#South Korea #Daejeon #Chungnam National University #Pediatric Research #Growth Hormone

New Predictive Model for Growth Hormone Therapy

Researchers at Chungnam National University have developed an advanced predictive model aimed at improving the treatment regimen for children suffering from idiopathic short stature (ISS). This condition is characterized by a height considerably lower than expected for a child's age and sex, with no identifiable medical cause. The conventional treatment for this issue is recombinant growth hormone therapy (GH), which has shown varying results. Some children experience significant growth while others do not respond as positively, leading to uncertainty for parents and clinicians about setting realistic treatment expectations.

The model was created by analyzing height percentile changes in children undergoing GH treatment. Unlike many previous studies, this research, published online in the journal Value in Health on July 21, 2026, utilized age and sex-adjusted height percentiles to provide a clearer understanding of growth in relation to peers. This method offers a more clinically intuitive measure of growth, focusing on percentile changes rather than just height gain in centimeters.

In the study, 91 prepubertal Korean children with ISS participated, of which 41 were boys and 50 were girls. They were treated with recombinant human GH (somatropin) from July 2020 to December 2023, with therapy involving injections six to seven times per week. The research team, led by Professor Jung-woo Chae, gathered data from medical records covering demographic information, growth progression, parental heights, and laboratory results. This data was utilized within a Gompertz nonlinear mixed-effects model, effectively characterizing both growth trajectories and differences in individual responses to the treatment.

Key Findings

Over an average treatment period of 619 ± 307 days, the study observed that the average height percentile of the children improved from 1.26 at the start of treatment to 9.16 at follow-up. The predictive model estimated an overall growth-response parameter of about 17.2 percentile points, although there was significant variation in individual responses. Notably, three baseline factors were linked to a more substantial predicted response: a higher body mass index (BMI), lower levels of insulin-like growth factor-binding protein 3 (IGFBP-3), and shorter paternal height. Among these, BMI showed the most significant correlation with treatment outcomes.

Further simulations over two years highlighted how predicted outcomes could vary based on individual characteristics and GH exposure regimens. The median predicted height percentiles ranged widely, from 4.0% in lower-response profiles to 31.0% in those with higher expected responses. This variation emphasizes the importance of personalizing treatment based on individual patient profiles.

To aid clinicians in applying this model, the research team developed GrowCast, an online tool capable of generating personalized height and percentile trajectories by inputting age, sex, height, weight, paternal height, IGFBP-3 levels, and GH dosage. Professor Chae explains that GrowCast serves as a valuable resource for clinicians, allowing them to simulate various treatment scenarios and effectively communicate growth expectations with families. However, it is crucial to note that this platform is still in its early stages and should not yet be considered a validated dosing or prescription tool. Additional research through larger, multicenter studies is needed before the model can be routinely implemented in clinical settings.

The development of this predictive modeling approach represents a significant step towards moving growth hormone treatment from an often arbitrary trial-and-error process to a more personalized, evidence-based practice. By accounting for both baseline characteristics and cumulative GH exposure, this model not only enhances the potential for improving individual growth outcomes but also provides a framework for better-informed treatment planning in pediatric endocrinology.

References

Original Article: Longitudinal Modeling and Simulation of Growth Hormone Efficacy in Children With Idiopathic Short Stature published in the journal Value in Health. DOI: 10.1016/j.jval.2026.05.006