Session: FP20-Growth: Clinical Trials & Observational Studies
Room 122 (Moscone Center)
Poster Board SUN-624
Methods: This randomized, open-label trial in 27 US pediatric endocrinology centers investigated safety and efficacy of treatment with rhGH alone (45µg/kg QD) compared with rhGH/rhIGF-1 co-administration (three co-admin groups: rhGH 45µg/kg QD plus separate injections of rhIGF-1 of 50, 100 or 150µg/kg QD) in treatment-naïve, prepubertal children (baseline height SDS ≤−2, IGF-1 SDS ≤−1 and maximum stimulated GH ≥10ng/mL). A total of 106 patients were randomized: 26 to rhGH alone and 80 to rhGH/rhIGF-1 co-administration.
Results: Safety data were available for the 106 patients (299 patient-years’ exposure). At baseline, overall mean±SD age was 8.8±2.1 years and height SDS was −2.5±0.4. Treatment emergent adverse events (TEAEs) of special interest included headache (rhGH: 54% vs co-admin: 61%), vomiting (35% vs 29%), otitis media (15% vs 21%), hypoglycemia (8% vs 9%), lipohypertrophy (0% vs 20%) and intracranial hypertension/papilledema (0% vs 3%). The most common TEAEs, in addition to headache, were upper respiratory tract infection (39% vs 40%) and pyrexia (35% vs 40%). Two single cases of serious TEAEs were reported: papilledema (150µg/kg co-admin group) and intracranial hypertension (100µg/kg co-admin group). Both events resolved after discontinuation of treatment and therapy was restarted thereafter. Treatment effects were not evident for C-peptide or fasting venous glucose. Mean glycosylated hemoglobin did not change significantly. At each rhIGF-1 dose, co-admin therapy resulted in greater first-year height velocity vs rhGH alone; rhGH mean±SD: 9.3±1.7cm/year vs co-admin 50, 100 and 150µg/kg dose groups: 10.1±1.3, 9.7±2.5 and 11.2±2.1cm/year, respectively (modified Intention-to-Treat population [n=105]; p<0.001, rhGH alone vs co-admin 150µg/kg).
Conclusion: Additional safety concerns were not evident with co-administration of rhGH/rhIGF-1 therapy in this trial compared with previously reported rhGH and rhIGF-1 safety profiles. rhGH/rhIGF-1 therapy resulted in a first-year height velocity exceeding that of rhGH alone.
Disclosure: BSM: Consultant, Ipsen, Consultant, Genentech, Inc., Consultant, Pfizer, Inc., Consultant, Novo Nordisk, Consultant, Eli Lilly & Company, Consultant, Sandoz, Consultant, Teva, Consultant, Endo Pharmaceuticals, Researcher, Ipsen, Researcher, Genentech, Inc., Researcher, Pfizer, Inc., Researcher, Novo Nordisk, Researcher, Eli Lilly & Company, Researcher, Endo Pharmaceuticals, Researcher, Abbott Laboratories. PFB: Investigator, Ipsen, Member of advisory committees or review panels, Ipsen. PD: Employee, Ipsen. AS: Independent Contractor (including contracted research), Ipsen. EL: Employee, Ipsen. BJR: Principal Investigator, Ipsen, Independent Contractor (including contracted research), Ipsen. Nothing to Disclose: DEH, MAS
*Please take note of The Endocrine Society's News Embargo Policy at http://www.endo-society.org/endo2013/media.cfm
See more of: Abstracts - Orals, Featured Poster Presentations, and Posters