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Retrospective Review Of Combined Sirolimus And Simvastatin Therapy In Lymphangioleiomyomatosis.
[lymphangioleiomyomatosis]
Background
.
Combined
simvastatin
and
sirolimus
therapy
reduce
TSC
2
-
null
lesions
and
alveolar
destruction
in
a
mouse
model
of
lymphangioleiomyomatosis
(
LAM
)
,
suggesting
that
therapy
with
both
drugs
may
benefit
LAM
patients
.
Methods
.
To
determine
whether
simvastatin
changed
the
prevalence
of
adverse
events
or
altered
the
therapeutic
effects
of
sirolimus
,
we
recorded
adverse
events
and
changes
in
lung
function
in
LAM
patients
treated
with
simvastatin
plus
sirolimus
(
n
=
14
)
,
sirolimus
(
n
=
44
)
,
or
simvastatin
(
n
=
20
)
.
Results
.
Sirolimus-related
adverse
events
in
the
simvastatin
plus
sirolimus
,
and
sirolimus
only
groups
,
were
64
and
66
%
for
stomatitis
,
50
and
52
%
for
diarrhea
,
50
and
45
%
for
peripheral
edema
,
36
and
61
%
for
acne
,
36
and
30
%
for
hypertension
,
29
and
27
%
for
proteinuria
,
29
and
27
%
for
leukopenia
,
and
21
and
27
%
for
hypercholesterolemia
.
The
frequency
of
simvastatin-related
adverse
events
in
the
simvastatin
,
and
simvastatin
plus
sirolimus
groups
were
60
and
50
%
for
arthralgias
,
and
35
and
36
%
for
myopathy
.
Before
simvastatin
plus
sirolimus
therapy
,
FEV
1
and
DLCO
yearly
rates
of
change
were
respectively
,
-
1
.
4
±
0
.
2
and
-
1
.
8
±
0
.
2
%
predicted
.
After
simvastatin
plus
sirolimus
therapy
,
these
rates
changed
to
+
1
.
2
±
0
.
5
(
p
=
0
.
635
)
and
+
0
.
3
±
0
.
4
%
predicted
,
respectively
(
p
=
0
.
412
)
.
In
44
patients
treated
with
sirolimus
alone
,
FEV
1
and
DLCO
rates
of
change
were
-
1
.
7
±
0
.
1
and
-
2
.
2
±
0
.
1
%
predicted
before
treatment
,
and
+
1
.
7
±
0
.
3
and
+
0
.
7
±
0
.
3
%
predicted
after
therapy
(
p
<
0
.
001
)
.
Conclusions
.
Therapy
with
sirolimus
and
simvastatin
does
not
increase
the
prevalence
of
drug
adverse
events
or
alter
the
therapeutic
effects
of
sirolimus
.
Combined
simvastatin
and
sirolimus
therapy
reduce
TSC
2
-
null
lesions
and
alveolar
destruction
in
a
mouse
model
of
lymphangioleiomyomatosis
(
LAM
)
,
suggesting
that
therapy
with
both
drugs
may
benefit
LAM
patients
.
To
determine
whether
simvastatin
changed
the
prevalence
of
adverse
events
or
altered
the
therapeutic
effects
of
sirolimus
,
we
recorded
adverse
events
and
changes
in
lung
function
in
LAM
patients
treated
with
simvastatin
plus
sirolimus
(
n
=
14
)
,
sirolimus
(
n
=
44
)
,
or
simvastatin
(
n
=
20
)
.
Sirolimus-related
adverse
events
in
the
simvastatin
plus
sirolimus
,
and
sirolimus
only
groups
,
were
64
and
66
%
for
stomatitis
,
50
and
52
%
for
diarrhea
,
50
and
45
%
for
peripheral
edema
,
36
and
61
%
for
acne
,
36
and
30
%
for
hypertension
,
29
and
27
%
for
proteinuria
,
29
and
27
%
for
leukopenia
,
and
21
and
27
%
for
hypercholesterolemia
.
The
frequency
of
simvastatin-related
adverse
events
in
the
simvastatin
,
and
simvastatin
plus
sirolimus
groups
were
60
and
50
%
for
arthralgias
,
and
35
and
36
%
for
myopathy
.
Before
simvastatin
plus
sirolimus
therapy
,
FEV
1
and
DLCO
yearly
rates
of
change
were
respectively
,
-
1
.
4
±
0
.
2
and
-
1
.
8
±
0
.
2
%
predicted
.
After
simvastatin
plus
sirolimus
therapy
,
these
rates
changed
to
+
1
.
2
±
0
.
5
(
p
=
0
.
635
)
and
+
0
.
3
±
0
.
4
%
predicted
,
respectively
(
p
=
0
.
412
)
.
In
44
patients
treated
with
sirolimus
alone
,
FEV
1
and
DLCO
rates
of
change
were
-
1
.
7
±
0
.
1
and
-
2
.
2
±
0
.
1
%
predicted
before
treatment
,
and
+
1
.
7
±
0
.
3
and
+
0
.
7
±
0
.
3
%
predicted
after
therapy
(
p
<
0
.
001
)
.
Therapy
with
sirolimus
and
simvastatin
does
not
increase
the
prevalence
of
drug
adverse
events
or
alter
the
therapeutic
effects
of
sirolimus
.
Diseases
Validation
Diseases presenting
"leukopenia"
symptom
cohen syndrome
dedifferentiated liposarcoma
lymphangioleiomyomatosis
pleomorphic liposarcoma
waldenström macroglobulinemia
This symptom has already been validated