CLINICAL NEWS
TABLE 1.
Disease-Related Complications
Total number of patients
Era A
Era B
Era C
Era D
<1958
1958–1975
1976–1982
1983–1992
Severe
Mild
Severe
Mild
Severe
Mild
Severe
Mild
688
843
1,542
787
1,121
427
1,548
530
Employment and Disability Status
Disabled
45.9%
12.9%
37.8%
14.7%
15.9%
3.5%
5.8%
1.9%
Employed/
Student
31.2%
49.5%
53.1%
74.5%
68.6%
86.4%
79.6%
87.6%
Retired
18.3%
31.7%
0.4%
0.5%
0%
0%
0%
0%
Other
3.5%
5.9%
8.6%
10.3%
15.5%
10.1%
14.5%
10.6%
Limitation to overall
activity level
68.8%
21.1%
49.4%
16.3%
25.9%
6.6%
14.9%
4.3%
School/work absenteeism (>10 days missed)
6.9%
2.6%
8.5%
5.2%
10%
5.6%
5.6%
3%
Assistive devices: intermittent
39.8%
16%
35.1%
15.4%
28.8%
13.6%
22.9%
11.3%
Assistive devices: always
18.5%
4.6%
6.6%
1.5%
1.7%
0%
1.4%
0.8%
≤2 joint bleeds in last six
months
45.8%
97.2%
38.1%
92.9%
41.3%
92%
51.7%
94.7%
≥5 joint bleeds in last six
months
42.6%
1.7%
48.8%
4.3%
46.6%
3.8%
35.5%
3%
Target joint
32.6%
3.2%
36.2%
7.2%
35.6%
5.8%
24.9%
2.3%
Physical Function
Bleeding Complications
TABLE 2.
Treatment-Related Complications
Era A
Era B
Era C
Era D
<1958
1958–1975
1976–1982
1983–1992
Severe
Mild
Severe
Mild
Severe
Mild
Severe
Mild
688
843
1,542
787
1,121
427
1,548
530
HBV
70.5%
33%
71.7%
34.4%
38.4%
9.1%
5.2%
2.4%
HCV
92.3%
54.2%
93%
60.6%
84.4%
33.3%
32.6%
6.2%
HBV and HCV
co-infection
69.2%
27.9%
61.2%
31.5%
34.2%
6.1%
3.7%
0.4%
HIV
42.9%
4.7%
61%
11.9%
26.2%
4%
1%
0.2%
HIV and HCV
co-infection
42.2%
4.2%
59%
10.8%
23.3%
3.7%
0.7%
0%
Not infected with HIV,
HBV, or HCV
5.5%
40.7%
4.1%
35.8%
10.4%
63.7%
65.4%
91.5%
17%
3.3%
13.6%
3.6%
11.5%
2.8%
15.6%
2.8%
Total number of
patients
Viral Infections
Inhibitor Development
Any inhibitor recorded
HBV = hepatitis B virus; HCV = hepatitis C virus; HIV = human immunodeficiency virus
recombinant coagulation
factor therapy since 1992.
The highest prevalence
of HIV infection occurred
among men born during
era B, and the prevalence
declined in each successive era – to 1 percent in
era D.
(See TABLE 1 and TABLE
2 for more details on
disease- and treatmentrelated complications for
each birth cohort.)
A total of 551 deaths
were reported during the
ASHClinicalNews.org
study period, with era A
and era B accounting for
82 percent of deaths in
the severe hemophilia
population and 96 percent
of deaths in the mild cohort. Liver failure was the
most commonly reported
cause of death overall for
both severe (33%) and
mild (26%) disease.
“If hemophilia treatment and access to treatment for men with severe
hemophilia had improved
markedly over the last
several decades, then
we would expect health
outcomes disparities to
narrow over time,” said Dr.
Mazepa and co-authors.
“Despite improved access
to comprehensive care
and to pathogen-free clotting factor for those born
in recent decades, the gap
between severe and mild
has not narrowed either
for target joints or for the
use of assistive devices
for mobility. These “disappointing” observations in-
dicate that there is a need
for continued improvement in strategies for
prevention and treatment
of hemophilia-associated
hemorrhage.
One limitation of this
large registry data analysis is that patients had to
survive until 1998 to be
included in the analysis;
evaluating only survivors
could underestimate
the adverse outcomes in
severe disease and in the
older eras. Interpretation
of some data is limited
by the lack of historical
data and treatment data,
for example, whether a
patient had past inhibitors
or the details of secondary
or primary prophylaxis. ●
REFERENCE
Mazepa MA, Monahan PE, Baker JR, et al.
Men with severe hemophilia in the United
States: Birth cohort analysis of a large
national database. Blood. 2016 March 16.
[Epub ahead of print]
ASH Clinical News
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