Study of 50 Years of Hemophilia Healthcare Outcomes Yields Surprises

Results from a new study indicate that despite 50 years worth of advances in the area of comprehensive care for patients with bleeding disorders, males with hemophilia still grapple with significant health-related issues affecting their quality of life. The study, “Men with Severe Hemophilia in the United States: Birth Cohort Analysis of a Large National Database,” was published online on March 16 in the journal Blood. The lead author of the article was Paul E. Monahan, M.D., of the Gene Therapy Center at the University of North Carolina. Co-investigators included professionals from the hemophilia treatment center network and the Centers for Disease Control and Prevention.

To better understand the connections between the many changes in hemophilia healthcare and the dynamics affecting overall health, Monahan and his team reviewed data collected from 4,899 men with severe hemophilia and 2,587 men with mild hemophilia. All of these men had received care at HTCs from 1998-2011. Data were organized into four time periods, or “eras,” representing major healthcare developments and therapeutic breakthroughs relevant to people with hemophilia: Era A included the oldest group, men born prior to 1958; Era B grouped men born between 1958 and 1975; Era C included men born from 1976-1982; and Era D represented the youngest group, men born between 1982 and1993.

The main findings of the study included:

• In Era D, more than one in three men with severe hemophilia reported frequent bleeds (more than five bleeds in six months), despite being treated with the most modern therapies. One in four of these men also reported a recurrent bleed in a “target joint.
• Across all eras, compared to men with mild disease, those with severe hemophilia were about three times more likely to report activity limitations. Further, they were twice as likely to report some use of assistive devices to help them move around, such as a cane or wheelchair.
• In every era, the proportion of men with severe hemophilia who missed at least 10 days of work or school in the last year due to upper or lower joint problems was two or three times that of men with mild hemophilia.
• Nearly half of the men in Era A were disabled and unable to work. Moreover, men with severe hemophilia were about three times more likely to be disabled as their mild hemophilia counterparts in every era.
• Infection-related health problems due to hepatitis B, hepatitis C and HIV were common among men with severe hemophilia, particularly in the older eras.
• Of the 551 deaths reported during the study period, liver failure was the most commonly reported cause of death, regardless of hemophilia severity or era. Bleed-related deaths accounted for 14.6 percent of deaths in men with severe hemophilia and 10.7 percent of deaths in men with mild hemophilia across all eras.

The study also yielded some unexpected findings. The overall rates of joint bleeding remained relatively high, even with the availability of more effective treatments. Also, despite the proven effectiveness of prophylactic factor therapy in preventing joint damage, men in all eras continued to underuse this option.

“Clear disparities remain in terms of frequent bleeding and disability between men with severe hemophilia and mild hemophilia across every decade of adult life. We thought the difference in functional outcomes would have narrowed over the years; that is, men with severe hemophilia should look more like those with mild disorder, given improved therapeutics and access to care, but this wasn’t the case,” said Monahan. “What needs examination is why—despite widespread availability of preventive and on-demand therapies for home use—we still see disparities. It speaks to the need for continued disease surveillance to monitor and inform hemophilia interventions and outcomes.”