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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
.