Extending recombinant factor IX Fc fusion protein dosing interval to 14 or more days in patients with hemophilia B
Amy D. Shapiro, K. John Pasi, Margareth C. Ozelo, Roshni Kulkarni, Christopher Barnowski, Bent Winding, Johan Szamosi, Stefan Lethagen
ARTIGO
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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...
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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
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Extending recombinant factor IX Fc fusion protein dosing interval to 14 or more days in patients with hemophilia B
Amy D. Shapiro, K. John Pasi, Margareth C. Ozelo, Roshni Kulkarni, Christopher Barnowski, Bent Winding, Johan Szamosi, Stefan Lethagen
Extending recombinant factor IX Fc fusion protein dosing interval to 14 or more days in patients with hemophilia B
Amy D. Shapiro, K. John Pasi, Margareth C. Ozelo, Roshni Kulkarni, Christopher Barnowski, Bent Winding, Johan Szamosi, Stefan Lethagen
Fontes
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Research and practice in thrombosis and haemostasis Vol. 3, no. 1 (Jan., 2019), p. 109-113 |