHt SDS, Insulin‐Like Growth Factor 1 SDS (IGF‐1 SDS), ΔIGF‐1 SDS, or height velocity (all p > 0.05). Thyroid function markers (T3, T4, FT3, FT4) and fasting plasma glucose levels remained within normal ranges throughout treatment, with no significant intergroup differences (all p > 0.05). No serious adverse events were observed. Conclusion PEG‐rhGH effectively promoted height gain in children with GHD and ISS, with similar therapeutic efficacy in both groups. However, children with ISS required a longer duration to achieve catch‐up growth to normal height, potentially due to reduced GH sensitivity and a need for higher dosing. PEG‐rhGH was well tolerated, with a favorable safety profile in both cohorts.