Long-Term Efficacy and Safety of Growth Hormone in Children Suffering from Short Stature in China (CGLS): An Open-Label, Multicenter, Prospective and Retrospective, Observational Study.
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引用次数: 0
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.
期刊介绍：
Advances in Therapy is an international, peer reviewed, rapid-publication (peer review in 2 weeks, published 3–4 weeks from acceptance) journal dedicated to the publication of high-quality clinical (all phases), observational, real-world, and health outcomes research around the discovery, development, and use of therapeutics and interventions (including devices) across all therapeutic areas. Studies relating to diagnostics and diagnosis, pharmacoeconomics, public health, epidemiology, quality of life, and patient care, management, and education are also encouraged.
The journal is of interest to a broad audience of healthcare professionals and publishes original research, reviews, communications and letters. The journal is read by a global audience and receives submissions from all over the world. Advances in Therapy will consider all scientifically sound research be it positive, confirmatory or negative data. Submissions are welcomed whether they relate to an international and/or a country-specific audience, something that is crucially important when researchers are trying to target more specific patient populations. This inclusive approach allows the journal to assist in the dissemination of all scientifically and ethically sound research.
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