Rare Diseases Symptoms Automatic Extraction
Home
A random Abstract
Our Project
Our Team
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
"pulmonary function"
symptom
allergic bronchopulmonary aspergillosis
cohen syndrome
congenital diaphragmatic hernia
inclusion body myositis
lymphangioleiomyomatosis
systemic capillary leak syndrome
You can validate or delete this automatically detected symptom
Validate the Symptom
Delete the Symptom