ESPE2024 Poster Category 1 Growth and Syndromes 1 (10 abstracts)
Long-term effectiveness and safety data from the Global Increlex® Registry in patients treated with rhIGF-1: Subgroup analysis of naïve pre-pubertal patients and patients with Laron syndrome
Marta Ramón Krauel 1,2,3 , Joachim Woelfe 4 , Michel Polak 5 , Mohamad Maghnie 6,7,3 , Iwona Beń-Skowronek 8 , Caroline Sert 9 , Valérie Perrot 9 & Peter Bang 10
1Hospital Sant Joan de Déu, Institut de Recerca Sant Joan de Déu (IRSJD), Barcelona, Spain. 2Centro de Investigación Biomédica en Red de Diabetes y Enfermedades Metabólicas Asociadas (CIBERDEM), Instituto de Salud Carlos III, Madrid, Spain. 3Endo-ERN European Reference Network on Rare Endocrine Conditions, Amsterdam, Netherlands. 4Department of Pediatrics and Adolescent Medicine, Friedrich-Alexander-University (FAU) Erlangen-Nürnberg, Erlangen, Germany. 5Department 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. 6Department of Pediatrics, IRCCS Istituto Giannina Gaslini, University of Genova, Genova, Italy. 7Department of Neuroscience, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health, University of Genova, Genova, Italy. 8Department of Paediatric Endocrinology and Diabetology with Laboratory of Endocrinology and Metabolism, Medical University of Lublin, Lublin, Poland. 9Ipsen, Boulogne-Billancourt, France. 10Division of Pediatrics, Department of Biomedical and Clinical Sciences, Faculty of Health Sciences, Linköping University, Linköping, Sweden
Background: The Global Increlex® Registry (NCT00903110) monitors real-world safety and effectiveness of recombinant human insulin-like growth factor (rhIGF-1; Increlex® [mecasermin]) in severe primary IGF-1 deficiency (SPIGFD). Patient characteristics, effectiveness and safety data from 2008–2023 are described.
Methods: Descriptive analyses of registry data from children/adolescents aged 2–18 years receiving rhIGF 1 for SPIGFD in the EU and US.
Results: At data cut-off (20 April 2023), 324 patients had enrolled. 59.6% were naïve pre pubertal (NPP) and 14.5% had Laron syndrome; 8.0% were NPP with Laron syndrome. For therapy-ended patients (n = 192/322) and patients who reached adult height (n = 102/324), median (Q1;Q3) treatment duration was 3.0 (1.5;4.5) and 3.9 (2.3;6.0) years, respectively. Height velocity was similar in NPP and Laron syndrome populations at Years 1 and 5, with greatest improvements in height outcomes observed in NPP patients with Laron syndrome (Table). Height standard deviation score (HtSDS) improvement in NPP patients from rhIGF-1 initiation to Years 1 (n = 144), 2 (n = 124) and 4 (n = 78) was greater in patients with lower baseline HtSDS (P <0.05). Overall, 64.0% (n = 206/322) experienced ≥1 treatment-emergent adverse event (TEAE). Hypoglycaemia was the most frequent (25.8%) targeted TEAE. 19 (5.9%) patients withdrew treatment due to TEAEs; 1 fatal TEAE was reported (myelodysplastic syndrome, unrelated to treatment).
| Year | Age,a years n Mean (SD) |
HtSDSb n Mean (SD) |
∆HtSDSb n Mean (SD) |
Height velocity,b cm/year n Mean (SD) |
|
| Overall (n = 312) | Baseline 1 3 5 |
323 9.4 (4.1) |
283 −3.7 (1.4) |
235 0.3 (0.5) 142 0.9 (0.8) 75 1.1 (0.9) |
171 4.8 (2.4) 225 6.9 (2.2) 130 5.7 (2.1) 61 5.1 (1.7) |
| NPP (n = 189) | Baseline 1 3 5 |
193 7.8 (3.6) |
172 −3.8 (1.5) |
144 0.4 (0.4) 98 1.0 (0.7) 57 1.3 (0.9) |
93 4.7 (1.7) 141 7.2 (2.1) 92 5.9 (1.8) 46 5.3 (1.7) |
| Laron syndrome (n = 47) | Baseline 1 3 5 |
47 8.9 (5.2) |
39 −4.9 (1.7) |
34 0.5 (0.6) 24 0.8 (1.0) 17 1.3 (1.2) |
24 5.7 (4.8) 32 7.0 (2.7) 23 4.8 (2.6) 13 5.2 (1.4) |
| NPP/Laron syndrome (n = 26) | Baseline 1 3 5 |
26 5.4 (3.5) |
20 −5.4 (1.9) |
17 0.7 (0.5) 14 1.2 (1.2) 12 1.6 (1.3) |
8 5.4 (1.4) 16 8.0 (2.1) 12 5.8 (1.6) 9 5.5 (1.4) |
| aEnrolled population; bRegistry population. | |||||
Conclusion: Long-term HtSDS improvements in registry-enrolled patients with SPIGFD are encouraging. Five-year treatment data are likely enriched by treatment-responsive NPP patients, especially those with Laron syndrome. Safety data aligned with previous reports.
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