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Clinical observations on enzyme replacement therapy in patients with Fabry disease and the switch from agalsidase beta to agalsidase alfa.
[fabry disease]
Fabry
disease
is
an
X-
linked
inherited
lysosomal
storage
disease
that
can
be
treated
with
the
enzymes
of
agalsidase
beta
(
Fabrazyme
)
and
agalsidase
alfa
(
Replagal
)
.
Since
June
2009
,
viral
contamination
of
Genzyme
's
production
facility
has
resulted
in
a
worldwide
shortage
of
agalsidase
beta
,
leading
to
the
switch
to
agalsidase
alfa
for
patients
with
Fabry
disease
in
Taiwan
.
T
he
medical
records
were
retrospectively
reviewed
for
nine
male
patients
with
Fabry
disease
from
the
start
of
agalsidase
beta
treatment
until
the
switch
to
agalsidase
alfa
for
at
least
1
year
.
After
12
-
112
months
of
enzyme
replacement
therapy
(
ERT
)
,
decreased
plasma
globotriaosylsphingosine
(
lyso-
Gb
3
)
was
found
in
five
out
of
seven
patients
,
indicating
improvement
in
disease
severity
.
Among
the
six
patients
with
available
echocardiographic
data
at
baseline
and
after
ERT
,
all
six
experienced
reductions
of
left
ventricular
mass
index
.
Renal
function
,
including
microalbuminuria
and
estimated
glomerular
filtration
rate
,
showed
stability
after
ERT
.
Mainz
Severity
Score
Index
scores
revealed
that
all
nine
patients
remained
stable
at
12
months
after
switching
to
agalsidase
alfa
.
ERT
improved
or
stabilized
cardiac
status
and
stabilized
renal
function
,
while
reducing
plasma
lyso-
Gb
3
.
ERT
was
well
tolerated
,
even
among
the
three
patients
who
had
hypersensitivity
reactions
.
The
switch
of
ERT
from
agalsidase
beta
to
agalsidase
alfa
appears
to
be
safe
after
1
year
of
follow-up
for
Taiwanese
patients
with
Fabry
disease
.
Diseases
Validation
Diseases presenting
"renal function"
symptom
aniridia
cholangiocarcinoma
cystinuria
erdheim-chester disease
fabry disease
familial hypocalciuric hypercalcemia
junctional epidermolysis bullosa
kabuki syndrome
megacystis-microcolon-intestinal hypoperistalsis syndrome
primary hyperoxaluria type 1
scrub typhus
systemic capillary leak syndrome
von hippel-lindau disease
waldenström macroglobulinemia
wolf-hirschhorn syndrome
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