The HERO initiative was developed to increase understanding of the psychosocial issues impacting people with moderate-severe hemophilia (PWH).
Following ethics review and informed consent, 675 adult PWH (≥ 18) and 561 parents of children with hemophilia (CWH) in 10 countries completed ∼45 min psychosocial assessment including EQ-5D, a health-related visual analog scale (0–100, coded as an eleven-point categorical response), and a 7 point scale assessing pessimistic (1) to optimistic (7) outlook on the next 5 years. US participants were recruited through National Hemophilia Foundation's eNote distribution and Facebook.
The 189 US PWH had a median (range) age of 35(18–74). The 190 US parents of CWH had a median (range) age of 37(23–59) years; mean age of oldest son <18 was 8.6. Most PWH/parents reported hemophilia A (111/126, 59%/66%) with 45/18 (24%/9%) reporting inhibitors. Most parent respondents were female (79%) and responsible for their son's care (75%). PWH reported bone/arthritis (48%), pain (39%), HCV 34%, HIV 16%, and psychological (35%) comorbidities related to hemophilia; 40% reported cardiovascular conditions. PWH/parents completed full time education (84%/88%) or were in school (12%/6%). Most PWH/parents were employed full- or part-time (72%/72%). Of PWH, 13% reported long-term disability and 24% disability-related benefits. Many PWH/parents (44%/30%) reported their selection of a job took into account caring for their/their son's hemophilia; few reported no impact on choice of job (25%/22%) or working life (17%/33%). Most PWH/parents reported office-based jobs (57%/47%) or light-to-moderate manual labor (23%/44%). Many felt current treatment allowed them to work in most situations (36%/43%). Negative impacts included voluntarily leaving a job (30%/19%), not being hired (21%/7%), not being promoted (17%/12%) or losing a job (16%/11%). Adult PWH tended to have a lower risk profile for current activities/sports than CWH (low/medium/high risk: PWH 81%/49%/17% vs. CWH 67%/66%/40%). Common activities for PWH/CWH included: swimming (28%/46%), fishing (38%/34%), walking (33%/24%), golf (29%/19%), cycling (22%/29%), gym (23%/11%), and Frisbee (17%/22%). The mean (SD) EQ-5D index of PWH was 0.763 (0.195), median 0.800. While fewer older patients were surveyed, they had lower EQ-5D indexes:
Overall, 42% rated their health 80–100 (see figure). The percent rating 80–100 was lower for older PWH (<30, 53%; 31–40, 45%; 41+, 24%), those with inhibitors (24% vs. 48%), spontaneous bleeds in the past 12 months (39% vs. 61%), musculoskeletal comorbidities (36% vs. 78%) and those unemployed (20% vs. 47%).
On the EQ-5D components, 71% reported moderate or extreme pain or discomfort. Within the prior 4 weeks, 92% reported pain interfered with their daily life (54% moderate-extreme pain). Moderate-extreme interference was more common in PWH aged 41+ (76% vs. 46%), and with inhibitors (62% vs. 51%) or musculoskeletal complications (65% vs. 7%). Of the 92% reporting pain interfering with functioning, 43% reported only bleed-related pain, 17% continuous pain, and 36% continuous pain worsened by bleeds. The mean/median outlook on the next five years was optimistic for PWH (5.32/6) and parents (5.82/6) and didn't vary by age group. Mean ratings were lower for PWH with inhibitors (4.73 vs. 5.51) and not working (4.81 vs. 5.49). PWH/parents with inhibitors (56%/56%) more than those without inhibitors (46%/35%) agreed, “My whole life revolves around [my/my son's] hemophilia”.
HERO provides a unique database with demographic and medical/treatment data collected to facilitate in-depth study of psychosocial issues. Despite physical disability and challenges of caring, adult PWH and parents of CWH respectively overcome significant barriers to maintain employment. Adult PWH actively participate in low-medium risk activities, while CWH participate more fully. Using generic tools to allow comparability, impairment of QoL was consistent with other hemophilia studies, lower than in the general population and similar to normative data in elderly populations with chronic/disabling conditions. This was reflected in often constant pain impacting daily life. Adult PWH and parents of CWH were none-the-less generally optimistic about the future.
Nugent: Novo Nordisk: Membership on an entity's Board of Directors or advisory committees. Guelcher:Novo Nordisk: Membership on an entity's Board of Directors or advisory committees. Forsyth:Novo Nordisk: Consultancy, Membership on an entity's Board of Directors or advisory committees. Frick:National Hemophilia Foundation: Employment. Rice:National Hemophilia Foundation: Employment. Iorio:Pfizer: Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding, Speakers Bureau; NovoNordisk: Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding, Speakers Bureau; Baxter: Membership on an entity's Board of Directors or advisory committees; Bayer: Research Funding; BioGen Idec: Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding, Speakers Bureau. Wisniewski:Novo Nordisk Inc.: Employment. Cooper:Novo Nordisk Inc.: Employment.