Near-Adult Height Outcomes in Patients Treated With rhIGF-1 for Severe Growth Failure: Real-World IGFD Registry Data
Ramon-Krauel M, Polak M, Maghnie M, Wölfle J, Sert C, Perrot V, Bang P (2026)
Publication Type: Journal article
Publication year: 2026
Journal
Book Volume: 111
Pages Range: e500-e511
Journal Issue: 2
Abstract
Context: The Global Increlex® Growth Forum Database (IGFD) Registry monitors real-world effectiveness and safety of recombinant human IGF-1 (rhIGF-1; Increlex® [mecasermin]) treatment in children and adolescents with severe growth failure due to severe primary IGF-I deficiency (SPIGFD). Objective: To report characteristics, effectiveness, and safety data from patients receiving rhIGF-1 treatment who achieved near-adult height (NAH), and determine factors that predict height gain to NAH. Methods: Descriptive analyses of patients included in the Global IGFD Registry (NCT00903110) who achieved NAH are reported for the overall population, treatment-naïve prepubertal (NPP) patients, and patients with Laron syndrome. Linear regression analyses of height gain to NAH are also reported. Results: One hundred and two patients enrolled in the Global IGFD Registry achieved NAH at data cut-off (April 20, 2023). Mean age at rhIGF-1 treatment initiation was 11.8 years; median treatment duration was 3.9 years. Mean (SD) height SD score (HtSDS) gain from rhIGF-1 initiation to NAH was 0.9 (1.1). In NPP patients, mean (SD) HtSDS gain was 1.4 (1.0). Almost half of NPP patients reached NAH within the normal range. Despite improved height in patients with Laron syndrome, only 10.5% reached NAH within the normal range; 3 patients with Laron syndrome were NPP. Treatment naivety was predictive of height gain in the overall NAH population. Safety data aligned with previous reports. Conclusion: In a real-world setting, despite patients with SPIGFD initiating rhIGF-1 treatment at a relatively advanced age, rhIGF-1 treatment resulted in improved NAH. The greatest improvements in height outcomes were observed in NPP patients.
Authors with CRIS profile
Involved external institutions
Hospital Sant Joan de Déu Barcelona
Spain (ES)
Ipsen Pharma
France (FR)
Hôpital Necker-Enfants malades
France (FR)
European Reference Network on Rare Endocrine Conditions
Netherlands (NL)
Linköpings universitet / Linköping University
Sweden (SE)
Spain (ES)
Ipsen Pharma
France (FR)
Hôpital Necker-Enfants malades
France (FR)
European Reference Network on Rare Endocrine Conditions
Netherlands (NL)
Linköpings universitet / Linköping University
Sweden (SE)
How to cite
APA:
Ramon-Krauel, M., Polak, M., Maghnie, M., Wölfle, J., Sert, C., Perrot, V., & Bang, P. (2026). Near-Adult Height Outcomes in Patients Treated With rhIGF-1 for Severe Growth Failure: Real-World IGFD Registry Data. Journal of Clinical Endocrinology and Metabolism, 111(2), e500-e511. https://doi.org/10.1210/clinem/dgaf390
MLA:
Ramon-Krauel, Marta, et al. "Near-Adult Height Outcomes in Patients Treated With rhIGF-1 for Severe Growth Failure: Real-World IGFD Registry Data." Journal of Clinical Endocrinology and Metabolism 111.2 (2026): e500-e511.
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