Effectiveness and Safety of rhIGF-1 Therapy in Children: The European Increlex (R) Growth Forum Database Experience
2015 (English)In: Hormone Research in Paediatrics, ISSN 1663-2818, E-ISSN 1663-2826, Vol. 83, no 5, 345-357 p.Article in journal (Refereed) Published
Background/Aims: We report data from the EU Increlex (R) 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 naive 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. (C) 2015 S. Karger AG, Basel
Place, publisher, year, edition, pages
KARGER , 2015. Vol. 83, no 5, 345-357 p.
Height; rhIGF-1 therapy; EU IGFD Registry; Short stature; Severe primary insulin-like growth factor-1 deficiency
IdentifiersURN: urn:nbn:se:liu:diva-120068DOI: 10.1159/000371798ISI: 000355975400007PubMedID: 25824333OAI: oai:DiVA.org:liu-120068DiVA: diva2:839934
Funding Agencies|Ipsen, France2015-07-062015-07-062015-07-06