Nonacog Beta Pegol (N9-Gp) Prophylaxis in Pediatric Patients with Severe Hemophilia B: A Single Center Real World...

Hemophilia B (Christmas disease) is a rare genetic bleeding disorder. Frequent FIX prophylaxis is challenging in pediatric patients because of difficult venous access and pain during factor administration. Nonacog beta pegol (N9-GP) is an extended half-life (EHL) recombinant FIX. The study assessed the efficacy of Nonacog beta pegol (N9-GP) in reducing bleeding episodes and its safety as prophylaxis in pediatric patients. The primary end-point was reduction in Annual bleeding rates (ABR) and Annual Joint bleeding rates (AJBR). The secondary objectives were to assess the days of school absenteeism, number of emergency visits, measurement of Hemophilia Joint Health Score (HJHS) 2.1 score, pediatric Hemophilia Activities List (pedHAL) score, development of inhibitor against FIX during treatment, and safety & adverse effects (AEs) profile. Ten pediatric patients managed with on-demand SHL FIX were included in the study. Their median age at study entry was 3 years (range, 1-13 years). The median duration of follow-up was 12 months (range, 6-60 months). They were subsequently started on N9-GP prophylaxis, and the mean duration of follow-up was 24 months (range, 11-30 months). There was a significant reduction in ABR (p-value, 0.002), AJBR (p-value, 0.022), and improvement in HJHS, pedHAL score, and reduction in school absenteeism in these children. Four patients experienced bleeding, which required treatment. Two patients developed an inhibitor to N9-GP. Mild adverse effects were noted in 20% of the patients. N9-GP prophylaxis compared to PD/r FIX replacement is a safe and effective strategy in prevention of bleeds, improving QOL and reducing joint related disability in pediatric patients with Hemophilia B.