ESPE2023 Poster Category 1 Growth and Syndromes (75 abstracts)
Characteristics, effectiveness and safety data for patients with growth failure treated with recombinant IGF-1 and achieving adult or near-adult height: results from the Increlex® Global Registry
Peter Bang 1 , Marta Ramón Krauel 2,3 , Mohamad Maghnie 4,5 , Joachim Woelfle 6 , Caroline Sert 7 , Valérie Perrot 7 , Daniele Pennestri 8 & Michel Polak 9
1Division of Pediatrics, Department of Biomedical and Clinical Sciences, Faculty of Health Sciences, Linköping University, Linköping, Sweden. 2Hospital Sant Joan de Déu, Institut de Recerca Sant Joan de Déu, Barcelona, Spain. 3Centro de Investigación Biomédica en Red de Diabetes y Enfermedades Metabólicas Asociadas (CIBERDEM), Instituto de Salud Carlos III, Madrid, Spain. 4Department of Pediatrics, IRCCS Istituto Giannina Gaslini, Genova, Italy. 5Department of Neuroscience, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health University of Genova, Genova, Italy. 6Department of Pediatrics and Adolescent Medicine, Friedrich-Alexander-University (FAU) Erlangen-Nürnberg, Erlangen, Germany. 7Ipsen, Boulogne-Billancourt, France. 8Ipsen, Slough, United Kingdom. 9Department of Pediatric Endocrinology, Gynaecology, and Diabetology, Assistance Publique – Hôpitaux de Paris, Hôpital Universitaire Necker-Enfants Malades, IMAGINE Institute, INSERM U1016, University of Paris, Paris, France
Background: severe primary insulin-like growth factor-1 deficiency (SPIGFD) is a rare growth disorder. Recombinant human insulin-like growth factor (IGF-1) (rhIGF-1; Increlex® [mecasermin]) replacement therapy is EU and US-approved for treating growth failure due to SPIGFD. The long-term therapeutic objective of rhIGF-1 treatment in SPIGFD is to improve adult height (AH). Objective: to describe the characteristics, safety and effectiveness data for patients with growth failure receiving rhIGF-1 achieving AH or near-AH and elucidate the factors that predict treatment response.
Methods: The ongoing Global Increlex® Registry is a multicentre, open-label, observational study (in ten European countries and the USA) monitoring the long-term safety and effectiveness of rhIGF-1 in children and adolescents with SPIGFD (NCT00903110). Eligible patients: aged 2–18 years, received rhIGF-1 for growth failure due to SPIGFD.
Results: At data cut-off (03 October 2022), 101 patients reached AH; 66.3% (n=67/101) were male. SPIGFD diagnosis was reported in 86.1% (n=87/101) of patients; at Baseline, 56.4% of patients were treatment-naïve (n=57/101) and 45.5% (n=46/101) were naïve prepubertal patients (NPP). 58.2% (n=57/98) performed a genetic test. 18.8% (n=19/101) had Laron syndrome. Table 1 summarises results for the overall and NPP populations. Multivariate analysis revealed height standard deviation score (SDS) (HtSDS) gain from rhIGF-1 initiation to final AH (FAH) in the NPP population (n=26) was higher in patients with lower Baseline HtSDS (P<0.001), higher predicted AH (PAH) (P=0.028) and higher biological mother height (BMH) (P=0.039). In the overall population, 66.3% (n=67/101) presented ≥1 treatment-emergent adverse event (TEAE). Hypoglycaemia was the most frequent TEAE and serious targeted TEAE.
| Overall population (n=101) | NPP population (n=46) | ||
| Age (years) at rhIGF-1 treatment | Initiation n Mean (SD) | 101 11.7 (3.4) | 46 10.0 (3.3) |
| End n Mean (SD) | 101 16.1 (2.7) | 46 15.2 (2.9) | |
| Treatment duration (years) | n Median Q1;Q3 | 101 3.9 2.3;5.9 | 46 4.6 3.5;7.0 |
| Year 1 annualised height velocity (cm/yr) | n Mean (SD) | 77 6.8 (2.4) | 36 7.6 (2.3) |
| FAH SDS | n Mean (SD) | 101 −2.8 (1.7) | 46 −2.2 (1.4) |
| Difference between FAH SDS and HtSDS at rhIGF-1 initiation | n Mean (SD) 95%CI | 91 0.9 (1.1) 0.7;1.1 | 42 1.4 (1.0) 1.1;1.7 |
| CI: confidence interval; SD: standard deviation; Q1;Q3: lower/upper quartile | |||
Conclusions: There was a significant gain in HtSDS from rhIGF-1 initiation to FAH in patients with SPIGFD receiving rhIGF-1. Baseline HtSDS, PAH and BMH were predictive of HtSDS gain at FAH in NPP patients. Safety data are consistent with the profile of mecasermin.
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