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Introduction: Standardized and disease‐specific patient‐reported outcome (PRO) instruments assessing pain, functional impairment and health‐related quality of life (HRQoL) in people with haemophilia (PWH) have been used in studies, but infrequently in comprehensive care settings for individual assessment or treatment planning. Aim: To assess the impact of pain and functional impairment on HRQoL in PWH. Methods: P‐FiQ enrolled 381 adult PWH with a history of joint pain/bleeding and included 5 PROs and a clinical joint evaluation (Hemophilia Joint Health Score v2.1 [HJHS]). Results: Median age was 34 years; 49.9% reported a history of joint procedure or surgery. On EQ‐5D‐5L, most reported problems with mobility (61.4%), usual activities (53.2%) and pain/discomfort (76.1%). On Brief Pain Inventory v2 Short Form, median worst pain (range 0‐10) was 6, least pain 1, average pain 3 and current pain 2. Ankles were most frequently reported as the most painful joints (37.4%), followed by knees (23.7%) and elbows (18.9%). On International Physical Activity Questionnaire, 51% reported no activity in the prior week. On SF‐36v2 health survey, median subscores were worse for 4 physical health domains vs 4 mental health domains. Among Hemophilia Activities List domains (range 0 [worst]‐100 [best]), functions of the legs (median, 66.7) and lying/sitting/kneeling/standing (median, 67.5) were most impacted and self‐care least impacted (median, 100.0). On HJHS, ankle scores (median, 6.0; range, 0‐40) were worse than elbow/knee scores (median, 4.0/4.0). Results were consistent across PROs/HJHS. Conclusion: Data demonstrate challenges of predominantly ankle/knee pain and lower extremity functional impairment in US adult PWH, affecting HRQoL across PROs/HJHS. [ABSTRACT FROM AUTHOR]

Recombinant activated factor VII ( rFVIIa) is indicated for treatment of bleeding in congenital haemophilia with inhibitors (CHwI) using 90 μg kg−1 every 2-3 h (EU and US) or a single 270 μg kg−1 dose (EU only) with ~90% efficacy reported for both regimens. Dosing of rFVIIa varies, and home treatment makes assessment of frequency of doses >90 μg kg−1, the intervals before additional treatment, and the risk for thromboembolic events (TEs) more difficult. This post hoc analysis assessed the safety and distribution of rFVIIa dosing in CHwI and the impact of >240 μg kg−1 dosing on subsequent bypassing agent (BPA) dosing interval and frequency. Data regarding on-demand or prophylactic rFVIIa dosing, TE incidence and subsequent BPA dosing after high rFVIIa doses were compiled from multiple sources incorporating safety surveillance. A total of 61 734 rFVIIa doses were reported in 481 patients treated for 3947 bleeds and for 43 135 prophylaxis days. Over half (52%) exceeded 120 μg kg−1, 37% exceeded 160 μg kg−1 and 15% exceeded 240 μg kg−1. Subsequent doses of BPA(s) were administered after 38% of initial and 49% of any rFVIIa dose >240 μg kg−1, and were most frequently administered ≥24 h after initial (40%) or any (53%) doses >240 μg kg−1. No TEs were reported. The findings of this analysis show that rFVIIa doses >90 μg kg−1 are utilized for 'real-world' treatment of children and adults. When additional BPA was administered following an rFVIIa dose >240 μg kg−1, reported intervals were prolonged, often ≥24 h. No safety issues were identified in the 61 734 doses analysed. [ABSTRACT FROM AUTHOR]