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Antiproteinuric effect of add-on paricalcitol in Fabry disease patients: a prospective observational study.
[fabry disease]
Proteinuria
is
the
predominant
risk
factor
for
renal
disease
progression
in
Fabry
disease
(
FD
)
.
When
urine
protein
excretion
is
controlled
to
<
0
.
50
g
/
24
h
,
the
rate
loss
of
glomerular
filtration
rate
(
GFR
)
is
not
significantly
different
from
0
.
However
,
enzyme
replacement
therapy
(
ERT
)
alone
does
not
decrease
proteinuria
and
it
has
been
recommended
that
patients
receiving
ERT
also
receive
anti-
renin
-angiotensin
system
(
RAS
)
therapy
.
Emerging
evidence
show
that
paricalcitol
(
PCT
)
reduces
proteinuria
in
the
presence
of
intensified
inhibition
of
RAS
;
however
,
there
is
no
evidence
in
FD
.
We
evaluated
the
antiproteinuric
effect
of
PCT
in
FD
patients
with
proteinuria
>
0
.
50
g
/
24
h
persisting
despite
ERT
and
anti-
RAS
therapy
titrated
to
maximum
tolerated
dosage
.
Fifteen
FD
patients
were
selected
and
studied
in
the
first
6
months
of
add-on
oral
PCT
(
1
µg
/
day
)
and
,
in
order
to
verify
the
dependence
of
proteinuria
reduction
on
PCT
,
3
months
after
drug
withdrawal
.
At
baseline
,
proteinuria
was
1
.
3
±
0
.
6
g
/
24
h
.
Six
months
of
add-on
PCT
significantly
decreased
proteinuria
to
0
.
4
±
0
.
3
g
/
24
h
,
with
levels
<
0
.
50
g
/
24
h
achieved
in
four
patients
at
Month
1
,
six
at
Month
3
,
and
in
12
by
Month
6
,
in
the
absence
of
changes
to
BP
and
GFR
.
Proteinuria
recovered
to
basal
value
after
drug
withdrawal
.
In
conclusion
,
our
study
is
the
first
evidence
that
PCT
is
effective
in
reducing
proteinuria
in
FD
patients
in
the
presence
of
ERT
and
anti-
RAS
therapy
.
Diseases
Validation
Diseases presenting
"first evidence"
symptom
congenital adrenal hyperplasia
dentin dysplasia
esophageal adenocarcinoma
fabry disease
homocystinuria without methylmalonic aciduria
krabbe disease
liposarcoma
phenylketonuria
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