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Long-Term Efficacy and Safety of Growth Hormone in Children Suffering from Short Stature in China (CGLS): An
1Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, China.
Insights
This 16-year real-world study evaluates the long-term safety and efficacy of pegylated recombinant human growth hormone (PEG-rhGH) and recombinant human growth hormone (rhGH) in treating childhood short stature (SS). Results will inform guidelines for pediatric growth hormone therapy.
Area of Science:
- Pediatric Endocrinology
- Pharmacology
- Clinical Research
Background:
- Childhood short stature (SS) stems from various growth-disrupting disorders.
- Pegylated recombinant human growth hormone (PEG-rhGH) offers long-acting treatment for pediatric growth hormone deficiency.
- Limited real-world data exists on PEG-rhGH's extensive use and long-term outcomes in diverse pediatric SS populations.
Purpose of the Study:
- To evaluate the long-term safety and efficacy of PEG-rhGH and recombinant human growth hormone (rhGH) in treating childhood SS.
- To assess adverse events and treatment effectiveness across various etiologies of SS.
- To provide real-world evidence for developing national guidelines on growth hormone therapy in China.
Main Methods:
- An open-label, multicenter, observational study (16-year duration).
- Recruitment of 10,000 Chinese children (≥2 years) with SS across retrospective, retrospective-prospective, and prospective cohorts.
- Primary objective: long-term safety (AEs, SAEs); Secondary objective: efficacy in SS patients with GHD, ISS, SGA, TS, PWS, NS, SHOX deficiency, and other causes.
Main Results:
- Data analysis from 10,000 patients will provide insights into treatment patterns.
- Evaluation of long-term safety profiles of PEG-rhGH and rhGH.
- Assessment of treatment efficacy across diverse pediatric short stature conditions.
Conclusions:
- The study will generate crucial long-term efficacy and safety data for PEG-rhGH and rhGH in childhood SS.
- Findings will serve as evidence for the Chinese academic community to establish national guidelines.
- This research addresses a critical need for real-world data on growth hormone therapy in pediatric populations.
Introduction:
Several primary and secondary disorders disrupting normal growth pattern are responsible for childhood short stature (SS; height less than 2 standard deviation score [SDS] or the third percentile). Pegylated recombinant human growth hormone (PEG-rhGH) is a long-acting growth hormone which has demonstrated efficacy and safety in pediatric growth hormone deficiency. However, limited data is present on its treatment pattern, extensive population use, and long-term follow-up. Therefore, a real-world study is required to evaluate the efficacy and safety of PEG-rhGH and recombinant human growth hormone (rhGH) in treating childhood SS.
Methods:
The proposed study will be an open-label, multicenter, prospective and retrospective, observational study that will recruit Chinese children aged ≥ 2 years with SS. The entire study will be categorized into three cohorts: retrospective, retrospective-prospective, and prospective. The study will recruit 10,000 patients including 3000 patients in the retrospective cohort and 7000 in the retrospective-prospective and prospective cohort, respectively. The total duration of this study will be 16 years. The primary objective will be to evaluate the long-term safety (incidence of all adverse events (AEs) and serious adverse events) of PEG-rhGH and rhGH for the treatment of patients with SS having growth hormone disorder (GHD), idiopathic short stature (ISS), small for gestational age (SGA), Turner syndrome (TS), Prader-Willi syndrome (PWS), Noonan syndrome (NS), deficiency of the short stature homeobox gene on the X-chromosome (SHOX deficiency), and other causes of SS. The secondary objective will be to evaluate the efficacy of PEG-rhGH and rhGH for the treatment of patients with SS with different etiologies.
Planned Outcomes:
The results may provide the evidence of long-term efficacy and safety of PEG-rhGH and rhGH by analyzing the existing patient data and will also provide a vast array of information, which can be used as reference evidence for the Chinese academic community to design national guidelines or consensus for patients with SS.
Trial Registration:
The study has been registered at ClinicalTrials.gov (NCT06110910). Date of registration October 31, 2023.
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