Patient characteristics and pharmacokinetic profiling
Real world data from 35 severe and 2 moderately-severe haemophilia B patients was available for assessment of the predictive performance of the published rFIX-Fc population PK model (Table 1). Median age was 15.8 years (range 2.3-71.0), and 14 patients were below the age of 12 years. Patients received a median dose of 36 IU/kg rFIX-Fc concentrate (range 10 – 132 IU/kg). In total, 287 FIX activity levels measured by OSA were available for analysis. Three FIX activity levels (1% of the data) were below LLOQ and therefore excluded in the analysis29,31. During PK profiling, a median of five FIX activity levels (range 3-7) in adolescent and adult patients (≥12 years of age) and four FIX activity levels (range 3-7, mean 4.5) in children (<12 years of age) were sampled. PK data was obtained during a median of 2 occasions per individual (range 1-9).