Near-Adult Height Outcomes in Patients Treated With rhIGF-1 for Severe Growth Failure: Real-World IGFD Registry Data.
Por:
Ramon-Krauel M, Polak M, Maghnie M, Woelfle J, Sert C, Perrot V and Bang P
Publicada:
21 ene 2026
Ahead of Print:
1 jul 2025
Resumen:
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. TRIAL REGISTRATION: NCT00903110.
Filiaciones:
Ramon-Krauel M:
Hospital Sant Joan de Déu, Institut de Recerca Sant Joan de Déu (IRSJD), Barcelona, Spain
Centro de Investigación Biomédica en Red de Diabetes y Enfermedades Metabólicas Asociadas (CIBERDEM), Instituto de Salud Carlos III, Madrid, Spain
Endo-ERN European Reference Network on Rare Endocrine Conditions, Amsterdam, The Netherlands
Polak M:
Department of Pediatric Endocrinology, Gynaecology, and Diabetology, Assistance Publique - Hôpitaux de Paris, Hôpital Universitaire Necker-Enfants Malades, IMAGINE Institute, INSERM U1016, Université Paris Cité, Paris, France
Maghnie M:
Endo-ERN European Reference Network on Rare Endocrine Conditions, Amsterdam, The Netherlands
Department of Pediatrics, IRCCS Istituto Giannina Gaslini, Genova, Italy
Department of Neuroscience, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health, University of Genova, Genova, Italy
Woelfle J:
Department of Pediatrics and Adolescent Medicine, Friedrich-Alexander-University (FAU) Erlangen-Nürnberg, Erlangen, Germany
Sert C:
Ipsen, Boulogne-Billancourt, France
Perrot V:
Ipsen, Boulogne-Billancourt, France
Bang P:
Division of Pediatrics, Department of Biomedical and Clinical Sciences, Faculty of Health Sciences, Linköping University, Linköping, Sweden
Green Submitted, hybrid
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