Health‐Related Quality of Life, Physical Activity and Joint Health in People With Severe Haemophilia A Receiving Emicizumab: Results From the Phase IV HemiNorth 2 Study

Introduction Despite factor (F)VIII prophylaxis, a perceived increased risk of bleeding for some people with severe haemophilia A (PwSHA) exists, limiting physical activity (PA) and restricting quality of life (QoL). Aim HemiNorth 2 (EudraCT# 2020‐003256‐32) is an interventional study evaluating the impact of switching from FVIII prophylaxis to emicizumab in PwSHA without FVIII inhibitors who have a need for improved prophylaxis in the Nordic countries. Methods Following completion of the HemiNorth non‐interventional study (NIS), eligible participants (aged ≥ 12–61 years) were enrolled in HemiNorth 2. The primary endpoint was health‐related QoL via the Comprehensive Assessment Tool for Challenges in Hemophilia (CATCH). Secondary endpoints included PA (International Physical Activity Questionnaire‐Short Form [IPAQ‐SF]), treatment preference (Emicizumab Preference [EmiPref] survey), joint health, model‐based annualised bleeding rates (ABRs) and adverse events. Results Overall, 28 physically active male PwSHA were enrolled. Most baseline CATCH domains were ≤ 25 and remained consistent; mean treatment burden considerably improved from baseline for adults (−17.8) and adolescents (+16.7). IPAQ‐SF scores were consistent throughout the study. Overall, 23 of 25 (92.0%) EmiPref respondents preferred emicizumab over FVIII prophylaxis. Model‐based ABRs for treated bleeds decreased from 5.9 (95% confidence interval [CI]: 3.8–9.1) to 1.6 (95% CI: 0.9–3.0) from the NIS to HemiNorth 2, and participants with zero treated bleeds increased from 8 (28.6%) to 16 (57.1%). No new safety signals were reported. Conclusions Emicizumab improved treatment burden and was preferred by most participants over FVIII prophylaxis. PA levels were consistently high, and bleeding rates improved with emicizumab versus prior FVIII prophylaxis.