کد مقاله | کد نشریه | سال انتشار | مقاله انگلیسی | نسخه تمام متن |
---|---|---|---|---|
6000441 | 1579202 | 2016 | 8 صفحه PDF | دانلود رایگان |
![عکس صفحه اول مقاله: Nonacog beta pegol (N9-GP) in haemophilia B: A multinational phase III safety and efficacy extension trial (paradigmâ¢4) Nonacog beta pegol (N9-GP) in haemophilia B: A multinational phase III safety and efficacy extension trial (paradigmâ¢4)](/preview/png/6000441.png)
- Nonacog beta pegol is an extended half-life glycoPEGylated factor IX (FIX).
- It was designed to enable prophylaxis with high activity levels/fewer weekly doses.
- Paradigmâ¢4 was an open-label, international extension trial of nonacog beta pegol.
- Safety/efficacy data were collected on prophylaxis and the treatment of bleeds.
- Nonacog beta pegol was well tolerated and efficacious in haemophilia B patients.
IntroductionParadigmâ¢4 was an international extension trial investigating the safety and efficacy of nonacog beta pegol, a recombinant glycoPEGylated factor IX (FIX) with extended half-life, in haemophilia B patients (FIX activity â¤Â 2%; aged 13-70 years) who had previously participated in phase III pivotal (paradigmâ¢2) or surgery (paradigmâ¢3) trials.MethodsPatients chose to continue treatment with nonacog beta pegol in either one of two once-weekly prophylaxis arms (10 IU/kg or 40 IU/kg), or an on-demand arm (40 IU/kg for mild/moderate bleeds; 80 IU/kg for severe bleeds). The primary objective was to evaluate immunogenicity; key secondary objectives included assessing safety and haemostatic efficacy in the treatment and prevention of bleeds.ResultsSeventy-one patients received prophylaxis or on-demand treatment. No patient developed an inhibitor and no safety concerns were identified. The success rate for the treatment of reported bleeds was 94.6%; most (87.9%) resolved with one injection. The median annualised bleeding rate for patients on prophylaxis was 1.36 (interquartile range [IQR] 0.00-2.23) and 1.00 (IQR 0.00-2.03) for the 10 and 40 IU/kg treatment arms, respectively. The mean FIX activity trough achieved for 10 and 40 IU once weekly was 9.8% and 21.3%, respectively. Fourteen patients on prophylaxis underwent 23 minor surgical procedures; haemostatic perioperative outcomes for all of those evaluated were 'excellent' or 'good'.ConclusionsNonacog beta pegol showed a favourable tolerability profile (with no safety issues identified) with good prophylactic protection and control of bleeding in previously treated adult and adolescent haemophilia B patients.
Journal: Thrombosis Research - Volume 141, May 2016, Pages 69-76