1. Effectiveness and Safety of rhIGF-1 Therapy in Children: The European Increlex® Growth Forum Database Experience
- Author
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Bang, P, Polak, M, Woelfle, J, Houchard, A, Hausler, G, Zwiauer, K, Adiceam, P, Carla Malpuech, H, Cessans, C, Cogne, M, Colle, M, Coutant, R, Houang, M, Lienhardt, A, Mallet, E, Nicolino, M, Petrus, M, Tauber, Mt, Wagner, K, Weill, J, Akkurt, I, Bechtold, S, Bierkamp Christophersen, D, Blanke, Jg, Bonfig, W, Doerr, Hg, Frühwald, M, Hartmann, K, Hauffa, B, Holterhus, Pm, Hübner, A, Ittner, J, Jourdan, C, Keller, A, Kim Berger HS, Köster, B, Krüger, J, Land, C, Leichter, H, Lorenzen, F, Meissner, T, Mohnike, K, Morlot, M, Müller, H, Ockert, C, Rohrer, T, Pankau, R, Partsch, Cj, Schönau, E, Schwab, K, Simic Shleicher, G, Tittel, B, Bernasconi, S, Cannavo', Salvatore, Cappa, M, Cavallo, L, Cherubini, V, Chiumello, G, Citro, G, Concolino, D, Perri, P, Lampis, A, Maghnie, M, Perrone, L, Pilotta, A, Sinisi, A, Zuccotti, G, Zucchini, S, Ben Skowronek, I, Birkholz, D, Bossowski, A, Hilczer, M, Korpal Szczyrska, M, Smyczynska, J, Szewczyk, L, Argente, J, Bezanilla, C, Carrascosa, A, Diaz, R, Fernandez, C, Hermoso, F, Luzuriaga, C, Martos, J, Prieto, P, Sanchez Del Pozo, J, Vela, A, Ekström, K, Nilsson, Nö, Ahmed, F, Denvir, L, Hussain, K, Johnstone, H, Mushtaq, T, Patel, L, Ramakrishnan, R, Rose, S, Shaw, N, Storr, H., Bang, P, Polak, M, Woelfle, J, Houchard, A, EU IGFD Registry Study, Group, and Perrone, Laura
- Subjects
Male ,Adolescent ,Databases, Factual ,Endocrinology, Diabetes and Metabolism ,MEDLINE ,EU IGFD registry ,computer.software_genre ,Short stature ,Endocrinology ,rhIGF-1 therapy ,Humans ,Medicine ,Insulin-Like Growth Factor I ,Child ,Growth Disorders ,Database ,Height ,business.industry ,severe primary insulin-like growth factor-1 deficiency ,Recombinant Proteins ,Europe ,short stature ,Child, Preschool ,Pediatrics, Perinatology and Child Health ,Female ,Observational study ,medicine.symptom ,Height, rhIGF-1 therapy, EU IGFD registry, short stature, severe primary insulin-like growth factor-1 deficiency ,business ,computer - Abstract
Background/Aims: We report data from the EU Increlex® Growth Forum Database (IGFD) Registry, an ongoing, open-label, observational study monitoring clinical practice use of recombinant human insulin-like growth factor-1 (rhIGF-1) therapy in children. Methods: Safety and effectiveness data on rhIGF-1 treatment of 195 enrolled children with growth failure were collected from December 2008 to September 2013. Results: Mean ± SD (95% CI) height velocity during first year of rhIGF-1 treatment was 6.9 ± 2.2 cm/year (6.5; 7.2) (n = 144); in prepubertal patients naïve to treatment, this was 7.3 ± 2.0 cm/year (6.8; 7.7) (n = 81). Female sex, younger age at start of rhIGF-1 therapy, and lower baseline height SDS predicted first-year change in height SDS. The most frequent targeted treatment-emergent adverse events (% patients) were hypoglycemia (17.6%, predictors: young age, diagnosis of Laron syndrome, but not rhIGF-1 dose), lipohypertrophy (10.6%), tonsillar hypertrophy (7.4%), injection site reactions (6.4%), and headache (5.9%). Sixty-one serious adverse events (37 related to rhIGF-1 therapy) were reported in 31 patients (16.5%). Conclusion: Safety and effectiveness data on use of rhIGF-1 in a ‘real-world' setting were similar to those from controlled randomized trials. Severe growth phenotype and early start of rhIGF-1 improved height response and predicted risk of hypoglycemia.
- Published
- 2015