Once-weekly prophylaxis regimen of nonacog alfa in patients with hemophilia B: an analysis of timing of bleeding event onset.

摘要: In a pivotal, multicenter, open-label study, 25 patients aged 12-54 years with moderately severe/severe hemophilia B received on-demand nonacog alfa (6 months; dose at investigator's discretion) followed by once-weekly prophylaxis 100 IU/kg (12 months). During prophylaxis, had median spontaneous annualized bleeding rate (sABR) of 1.0 and significant reductions in ABR (P < 0.0001). This post hoc analysis examined the time onset events (sBEs) target joint (sTJBEs). The postdosing day (D) sBEs observed during steady-state FIX activity data (FIX:C) between 144 196 h postdose were collected weeks 26 78. Twelve (48%) no sBEs; remaining 13 (52%) following sBEs: less than 1 D (0%), to 2D (5%), 2 3 (22%), 4 (9%), 5D 5 6D (23%), 6 7D (11%), least (8%). Reductions sTJBEs versus treatment experienced all patients. Target sABR was 0 for 5/13 reduction ranged from 66.1% (27.2→9.2) 97.8% (46.2→1.0); sTJBE 6.2% (2.1→2.0) 100% (from 40.1, 19.1, 3.9, 9.0, 6.1--0). 47% (8/17) trough samples more 2%. sBE patients, number TJBEs reduced alfa. When occurred, they apparent pattern occurrence. Clinicaltrials.gov identifier: NCT01335061.