Key Findings: Study of Hemophilia Care Outcomes over 50 Year Span Reveals Progress, Ongoing Health Challenges

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New study shows medical care, treatment and support services for hemophilia have improved over the past 50 years. Yet people affected by this disorder still have health challenges. Read more of what we learned.

Medical care, treatment and support services for men and boys with hemophilia have improved over the last five decades. Yet people affected by this disorder still experience significant health challenges throughout their lifetime.

To continue the medical care for this population, it’s important to keep systems in place to monitor and learn more about the health of people with hemophilia. This knowledge can then be used to develop programs to improve health and quality of life for those living with hemophilia.

About This Study

Researchers from the Centers for Disease Control and Prevention (CDC) and the U.S. Hemophilia Treatment Center Network conducted a study of the health of men (19 years or older) with hemophilia in the United States (U.S). The study aimed to find out the links between

• Changes in hemophilia treatment and healthcare delivery over the last fifty years, and
• Factors that affect health (such as access to care, physical and social functioning, health problems, and death)

Researchers analyzed information collected from 4,899 men with severe and 2,587 men with mild hemophilia. These men received care at U.S. hemophilia treatment centers from 1998-2011. Data were organized into four time periods (called Eras) that reflected major developments in therapies and health care for people with hemophilia. Listed below are these Eras from oldest to youngest:

• Era A included men born prior to 1958 (oldest participants)
• Era B included men born 1958-1975
• Era C included men born 1976-1982
• Era D included men born 1982-1993 (youngest participants)

For men with severe versus mild hemophilia within each Era, study researchers compared

• Demographic information, such as race and ethnicity;
• Healthcare access information, such as age at diagnosis and type of health insurance;
• Hemophilia-related and treatment-related health problems; and
• Number and causes of death.

Read the hemophilia study’s manuscript

Main Findings From This Study

• In the youngest age group (Era D) more than 1 in 3 men with severe hemophilia reported frequent bleeds (more than 5 bleeds in six months) despite being treated with the most modern therapies. One in 4 of these men also reported a recurrent bleed in a target joint (a bleed that occurs often or repeatedly in a particular joint).
• Across all Eras, compared to men with mild disease, those with severe hemophilia were about three times more likely to report activity limitations, and twice as likely to report some use of devices to help them move around, such as a cane or wheelchair.
• In every Era, the proportion of men with severe hemophilia that 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 the oldest age group (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 are 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. Hemophilia-related deaths (bleeds) 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.

Critical Gaps & Future Directions

Gaps

A number of observations from this study need further investigation

• Joint bleeding among men with hemophilia is more common than expected given widespread availability of proven therapies that are accessible for home use.
• Treatment of hemophilia through the regular infusion or injection of clotting factor concentrates into a vein to prevent bleeding (called prophylaxis), has been shown to result in better joint outcomes. However, this study found that men with hemophilia in all Eras tended to under-utilize prophylaxis.
• The differences in overall health between men with mild and severe hemophilia has not changed over time.
• There are subgroups of men in all Eras with mild hemophilia who experience higher levels of bleeding than expected.

Future directions

Research is needed to understand the reasons for the apparent underutilization of prophylaxis by some men with hemophilia, and to develop strategies that are successful in changing behavior.

More work is needed to understand the factors that contribute to joint bleeds and to develop treatment strategies for men with hemophilia who have a higher risk for joint disease.

Focused studies are needed on men with mild hemophilia who have unusually high rates of bleeding in order to identify the reasons behind their increased bleeding tendency.

More Information

Please visit the following links for more information about

Hemophilia

https://www.cdc.gov/ncbddd/hemophilia/index.html

Inhibitors

https://www.cdc.gov/ncbddd/hemophilia/inhibitors.html

Research on inhibitors

https://www.cdc.gov/ncbddd/hemophilia/features/inhibitors.html and https://www.cdc.gov/ncbddd/hemophilia/research.html

Inhibitor testing

http://www.hemophilia.org/Newsroom/Medical-News/CDC-Hosts-Inhibitor-Testing-Webinar

Key Findings Reference

Mazepa MA, Monahan PE, Baker JR, Riske BK, and Soucie JM. Men with severe hemophilia in the United States: Birth cohort analysis of a large national database. Blood. Jan 2016, DOI: 10.1182/blood-2015-10-675140