OR03-07 Real-World Effectiveness and Safety of Recombinant Human IGF-1 Treatment in Naïve Prepubertal Patients with Severe Primary IGF-I Deficiency at Near-Adult Height: Results from the Global IGFD Registry

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Abstract Disclosure: P. Backeljauw: Ascendis, BioMarin, Cavalry Biosciences, Eton Pharmaceuticals, Ipsen, Novartis/Sandoz, Novo Nordisk, Tolmar. J. Argente: Novo Nordisk, Pfizer, Inc., Sandoz. A. Bossowski: IBSA, Ipsen, Novo Nordisk, Pfizer, Inc., Sandoz. J. De Schepper: Alexion Pharmaceuticals, Inc., Eli Lilly & Company, Ferring Pharmaceuticals, Ipsen, Kyowa Kirin, Menarini, Novo Nordisk, Pfizer, Inc., Sandoz, Siemens Healthcare. M. Maghnie: BioMarin, Ferring Pharmaceuticals, Ipsen, Merck Serono, Novo Nordisk, Pfizer, Inc., Sandoz. M. Polak: Ipsen, Merck, Novo Nordisk, Pfizer, Inc., Sanofi, Sandoz, French Public Research funds (ANR and PHRC). M. Ramon-Krauel: Merck, Novo Nordisk, Pfizer, Inc., Sandoz. J. Woelfle: Ascendis, BioMarin, Hexal, HRA Pharmaceuticals, Merck, Novo Nordisk, Pfizer, Inc., Roche Pharmaceuticals, Serono, endpoint adjucation committee for macimorelin (ICON). C. Sert: Ipsen. V. Perrot: Ipsen. P. Bang: Eli Lilly & Company, Ipsen. Background: The Global Increlex® Growth Forum Database (IGFD) Registry monitors the real-world effectiveness and safety of recombinant human insulin-like growth factor-1 (rhIGF-1; Increlex® [mecasermin]) in treating patients aged 2-18 years with growth failure due to severe primary insulin-like growth factor-I deficiency (SPIGFD). The therapeutic objective of rhIGF-1 is to improve adult height (AH). Previous data show that prepubertal patients naïve to growth-promoting therapies at rhIGF-1 initiation (naïve prepubertal [NPP]) have better height outcomes than non-NPP patients.1 Here, we report characteristics, effectiveness, and safety data for NPP patients achieving near-AH (NAH). Methods: Descriptive analyses of NPP patients who achieved NAH (last height velocity <1 cm/year) in the Global IGFD Registry (NCT00903110). Results are presented by gender and age at rhIGF-1 treatment initiation. Results: At data cut-off (April 20, 2023), 45 NPP patients had reached NAH; 57.8% (26/45) were boys, and 88.9% (40/45) were reported to have SPIGFD, including 6.7% (3/45) with Laron syndrome confirmed by genetic analysis. Mean (standard deviation [SD]) age at rhIGF-1 initiation and completion was 10.8 (3.3)/9.2 (3.1) years and 16.4 (2.3)/14.0 (3.0) years for boys/girls, respectively. Median (Q1; Q3) rhIGF-1 treatment duration for boys/girls was 4.9 (3.5; 7.1)/4.0 (3.0; 6.9) years; treatment duration in the prepubertal period for boys/girls was 2.2 (1.3; 3.5)/2.6 (1.9; 4.0) years and during puberty was 2.9 (1.6; 3.8)/2.2 (0.0; 3.7) years. Mean (SD) height standard deviation score (HtSDS) for boys/girls at rhIGF-1 treatment initiation was −3.3 (1.1)/−3.9 (1.8) and at NAH was −2.0 (1.4)/−2.5 (1.3), with a mean (SD) HtSDS gain of 1.3 (0.8)/1.5 (1.2). Cumulative HtSDS gain increased over time, with greatest height velocity observed in the first year of rhIGF-1 treatment in boys and girls. Height velocity decreased each year, but remained above baseline in treated patients through 5 years follow-up for boys and 4 years follow-up for girls. Mean (SD) NAH was 161.3 (14.1)/148.5 (9.0) cm for boys/girls. Normal AH range (HtSDS >−2) was achieved by 53.8% (14/26) of boys and 36.8% (7/19) of girls at NAH. Patients who initiated rhIGF-1 at ≤10 (n=21) vs >10 years (n=24) had a slightly greater HtSDS gain from rhIGF-1 treatment initiation to NAH (mean [SD]: 1.5 [1.3] vs 1.3 [0.8]). Safety data for NPP patients who reached NAH were consistent with previous reports. Conclusions: There was a significant gain in HtSDS from rhIGF-1 initiation to NAH in NPP patients. Patients who initiated treatment at a younger vs older age had a marginally greater HtSDS gain to NAH. Reference: 1. Bang P et al. Horm Res Paediatr 2015;83:345-57. Funding: This study was sponsored by Ipsen. Presentation: Saturday, July 12, 2025

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