Model predicts growth hormone therapy responses in children with short stature
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Idiopathic short stature (ISS) describes children whose height is substantially below the expected range for their age and sex without any identifiable medical cause. Recombinant growth hormone (GH) therapy is often used to support growth, but responses vary widely. While some children experience meaningful catch-up growth, others show more limited improvement, making it difficult for clinicians and families to anticipate treatment benefits and set realistic expectations.
To address this challenge, researchers led by professor Jung-woo Chae investigated changes in height percentile over time among children with ISS receiving GH treatment.
Published in Value in Health, the study used age- and sex-adjusted height percentiles rather than height gain in centimeters alone. This approach captures changes in height percentile relative to peers of the same age and sex, providing a clinically intuitive measure of catch-up growth.
The retrospective, single-center study analyzed 91 prepubertal Korean children with ISS—41 boys and 50 girls—who received recombinant human GH (somatropin) between July 2020 and December 2023. GH was administered subcutaneously six to seven times per week, with doses adjusted during routine care.
Researchers collected demographic, growth, parental height, laboratory and treatment data from medical records. Cumulative GH exposure was incorporated into a Gompertz nonlinear mixed-effects model to characterize growth trajectories and differences in individual treatment response.
Over a mean treatment period of 619 ± 307 days, the mean height percentile increased from 1.26 at baseline to 9.16 at follow-up. The model estimated a total growth-response parameter of approximately 17.2 percentile points, although responses differed substantially among patients.
Chae explains, "Three baseline characteristics were associated with a greater predicted response: higher body mass index (BMI), lower insulin-like growth factor-binding protein 3 (IGFBP-3) levels and shorter paternal height, with BMI showing the strongest association."
Two-year simulations further illustrated how predicted outcomes could differ by patient profile and GH-exposure regimen. Median predicted height percentiles ranged from 4.0% in lower-response profiles to 31.0% in higher-response profiles, depending on BMI, IGFBP-3, paternal height and GH exposure.
These findings underscore the potential value of considering individual characteristics when assessing the expected response to GH therapy.
The team also developed GrowCast, a web-based tool that generates individualized predicted height and percentile trajectories using age, sex, height, weight, paternal height, IGFBP-3 level and GH dose.
Chae says, "GrowCast enables clinicians to compare modeled treatment scenarios and communicate potential growth trajectories with patients and families."
The platform may support clinician–family discussions and treatment planning, but it is not yet a validated dosing-prescription tool. Larger prospective, multicenter studies are needed before routine clinical implementation.
Overall, the study offers a promising framework for more personalized GH treatment in ISS. By combining baseline characteristics with cumulative GH exposure, the model may help clinicians estimate individual growth trajectories and support more informed treatment planning.
More information
Hyojin Cho et al, Longitudinal Modeling and Simulation of Growth Hormone Efficacy in Children With Idiopathic Short Stature, Value in Health (2026). DOI: 10.1016/j.jval.2026.05.006
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