At enrollment, the patient (Figure 4A) was 11 years old. After completing the trial, he entered pathfinder 8 and is currently studying at university. Prior to pathfinder 5 enrollment, the patient was receiving SHL rFVIII twice weekly but continued to experience bleeds on this PPX regimen. Of note, his estimated FVIII trough level was 2.5% at 48 hours and, as such, it was estimated that his levels would be <1% at 72 hours. According to the treating physician, during the 12 months prior to pathfinder 5 inclusion his ABR was 3, with all bleeds involving traumatic injuries to joints. Without N8-GP treatment his physician felt that he would have needed to escalate to SHL rFVIII injections three times per week, which the patient was keen to avoid.
During the 5 years that he received N8-GP in pathfinder 5, the patient had an ABR of 1 and a trough FVIII level of 2.9%. He was initially able to maintain twice-weekly injections and this supported his participation in sports. Later sports became his priority and he started to play American football and rugby. To support this lifestyle, he was escalated to three injections per week to provide higher FVIII trough levels when he transitioned to pathfinder 8 at around 16 years of age. Now at 18 years of age, he is once again less active (mainly due to COVID-19 pandemic) and has reverted back to a twice-weekly regimen.