case_images: PMC11085023_01
case_images: Patients (%) with zero spontaneous bleeding events over time for any dosing regimen or a twice-weekly regimen in children receiving long-term simoctocog alfa prophylaxis in the NuProtect-Extension.
case_images: 10.1177_20406207241245511-fig3.jpg
case_images: PMC11085023_01_10.1177_20406207241245511-fig3.jpg
case_images: fig3-20406207241245511
case_images: ABRs of 0.3 for spontaneous bleeds, 0.4 for joint bleeds, and 1.7 for all bleeds.   In PUPs, 102 patients in the NuProtect study who received at least one prophylactic dose had median ABRs of 0 for spontaneous bleeds and joint bleeds and 2.8 for all bleeds during inhibitor-free periods.   These low rates were maintained in the 47 children who entered and were treated in the NuProtect-Extension study, with ABRs of 0 for spontaneous bleeds and joint bleeds and 1.0 for all bleeds.   During the median follow-up of 24 months, 34 (72%) patients had zero spontaneous bleeds. The most common initial prophylaxis regimen in the NuProtect-Extension was twice weekly (68%), followed by three times weekly (11%), every other day (6%), or once weekly (15%). At study completion, 85% of patients were on a twice-weekly regimen, 12% were treated three times weekly, and 3% were treated once weekly.   Analysis of efficacy over time in the NuProtect-Extension study demonstrated that 86-97% of children across all treatment regimens, and 90-97% of those on a twice-weekly regimen, had zero spontaneous bleeds (Figure 3).  