Rare Diseases Symptoms Automatic Extraction
Home
A random Abstract
Our Project
Our Team
Outcomes of patients treated through the Canadian Fabry disease initiative.
[fabry disease]
The
Canadian
Fabry
disease
initiative
(
CFDI
)
tracks
outcomes
of
subjects
with
Fabry
disease
treated
enzyme
replacement
therapy
(
ERT
)
given
to
subjects
who
meet
evidence-based
treatment
guidelines
and
cardiovascular
risk
factor
modification
.
We
report
5
year
follow-up
data
on
362
subjects
for
a
composite
endpoint
(
death
,
neurologic
or
cardiovascular
events
,
development
of
end-
stage
renal
disease
or
sustained
increase
in
serum
creatinine
of
50
%
from
baseline
)
.
At
enrollment
,
86
subjects
had
previously
received
ERT
(
Cohort
1
a
)
and
67
subjects
were
newly
started
(
Cohort
1
b
)
and
randomized
to
agalsidase
alfa
or
agalsidase
beta
.
209
subjects
did
not
initially
meet
ERT
criteria
(
Cohort
1
c
)
,
25
of
whom
met
ERT
criteria
in
follow-up
and
were
moved
to
Cohort
1
b
(
total
N
=
178
ERT
treated
subjects
)
.
Use
of
supportive
therapies
such
as
aspirin
(
78
%
)
,
renin
-angiotensin
blockade
(
59
%
)
,
and
statins
(
55
%
)
was
common
in
ERT
treated
subjects
.
In
Cohort
1
a
,
32
subjects
met
the
composite
endpoint
with
8
deaths
.
In
Cohort
1
b
,
16
subjects
met
the
composite
endpoint
with
1
death
.
Cohort
1
b
had
fewer
clinical
events
than
Cohort
1
a
(
p
=
0
.
039
)
suggesting
that
the
treatment
protocol
was
effective
in
targeting
subjects
at
an
earlier
stage
.
19
.
4
%
of
Cohort
1
b
subjects
on
agalsidase
alfa
and
13
.
3
%
on
agalsidase
beta
had
a
clinical
event
(
p
=
0
.
57
)
.
10
Cohort
1
c
subjects
had
clinical
events
,
none
of
which
would
have
been
prevented
by
earlier
use
of
ERT
.
C
ardiovascular
risk
factor
modification
and
targeted
use
of
ERT
reduce
the
risk
of
adverse
outcomes
related
to
Fabry
disease
.
Diseases
Validation
Diseases presenting
"targeted use of ert reduce the risk of adverse outcomes related to fabry disease"
symptom
fabry disease
You can validate or delete this automatically detected symptom
Validate the Symptom
Delete the Symptom