ESPE2023 Poster Category 1 Growth and Syndromes (75 abstracts)
Pathway to assess severe primary IGF-1 deficiency diagnosis by using the IGF-1 generation test in a real-life setting: data from the Global Increlex® Registry
Jean De Schepper 1 , Artur Bossowski 2 , Jesús Argente 3 , Caroline Sert 4 , Valérie Perrot 4 , Daniele Pennestri 5 & Peter Bang 6
1Department of Pediatrics, UZ Brussels, Free University of Brussels, Brussels, Belgium. 2Department of Paediatrics, Endocrinology, and Diabetology with Cardiology Division, Medical University of Bialystok, Bialystok, Poland. 3Hospital Infantil Universitario Niño Jesús, Instituto de Investigación La Princesa, Universidad Autónoma de Madrid, Centro de Investigación Biomédica en Red de la Fisiopatología (CIBER) de Fisiopatología de Obesidad y Nutrición (CIBEROBN), Instituto de Salud Carlos III and IMDEA Food Institute, Madrid, Spain. 4Ipsen, Boulogne-Billancourt, France. 5Ipsen, Slough, United Kingdom. 6Division of Pediatrics, Department of Biomedical and Clinical Sciences, Faculty of Health Sciences, Linköping University, Linköping, Sweden
Background: severe primary insulin-like growth factor-1 deficiency (SPIGFD) is a rare growth disorder, for which insulin-like growth factor-1 (IGF-1) generation test (IGFGT) is debated as a complementary diagnostic analysis. Diagnostic workup for SPIGFD varies geographically and diagnosis is delayed by the rarity of the condition (<1/10,000). Evaluation of real-world practices of IGFGT could help facilitate diagnosis and test use. Objective: to describe real-life IGFGT results from patients with SPIGFD in the Global Increlex® Registry.
Methods: The ongoing Global Increlex® Registry is a multicentre, open-label, observational study (in 10 European countries and the USA) monitoring the long-term safety and effectiveness of recombinant human IGF-1 (rhIGF-1; Increlex® [mecasermin]) in children and adolescents with SPIGFD (NCT00903110). Eligible patients: aged 2–18 years of age, received rhIGF-1 for growth failure due to SPIGFD.
Results: At data cut-off (03 October 2022), 85.4% (n=263/308) of enrolled patients had a SPIGFD diagnosis; 14.6% (n=45/308) had Laron syndrome (LS) and 62.0% (n=191/308) were naïve patients with SPIGFD. IGFGT was reported in 42.4% (n=128/302) of enrolled patients; 44.7% (n=115/257) of patients with SPIGFD, 63.6% (n=28/44) of patients with LS and 45.5% (n=85/187) of naïve patients with SPIGFD. Between countries, IGFGT ranged from 100% (n=22/22) in Poland to 22.5% (n=18/80) in France. In naïve patients with SPIGFD, ΔIGF-1 (ng/mL) was <15 in 64.2% (n=52/81) and ≥15 in 35.8% (n=29/81). ΔIGF-1 (ng/mL) was <15 in all naïve patients with SPIGFD with LS. Table 1 presents ΔIGF 1 (ng/mL) <15 and ≥15 subgroup results for naïve patients with SPIGFD; multivariate analysis demonstrated age at rhIGF-1 initiation (rII) was predictive of IGF-1 stimulation response.
| ΔIGF-1 (ng/mL) | ||||
| <15 (n=52) | ≥15 (n=29) | Univariate analyses (P-value)* | ||
| Pubertal at rII | n (%) | 4 (7.7%) | 2 (6.9%) | - |
| Age at rII (years) | Mean (SD) 95%CI | 6.8 (4.0) 5.7;7.9 | 10.3 (3.3) 9.0;11.5 | <0.001** |
| Basal IGF-1 (ng/mL) | Mean (SD) 95%CI | 39.0 (36.9) 28.8;49.3 | 65.7 (33.8) 52.9;78.6 | 0.005 |
| Height SDS at rII | n Mean (SD) 95%CI | 43 -4.2 (1.9) -4.7;-3.6 | 25 -3.2 (0.9) -3.6;-2.8 | 0.029 |
| *Significance level of 0.05; **Significant in the multivariate analysis. CI: confidence interval; SD(S): standard deviation (score) | ||||
Conclusions: In patients diagnosed with SPIGFD, IGFGT was performed in >40.0%. However, there is geographic heterogeneity. ΔIGF-1 (ng/mL) ≤15 was reported in >60.0% of naïve patients with SPIGFD; ΔIGF-1 (ng/mL) increased with age at rhIGF-1 initiation. All naïve patients with SPIGFD with LS had ΔIGF-1 (ng/mL) <15.
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