Use of PEGylated Recombinant Human Growth Hormone in Chinese Children with Growth Hormone Deficiency: A 24-Month Follow-Up Study

Abstract

Objective. Once-weekly PEGylated recombinant human growth hormone (rhGH) is the sole long-acting GH formulation available currently for pediatric patients with GH deficiency (GHD). The aim of this study was to evaluate the efficacy and safety of PEGylated rhGH therapy compared to daily rhGH therapy in GHD children treated for two years. Methods. A total of 98 children (49 children for the PEGylated rhGH group and 49 children for the daily rhGH group) with GHD were enrolled in this single-center, prospective, nonrandomized cohort study. PEGylated rhGH or daily rhGH was administered for 2 years. Height, height SDS, height velocity (HV), IGF-1, bone age (BA), and adverse events were determined throughout the treatment. Results. HV significantly increased over the baseline and was similar in both groups. In the PEGylated rhGH cohort, the mean ± SD HV was improved from 3.78 ± 0.78 cm/y at the baseline to 12.44 ± 3.80 cm/y at month 3, to 11.50 ± 3.01 cm/y at month 6, to 11.00 ± 2.32 cm/y at month 12, and finally 10.08 ± 2.12 cm/y at month 24 in the PEGylated rhGH group. In the daily rhGH group, HV was 3.36 ± 1.00 cm/y at baseline, increasing to 12.56 ± 3.71 cm/y at month 3, to 11.82 ± 2.63 cm/y at month 6, to 10.46 ± 1.78 cm/y at month 12, and to 9.28 ± 1.22 cm/y at month 24. No serious adverse event related to PEGylated rhGH or daily rhGH occurred during the 24-month study. Conclusion. PEGylated rhGH replacement therapy is effective and safe in pediatric patients with GHD. The adherence to once-weekly PEGylated rhGH therapy is superior to daily rhGH in children with GHD.

RETRACTED: Effect of Weekly Long-Acting Growth Hormone Replacement Therapy Compared to Daily Growth Hormone on Children With Short Stature: A Meta-Analysis

“…A total of 1,245 unique studies were identified, of which 11 studies (between 2012 and 2021) fulfilled the inclusion criteria and were included in the study (25)(26)(27)(28)(29)(30)(31)(32)(33)(34)(35).…”

“…However, long-acting growth hormone replacement therapy had no significant difference in height velocity, height standard deviation scores bone age, insulin-like growth factor 1 standard deviation scores, and incidence of adverse events compared to daily growth hormone in children with short stature (25)(26)(27)(28)(29)(30)(31)(32)(33)(34)(35). However, the analysis of outcomes should be done with caution because of the small sample size of most of the selected studies in our meta-analysis (eight studies out of 11 <100 subjects); proposing the need for more studies to confirm these results or possibly to significantly affects confidence in the outcome assessment, e.g., height standard deviation scores bone age comparison with its relatively low p-value = 0.16.…”

“…This meta-analysis study based on 11 studies included 1,232 children with short stature treated with growth hormone replacement therapy at the start of the study; 737 of them were using weekly long-acting growth hormone replacement therapy and 495 were using daily growth hormone (25)(26)(27)(28)(29)(30)(31)(32)(33)(34)(35).…”

“…These adverse events were generally mild, and none of the adverse events caused subject withdrawal. The most frequently described adverse events in subjects taking long-acting growth hormone therapy were injection site pruritus, anemia, hypothyroidism, pyrexia, rash, headache, and vomiting (25)(26)(27)(28)(29)(30)(31)(32)(33)(34)(35).…”

PEGylated Recombinant Human Growth Hormone Jintrolong® Exhibits Good Long-Term Safety in Cynomolgus Monkeys and Human Pediatric Growth Hormone Deficiency Patients

“…Pediatric GHD patients receive years of daily rhGH subcutaneous (SC) injections since the serum half-life of rhGH is only 3.4 h (6,7). Approximately 23% of teenage patients miss two or more injections per week because daily injections are distressing and inconvenient, resulting in poor compliance and treatment outcomes (8). To improve patient compliance, several long-acting rhGH preparations have been developed recently.…”