Impact of Mild to Severe Hemophilia B on Quality of Life Including Pain and Functional Abilities in Affected Men/Women and Caregivers of Affected Boys/Girls: Analysis of Patient Reported Outcomes in the Bridging Hemophilia B Experiences Results and Opportunities into Solutions (B-HERO-S) Study

Introduction: Health-related quality-of-life (HRQoL) is impaired in patients with severe congenital hemophilia due to pain and functional impairment associated with hemophilic arthropathy. The impact of mild or moderate hemophilia on HRQoL and on women with hemophilia in particular is less well characterized. Use of standardized instruments for assessment of pain/depression/anxiety also remains uncommon in patients with hemophilia.

Objective: B-HERO-S was designed to characterize factors that affect HRQoL in adult patients with hemophilia B (PWH) and caregivers (CG) of affected children (CWH).

Methods:US PWH (either gender, age ≥18 years) and CG (age >18 years) of CWH (either gender, age <18 years) were recruited through 3 patient organizations to complete distinct (PWH/CG) ~1 hour internet-based, IRB-approved surveys. In addition to descriptive surveys, adults completed 4 standardized patient reported outcome (PRO) instruments (EQ5D-5L with VAS [0‒100 scale] and calculated index [-0.11‒1.00], brief pain inventory [BPI SFv2, 0‒10 scale], PHQ9 [0‒27 score], and international physical activity questionnaire [IPAQ, low/moderate/high category]); and one disease-specific PRO (hemophilia activities list [HAL, 0‒100 score for domains]). Caregivers completed 2 standardized PROs for depression and anxiety (PHQ9, GAD7 [0‒21 score]).

Results PWH: 213 men and 86 women responded to the survey. Median age of participants was 29 years (range 18‒70 years). Severity was self-reported as mild/moderate/severe/inhibitor (74/189/32/4). Comorbidities associated with hemophilia were arthritis (48%), osteoporosis/fractures (43%/11%), pain (acute/chronic: 29%/13%), anxiety (23%), and depression (22%). Most were educated past high school (87%), employed (81%), and married/in a long-term relationship (54%). Despite the preponderance of mild/moderate PWH, most PWH had some pain (EQ5D-5L) and nearly all had problems with usual activities, anxiety/depression, mobility, and self-care (Table 1). PWH reported moderate pain severity/interference (median 5/5); higher scores were observed for women (7/7), PWH with moderate hemophilia (6/7), and those on routine infusions (6/6). Based upon PHQ9, at least mild depression was observed in >75% of the PWH respondents and more often in moderate and severe PWH. More women than men had depression, and those treated with routine infusions reported higher depression scores than those treated on demand. While 94% reported some physical activity, two-thirds were categorized as moderate or high activity; unexpectedly, there were more mild PWH reporting low activity and more severe PWH categorized as high. There were no apparent differences in upper and lower extremity functional ability (HAL) domains or composite scores across hemophilia severity or by gender; PWH on routine infusions had worse scores than those treated on demand.

Results CG:150 CG participants, all parents, included fathers/mothers (34/116) with median age 35 years (range 21‒53 years) describing their oldest children <18 years (boys/girls: 121/29; median age 10 years, range <1‒18 years), with mild/moderate/severe/inhibitor (27/84/33/6). Most CG were educated past high school (86%), employed (86%), and married/in a long-term relationship (89%). Based on PHQ9 results, more than half of CG reported at least mild depression; just under half reported mild anxiety according to the GAD7 scores (Table 2). Compared with the PWH results, the differences in depression and anxiety were much more pronounced in CG of children with moderate hemophilia and in mothers. CG of CWH treated with routine infusions vs. on demand reported more depression and anxiety.

Limitations: Voluntary response bias may limit generalizability of these findings as PWH/CG with greater impact may have been more likely to participate.

Conclusions: These findings indicate that pain, functional impairment, and depression/anxiety may contribute to the impact of hemophilia B on education, work, activity, and relationships described in prior reports from B-HERO-S. This is the first study to examine HRQoL in PWH of both genders and CG of affected CWH with mild-to-severe hemophilia B using multiple PRO instruments. These results suggest unmet needs particularly in mild/moderate PWH and women. Comparisons between known groups and analysis of HRQoL predictors are underway.

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