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Efficacy and safety of low-dose sirolimus for treatment of lymphangioleiomyomatosis.
[lymphangioleiomyomatosis]
Lymphangioleiomyomatosis
(
LAM
)
is
a
rare
disease
caused
by
dysregulated
activation
of
the
mammalian
target
of
rapamycin
(
mTOR
)
.
Sirolimus
,
an
inhibitor
of
mTOR
,
has
been
reported
to
decrease
the
size
of
angiomyolipomas
and
stabilize
pulmonary
function
in
patients
with
LAM
.
However
,
the
optimal
dose
for
the
treatment
of
LAM
remains
unclear
.
We
conducted
a
retrospective
,
observational
study
of
15
patients
with
LAM
who
underwent
sirolimus
therapy
for
more
than
6
months
.
The
efficacy
was
evaluated
by
reviewing
the
patients
'
clinical
courses
,
pulmonary
function
and
chest
radiologic
findings
before
and
after
the
initiation
of
sirolimus
treatment
.
All
patients
had
blood
trough
levels
of
sirolimus
lower
than
5
ng
/
mL
.
Sirolimus
treatment
improved
the
annual
rates
of
change
in
FVC
and
FEV
1
in
the
9
patients
who
were
free
from
chylous
effusion
(
FVC
,
-
101
.
0
vs
.
+
190
.
0
mL
/
y
,
p
=
0
.
046
and
FEV
1
,
-
115
.
4
vs
.
+
127
.
8
mL
/
y
,
p
=
0
.
015
)
.
The
remaining
7
patients
had
chylous
effusion
at
the
start
of
sirolimus
treatment
;
the
chylothorax
resolved
completely
within
1
-
5
months
of
treatment
in
6
of
these
cases
.
These
results
resembled
those
of
previous
studies
in
which
blood
trough
levels
of
sirolimus
ranged
from
5
to
15
ng
/
mL
.
Low
-dose
sirolimus
(
trough
level
,
5
ng
/
mL
or
less
)
performed
as
well
as
the
higher
doses
used
previously
for
improving
pulmonary
function
and
decreasing
chylous
effusion
in
patients
with
LAM
.
Diseases
Validation
Diseases presenting
"dysregulated activation of the mammalian target of rapamycin"
symptom
lymphangioleiomyomatosis
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