Objective:
To assess treatment of young adult (YA) patients with hemophilia (PWH) and issues around access to and use of factor and comprehensive care.
Methods:
Analysis of US respondents aged 18-30 years in the international HERO study conducted in 2010-2011.
Summary:
Of 189 adult PWH HERO respondents in the United States, 66 were aged 18-30 years. More YA-PWH were on prophylaxis (50%) than on-demand alone (24%) or with occasional short-term prophylaxis (24%). Only 27% used treatment medication exactly as prescribed, with 27% a little less, and 21% varying (sometimes more/less). Twenty-six percent reported issues with access to factor in the prior 5 years due to availability or affordability, with 82% citing financial issues. YA-PWH reported a median of 2 bleeds in the prior month and median (IQR) annual bleed rate of 9.5 (12-22) bleeds. Similar to older adults, 80% of YA-PWH reported that a single joint suffers more bleeds than others; the most common joints reported were the ankle (77%), elbow (26%), and knee (21%). YA-PWH rated perceived disease control (1-10 scale, 10=most control) as median (IQR) 8 (7-9). HTC visit frequency was median (mean) 1 (1.66) per year. Similar to older adults (aged >40 years), 21% of YA-PWH reported difficulty in visiting the HTC. Accessibility was the most common reason (79%)—distance to travel (57%) or time to travel (29%); time constraints were more commonly reported by YA-PWH (57%), including being unable to get time off work (50%). When identifying health care professionals (HCPs) involved in management of their hemophilia, YA-PWH named hematologists (83%), nurses (67%), social workers (48%), counselors/psychologists (30%), and physical therapists (26%). The majority were satisfied with care provided by HCPs. YA-PWH reported being very/somewhat knowledgeable about hemophilia (91%). When looking to the future (pessimistic=1 to optimistic=7), YA-PWH were generally optimistic, with median (IQR) 5 (5-7).
Conclusions:
YA-PWH in the United States are more likely to be on prophylaxis than older adults, but less likely to use medication exactly as prescribed. Additional research is warranted to better understand why prescribed regimens are not followed. Despite 50% prophylaxis use, bleeding occurred ~1-2 times/month and YA-PWH reported high perceived disease control. During this period of transition to independence, it is important to note one quarter reported issues around access to treatment and one fifth reported difficulty in visiting the HTC. YA-PWH were satisfied with care, but infrequently reported social workers and physical therapists as part of management.