|Title:||Nonacog beta pegol in previously treated children with hemophilia B: results from an international open-label phase 3 trial||Authors:||Carcao, M.
Karim, F. Abdul
Lu, M. -Y.
|Keywords:||factor IX;hemophilia B;nonacog beta pegol;pediatrics;prophylaxis||Issue Date:||2016||Start page/Pages:||1521-1529||Source:||Journal of Thrombosis and Haemostasis||Abstract:||
Background Nonacog beta pegol is a recombinant glycoPEGylated factor IX with an extended half-life, developed to improve care for patients with hemophilia B. ;Objectives To investigate the safety, efficacy and pharmacokinetics of nonacog beta pegol for the prophylaxis and treatment of bleeds in previously treated children with hemophilia B. ;Patients/Methods This phase 3 trial, paradigm5, enrolled and treated 25 children (aged 12 years) with hemophilia B (FIX 2%). Patients were stratified by age (0-6 years and 7-12 years), and received once-weekly prophylaxis with 40 IU kg(-1) nonacog beta pegol for 50 exposure days. ;Results No patient developed inhibitors, and no safety concerns were identified. Forty-two bleeds in 15 patients were reported to have been treated; the overall success rate was 92.9%, and most bleeds (85.7%) resolved after one dose. The median annualized bleeding rates (ABRs; bleeds per patient per year) were 1.0 in the total population, 0.0 in the 0-6-year group, and 2.0 in the 7-12-year group; the estimated mean ABRs were 1.44 in the total population, 0.87 in the 0-6-year group, and 1.88 in the 7-12-year group. For 22 patients who had previously been receiving prophylaxis, the estimated mean ABR was 1.38 versus a historical ABR of 2.51. Estimated mean steady-state FIX trough levels were 0.153 IU mL(-1) (0-6 years) and 0.190 IU mL(-1) (7-12 years). ;Conclusion Nonacog beta pegol was well tolerated in previously treated children with hemophilia B; a 40 IU kg(-1) dose provided effective once-weekly prophylaxis and hemostasis when bleeds were treated.
|Appears in Collections:||醫學系|
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