Abstract
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.