International journal of endocrinology · 2026
Efficacy and Safety of PEGylated Recombinant Human Growth Hormone in Children With Growth Hormone Deficiency and Idiopathic Short Stature: A Real-World Cohort Study.
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Ge Liping, Zhao Canmiao, Yang Yang, Xu Lijun, Sun Meiyuan, Su Yanfang, Xu Fang, Huang Qi, Tao Na
Аннотация
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.
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.