Effectiveness and safety of rhIGF1 therapy in patients with or without Laron syndrome
Bang P, Wölfle J, Perrot V, Sert C, Polak M (2021)
Publication Type: Journal article
Publication year: 2021
Journal
Book Volume: 184
Pages Range: 267-276
Journal Issue: 2
DOI: 10.1530/EJE-20-0325
Abstract
Objective: The European Increlex® Growth Forum Database Registry monitors the effectiveness and safety of recombinant human insulin-like growth factor-1 (rhIGF1; mecasermin, Increlex®) therapy in patients with severe primary IGF1 deficiency (SPIGFD). We present data from patients with and without a reported genetic diagnosis of Laron syndrome (LS). Design: Ongoing, open-label, observational registry (NCT00903110). Methods: Children and adolescents receiving rhIGF1 therapy from 10 European countries were enrolled in 2008-2017 (n = 242). The treatment-naïve/prepubertal (NPP) cohort (n = 138) was divided into subgroups based on reported genetic diagnosis of LS (n = 21) or non-LS (n = 117). Multivariate analysis of the NPP-non-LS subgroup was conducted to identify factors predictive of growth response (first-year-height standard deviation score (SDS) gain ≥ 0.3). Assessments included change in height and weight over 5 years and adverse events (AEs). Results: Height SDS gain from baseline was greater in the NPP-LS than the NPP-non-LS subgroup after 1 years' treatment (P < 0.05). In the NPP-non-LS subgroup, 56% were responders; young age at baseline was a positive independent predictive factor (P < 0.001). NPP-non-LS-responders and the NPP-LS subgroup had a similar mean age (6.07 years vs 7.00 years) at baseline and height SDS gain in year 1 (0.64 vs 0.70), although NPP-non-LS-responders were taller (P < 0.001) at baseline. BMI SDS changes did not differ across subgroups. Treatment-emergent AEs were experienced by 65.3% of patients; hypoglycaemia was most common. Conclusions: In most NPP children with SPIGFD, with or without LS, rhIGF1 therapy promotes linear growth. The safety profile was consistent with previous studies.
Authors with CRIS profile
Involved external institutions
Hôpital Necker-Enfants malades
France (FR)
Ipsen Pharma
France (FR)
Linköpings universitet / Linköping University
Sweden (SE)
France (FR)
Ipsen Pharma
France (FR)
Linköpings universitet / Linköping University
Sweden (SE)
How to cite
APA:
Bang, P., Wölfle, J., Perrot, V., Sert, C., & Polak, M. (2021). Effectiveness and safety of rhIGF1 therapy in patients with or without Laron syndrome. European Journal of Endocrinology, 184(2), 267-276. https://doi.org/10.1530/EJE-20-0325
MLA:
Bang, Peter, et al. "Effectiveness and safety of rhIGF1 therapy in patients with or without Laron syndrome." European Journal of Endocrinology 184.2 (2021): 267-276.
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