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Clinical course of patients with Fabry disease who were switched from agalsidase-β to agalsidase-α.
[fabry disease]
Between
2009
and
2012
,
there
was
a
worldwide
shortage
of
agalsidase-β
for
the
treatment
of
Fabry
disease
.
Therefore
,
alternative
treatments
were
needed
,
including
switching
to
a
different
enzyme-replacement
therapy
.
This
is
an
ongoing
observational
study
assessing
the
effects
of
switching
from
agalsidase-β
(
1
.
0
 
mg
/
kg
every
other
week
)
to
agalsidase-α
(
0
.
2
 
mg
/
kg
every
other
week
)
in
11
patients
with
Fabry
disease
.
Clinical
data
were
collected
for
5
years
-
2
years
before
switching
and
3
years
after
switching
.
Measures
of
renal
function
such
as
estimated
glomerular
filtration
rate
remained
stable
during
the
3
years
after
switching
to
agalsidase-α
.
Improvements
in
cardiac
mass
were
recorded
in
both
male
and
female
patients
12
months
after
switching
to
agalsidase-α
,
and
the
benefit
was
maintained
during
36
months
of
follow-up
.
There
was
no
significant
difference
in
the
severity
of
pain
experienced
by
patients
before
and
after
switching
enzyme-replacement
therapy
,
and
no
difference
in
quality-of-life
parameters
.
Agalsidase-α
was
generally
well
tolerated
,
and
no
patients
experienced
allergy
or
developed
antibodies
to
agalsidase-α
.
This
observational
study
supports
the
safety
of
switching
from
agalsidase-β
to
agalsidase-α
at
the
approved
doses
,
with
no
loss
of
efficacy
.
It
also
suggests
that
if
an
infusion-related
allergic
reaction
occurs
in
a
patient
receiving
agalsidase-β
,
switching
to
agalsidase-α
may
be
a
viable
option
.
Genet
Med
16
10
,
766
-
772
.
