Severe hemophilia still tough to manage, study shows
A large study suggests that, despite treatment advances, men with severe hemophilia have not seen great reductions in bleeding events.
Data on more than 7000 men with hemophilia revealed substantial differences in the care received by men born before 1958 and those born between 1983
and 1992.
However, the data also showed that frequent bleeding was common in patients with severe hemophilia, regardless of when they were born.
These data were published in Blood.
“Our analysis provides a snapshot of how improvements in care have translated into outcomes across different generations of men with hemophilia,” said study author Paul E. Monahan, MD, of The University of North Carolina-Chapel Hill.
“While there is reason to be pleased with the progress we’ve made, our data show some surprising deficits and suggest that efforts are needed to more consistently apply the integration of standard-of-care multidisciplinary services and preventive blood clotting factor treatments to further normalize the lives of men living with hemophilia.”
Dr Monahan and his colleagues analyzed data on 7486 men—4899 with severe hemophilia (65.4%), 2587 with mild hemophilia (34.6%), 6094 with hemophilia A (81.4%), and 1392 with hemophilia B (18.6%).
The data were collected prospectively by the US Centers for Disease Control and Prevention and 130 federally supported Hemophilia Treatment Centers (HTCs) between 1998 and 2011. This represents the largest database of men living with hemophilia.
The researchers grouped the men into 4 eras (birth cohorts) to evaluate how outcomes—access to care, physical and social functioning, complications, and mortality—have changed over the last 50 years.
The cohorts were as follows:
- Era A: born before 1958 (median age 58)
- Era B: born between 1958 and 1975 (median age 40)
- Era C: born between 1976 and 1982 (median age 28)
- Era D: born between 1983 and 1992 (median age 21).
Access to care
The researchers found that the proportion of men who started home infusions before age 6 was far greater in Era D than Era A—50.4% and 2%, respectively. And the proportion of patients reporting a first HTC visit before age 2 rose nearly 10-fold from Era A to Era D—8.8% and 100%, respectively.
In addition, the use of a continuous prophylactic regimen was nearly 3-fold greater in Era D than Era A—46.7% and 16%, respectively.
However, patients in the youngest 2 birth cohorts were more likely than their older counterparts to be uninsured. The proportion of uninsured patients was 16.4% in Era D, 20.5% in Era C, 11.1% in Era B, and 5.7% in Era A.
Bleeding events
The proportion of patients reporting frequent bleeds decreased from Era A to Era D. However, frequent bleeding was common in men with severe hemophilia regardless of when they were born.
Even men from Era D—who had access to effective, safe clotting therapies and multidisciplinary care throughout their lifetimes—reported frequent bleeds.
The proportion of patients with severe hemophilia reporting 5 or more joint bleeds in the last 6 months was 42.6% in Era A and 35.5% in Era D. The proportion of patients with a joint affected by recurrent bleeding was 32.6% and 24.9%, respectively.
Functioning
The researchers also discovered that patients with severe hemophilia were 3 times as likely to report limitations in their activities or to be disabled, when compared to patients with mild hemophilia, regardless of when they were born.
Still, men from Era A were more likely to report limitations in their overall activity level—68.8% of severe hemophilia patients and 21.1% of mild hemophilia patients—than men from Era D—14.9% of severe hemophilia patients and 4.3% of mild hemophilia patients.
