In the phase 3 B-LONG study (NCT01027364), prophylaxis with recombinant factor IX Fc fusion protein (rFIXFc) every 7 to >14 days was associated with low annualized bleed rates (ABRs) in males aged >= 12 years with severe hemophilia B. The long-term safety and efficacy of rFIXFc prophylaxis was...

In the phase 3 B-LONG study (NCT01027364), prophylaxis with recombinant factor IX Fc fusion protein (rFIXFc) every 7 to >14 days was associated with low annualized bleed rates (ABRs) in males aged >= 12 years with severe hemophilia B. The long-term safety and efficacy of rFIXFc prophylaxis was confirmed in the B-YOND study (NCT01425723), an extension of the B-LONG clinical trial. The aim of this post-hoc analysis was to evaluate the efficacy of a >= 14-day rFIXFc dosing interval in patients treated prophylactically during B-LONG or B-YOND. The analysis included 22 patients aged >= 12 years who received prophylactic rFIXFc with a >= 14-day dosing interval at any time during B-LONG or B-YOND up until the second interim analysis of B-YOND (September 2015). The median (interquartile range [IQR]) rFIXFc exposure on the >= 14-day dosing interval was 3.4 (1.8-4) years. Patients treated with a >= 14-day dosing interval were well controlled with a median (IQR) overall ABR of 1.6 (0.6-2.7) and a median (IQR) spontaneous ABR of 0.7 (0.3-1.1) in 18 evaluable patients. A rFIXFc dosing interval of >= 14 days was well tolerated, with no new safety concerns identified. Most patients on rFIXFc prophylaxis, with a dosing interval of >= 14 days, remained well controlled; ABRs were consistent with those reported in the overall study population. A >= 14-day dosing interval can be utilized in some well controlled individuals and reduces the burden imposed by frequent prophylactic injections while maintaining adequate bleed suppression

Fechado